SIP Report - Priyanka Saha - MR1 - 2019-2005-0001-0040

SUMMER INTERNSHIP
REPORT
A STUDY ON GENE THERAPY & ITS FUTURE PROSPECTS IN INDIA
done at
CHROMATUS CNSULTING
by
PRIYANKA SAHA
2019-2005-0001-0040
under the guidance of
VIJAY NATEKAR, Operation Head
&
BIBHAS B., Dean

Summer Internship Report, Pune Institute of Business Management, Pune
Acknowledgment
The meaning of adding this page at the beginning of my project is much than a clique
acknowledgment page. As this study comes to an end I feel indebted to all those who have lent a
helping hand during our research work.
I owe immensely to my external mentor Mr. Vijay Natekar for his valuable time, guidance &
encouragement during our entire project report.
I also want to express my sincere gratitude to my Internal mentor, Prof. Bibhas Basumatary for
his suggestions and conceptual knowledge imparted by him during my project.
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CERTIFICATE FROM THE CROMATUS CONSULTING
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(Certificate from the Institute- it should be in color. This header and

footer should look exactly same like this)
To Whomsoever It May Concern
This is to certify that the Project Report titled A Study on Gene Therapy &
its future prospects in India is an authentic work carried out by Ms.
Priyanka Saha from PGDM – MR1, Batch 2019-21 of IAEER’s Pune
Institute of Business Management, Pune as a fulfillment of MBA/ PGDM
Course. He/ She has worked under our guidance and satisfactorily
completed his/ her project work.
Place: Date:
Signature of Internal Guide
Signature of Director / COE
Contents
1. PROJECT SYNOPSIS................................................................................................................................................7
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2. EXECUTIVE SUMMARY........................................................................................................................................8
3. OBJECTIVE OF THE STUDY..................................................................................................................................8
4. ABOUT THE SECTOR..............................................................................................................................................9
HISTORY OF THE SECTOR -..................................................................................................................................9
CURRENT INDUSTRY OVERLOOK -...................................................................................................................9
Type of Firms -.........................................................................................................................................................10
HISTORICAL GROWTH OF THE SECTOR –......................................................................................................11
5. ABOUT THE COMPANY & DEPARTMENT.......................................................................................................12
About the company -.................................................................................................................................................12
Product Portfolio & Services –.................................................................................................................................12
6. INTERNSHIP SUMMARY.....................................................................................................................................15
7. PROJECT DESCRIPTION.......................................................................................................................................16
1. INTRODUCTION...........................................................................................................................................16
2. RESEARCH METHODOLOGY....................................................................................................................17
3. EXECUTIVE SUMMERY –...........................................................................................................................19
4. MARKET OVERVIEW..................................................................................................................................19
2. INDUSTRY TRENDS................................................................................................................................22
2.5. CURRENT SCENARIO CONSIDERING THE IMPACT OF COVID-19 PANDEMIC –......................23
Before Covid-19 –.....................................................................................................................................................23
4. GENE THERAPY MARKET SEGMENTATION BY VECTOR.................................................................24
5. GENE THERAPY MARKET SEGMENTATION BY APPLICATION.......................................................27
5. GENE THERAPY MARKET SEGMENTATION BY TYPE.......................................................................29
6. GENE THERAPY MARKET SEGMENTATION BY DELIVERY METHOD...........................................30
7 COMPANY PROFILE..........................................................................................................................................31
1.1. Spark Therapeutics......................................................................................................................................31
1.1.1. COMPANY OVERVIEW..................................................................................................................31
1.1.3. RECENT DEVELOPMENTS............................................................................................................32
1.2. Alnylam Pharmaceuticals............................................................................................................................33
1.3. ArrowHead Pharmaceuticals.......................................................................................................................35
1.4. SQZ Biotechnologies..................................................................................................................................36
1.5. AveXis.........................................................................................................................................................38
1.6. Asklepios Bio-Pharmaceutical (AskBio)....................................................................................................39
1.7. Biogen (US).................................................................................................................................................40
1. COMPANY OVERVIEW –............................................................................................................................40
1.8 Novartis AG (Switzerland)..........................................................................................................................43
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1.9 Orchard Therapeutics plc. (UK)..................................................................................................................45

1.10 Bluerock Therapeutics.................................................................................................................................46
8. CASE STUDY – A SUCCESS STORY OF NOVERTIES WITH THE INNOVATIVE IDEA OF JOE
JIMENEZ, CEO OF NOVERTIES -........................................................................................................................48
9. CASE STUDY - Drug Launch: Zolgensma....................................................................................................50
8. LEARNING/ FINDING........................................................................................................................................52
10. REFERENCES.......................................................................................................................................................53
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1. PROJECT SYNOPSIS
A Study on Gene Therapy & Its future

Project Title
prospects in India
Company Name Chromatus Consulting
Student Name Priyanka Saha
Student Roll No 2019-2005-0001-0040
Project Guide Name (External Mentor) Mr. Vijay Natekar

Project Guide Designation (External
Operation Head
Mentor)
Project Guide Name (Internal Mentor) Prof. Bibhas B.
Project Guide Designation (Internal
Dean
Mentor)
Confidential Report (Yes/No) Yes
Duration of project 60 days
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2. EXECUTIVE SUMMARY
Businesses around the world have more and more to rely in consultancy to thrive through a more
complex and disruptive fast changing economic, social and technological fast changing world.
The consultancy industry is an increasing part of the business life and special as the international
economy gets increasingly more volatile and software driven. In these scenario and as it stands
the global consulting industry revenues – HR, IT, strategy, operations, management and business
advisory services – will be about $470 billion in 2016, according to the company Plunkett
Research estimative.
The numbers presented represent a reasonable growth from $449 billion during the previous
year. In the U.S., consulting of all types, including management, scientific and technical,
generated $257.6 billion during 2015, up from $233.9 billion the previous year.
3. OBJECTIVE OF THE STUDY

The basic objectives of the project are –
1. To get the overview of the industry
2. To understand the current market growth of the industry
3. To understand the covid-19 impact on the industry
4. To understand the new trends in that particular industry
5. To understand the gene therapy Market
6. To understand the current scenario of Gene therapy in India
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7. To understand the future prospects of gene therapy in India considering the covid-19
situation.
4. ABOUT THE SECTOR
SECTOR – CONSULTING
HISTORY OF THE SECTOR -

Management Consulting began to develop shortly after the rise of management as a unique field of
enterprise in the late 19th Century, along with the Industrial Revolution. The early firms were started by
university professors. The first management consulting firm was named Arthur D. Little, after the
founding MIT professor, in the late 1890s. This firm originally specialized in technical research, but later
became a general Management Consultancy. Booz Allen Hamilton was founded as a Management
Consultancy by Edwin G. Booz, a graduate of the Kellogg School of Management at Northwestern
University, in 1914, and was the first to serve both industry and government clients. This firm later
changed its name to Booz & Co.
The first pure Management Consulting company was McKinsey & Company. McKinsey was founded in
Chicago during 1926 by James O. McKinsey (known as Mac to most people), an accounting professor
from the University of Chicago. Marvin Bower, hired in the late 1930s as a partner, ran the company for
30 years and crafted the firm into what it is today. He believed that Management Consultancies should
adhere to the same high professional standards as lawyers and doctors. Thus McKinsey, under Brower, is
generally credited with developing into the first Management Consulting firm in the modern sense.
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CURRENT INDUSTRY OVERLOOK -
Since 2004, the Consulting industry has recovered substantially—all firms are now aggressively
recruiting again. Most firms were surprisingly resilient to the Great Recession of 2008 and 2009.
Currently, most consulting offices are working at full capacity and the outlook for the sector as a whole is
very positive. Top Consulting firms continue to compete with Investment Banks and each other for the
top candidates from universities and business school programs across the country, offering highly
attractive compensation packages and career opportunities. At many top business schools, as much as 1/3
of the graduating class will sign with Consulting firms upon graduation.
The current trend in the market is a clear segmentation of Management Consulting firms by function.
Major/Strategy-Focused firms such as Bain, BCG and McKinsey retain their dominant global brand in
strategy-oriented projects, with smaller, more specialized firms such as L.E.K. and Oliver Wyman
competing effectively in the high-end market for specific projects in which they have a competitive
advantage. Many other generalist Management Consultancies are broadening their offering include higher
volume, lower margin projects such as IT deployment and retail sales analysis. There has always been a
bit of an unclear dividing line between Management Consulting and other Consulting practices, such as
Information Technology Consulting and Human Resources Consulting, and this line continues to be
blurred.
Management Consulting also continues to branch out more and more into non-business related fields as
well—specifically, working with governments, quasi-government agencies, and not-for-profit
organizations. As the need for professional and specialized advice in these areas grows, these other
institutions are relying more and more on the same strategic and analytical principles that have helped
corporations for decades.
Type of Firms -
There is plenty of overlap across disciplines, and specific Management Consulting firms may practice
multiple disciplines. But broadly speaking, Management Consulting firms focus on engagements that can
be grouped into four different, important areas.
Major (Strategy-Focused): Consulting geared towards high-level, corporate decisions. This category

can include broad industry analysis, competitor assessment, merger integration, new product strategy, etc.
In other words, this category fits most large-scale, high-level Consulting engagements. Most MBA
Graduates are hired by these types of firms.
Information Technology (IT): Consulting that focuses on Information Technology development in

several different areas (Assessment, Design, Implementation, Infrastructure, and other areas).
Human Resources (HR): Consulting that focuses on maximizing value created from Human Resources,
or employees, in an organization. Can include topics such as Compensation schemes, Health & Benefits
planning, Recruitment processes, etc.
Niche: Consulting that tends to focus on a specific technology, research, or analysis method, or is

particular to a specific industry or region. For example, a Management Consulting firm that only serves
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the Bioengineering industry would be a type of Niche consulting; another example would be a firm that
has developed a proprietary profitability analysis technique and sells it to various firms in the Financial
Services Industry. Often, Niche Consulting firms were founded by ex-partners at Major/Strategy-Focused
firms that had developed a strong Consulting practice in a particular area during their tenure there.
Specialized firms have increased pressure on larger, more generalist Consulting firms to increase their
sophistication in industry and geography-specific knowledge in order to compete for business.
HISTORICAL GROWTH OF THE SECTOR –

1. The size of the global management consulting market has grown to US$160 bn in 2019 from
US$107bn in 2011 (source: statista).
2. Due to the coronavirus outbreak, the size of the consulting market is estimated to decrease to 132
billion U.S. dollars in 2020.
3. The number of people employed in management consulting in the United States has grown
annually over the past five years and in 2018 there were around 684,470 management consultants
employed in the U.S. Despite the increasing number of consultants, finding and keeping good
employees was one of the leading business challenges for management consulting firms. The
most pressing issue for companies was, by far, attracting and developing new businesses, with 79
percent of senior managers citing this a leading challenge.
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5. ABOUT THE COMPANY & DEPARTMENT

CHROMATUS CONSULTING
About the company -

Chromatus is one of the world’s top consulting firm with its top specialists helping clients to grow in long
term business goals. Chromatus, for past 10 years has been continuously observing, collecting and
analyzing data with latest market trends. It has provided us to have an ultimate edge over the competitors
in understanding and interpreting the business problems. We make a meaningful and actionable influence
on our clients’ businesses with the usage of our in-depth insights and detailed knowledge of the subject.
This has helped numerous clients in building strong brand value driving marketing efficiency.
Their innovative consulting and market research services with a proven framework of data collection sets
us apart from the rest. Chromatus has partnered number of key brands to understand the market, analyse
the competition and identify the target customers to maximize the profit in a better way. The in depth 360
degree research design has resulted in considering holistic views of all the stakeholders in the project.
Chromatus is a dynamic research entity who can partner or collaborate with a wide network of data
collection, advertising, research and branding experts around the world, bringing added value to our
clients. We are well equipped with the latest in-house data analysis tools, technologies and innovation to
design flexible solutions marketers need to grow businesses.15 years of Quantitative & Qualitative
Research Experience.
Product Portfolio & Services –
PRODUCTS DESCRIPTION
1 . P RIMA RY MA RKE T RESEA RCH

MARKET RESEARCH 2 . SECON DARY MA RKE T RESEA RC H
3 . B2 B MA RKE T RESEA RCH
CONSUMER Any marketer wants to have an understanding of the

INSIGHTS
consumers and the factors influencing a trier/purchase and
contributing to the repeat purchase. We help in achieving
the ultimate goal of any marketer to identify the consumers,
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to reach the end-users in a more innovative way and find a

way to influence their buying decisions.
It is important for a marketer to establish what they want to
achieve from the research:
Chromatus will help the customers in performing a
consumer survey as per the client’s objective.
The chromatus team is responsible for the management of
the branding who helps in building brands to look after
long term strategic goals.
Chromatus helps in achieving branding goals, their research
team considers the following:
In tapping the competition branding in terms of its
products, prices, strategies, key divisions, and subsidiaries.
To understand the awareness and loyalty of customers
BRAND
towards various brands.
MANAGEMENT
To track the target customers purchasing patterns, brand
influence, media strategy on a periodical basis.
To gain insights on the relationship of one part of the
business to another and the scope for cross-selling between
them.
To understand the current methods of managing the brands
across companies and garner any advantages which could
be gained from separating them or bringing them together.
The operational assistance by chromatus – CSR majorly
revolves around the fact that the firms can outsource the
operations to us in order to focus on their core business
activities. We will help you in collecting and analyzing
data in order to create a sustainable model for the
CSR CONSULTANT
beneficiaries in order to create wealth for companies and
for society. The data-driven decisions by chromatus in
project implementation will garner to anticipate and
address issues associated with their relations with other
stakeholders to succeed in their business endeavors.
CUSTOMER They at chromatus will assist the customer in assessing the
SATISFACTION consumer satisfaction levels and help the customer in
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building appropriate strategies. Chromatus conducts

consumer satisfaction surveys (csat) with its proven
methodologies that will surely bring actionable results.
They analyse the survey results with chromatus internal
SURVEY analytical tools and statistical techniques.

How do they measure customer satisfaction?
Promoters – they are loyal customers and they strongly
recommend the brand
Neutrals – they are having neutral opinion towards their
brand
Detractors – these are the unsatisfied chunk of customers
Chromatus helps in tracking a study which mostly on a
longitudinal basis, and helps in examining the changing
views of the same people over a period of time. On the
other hand, a cross-sectional tracker study helps in
understanding the consumers’ views at a single point in
time. While marketer has the option to choose the study
TRACKER STUDY pattern which can perform a series of data-points to
uncover the key insights from different groups of people. In
other words, a tracking study by Chromatus has given their
client strong confidence in the shifting in consumer
sentiment reflecting actual changes, since we have over the
period of time are asking the same respondent the same
questions at different times.
6. INTERNSHIP SUMMARY
Due to Covid-19 the internship was work from home based and totally on secondary research. Firstly we
got the training on secondary research data gathering, brand management, storytelling, SPSS. During
these training we were assigned different tasks also. Those are -
1. During this internship period I was basically assigned for creating Press Released table of content
(PR ToC), which is nothing but the strategy of brand awareness. For making awareness of their
brand they create PR ToC on a particular topic and after that post it on their websites. After this if
any customer show the interest and ask for sample report then they provide them 30-40 pages
sample research paper, through this the client get the idea about the research paper that exactly
what point the company is going to cover for that particular topic.
Here I have created 30 PR ToC for the company.
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2. The second work was to company profile listing, for this we had to post the company information
on different platforms.
3. The third work was to create infographics. I created infographics for the company using Canva.
4. The other work was live project. I have worked on 7 live projects during this internship. The
duration of those life projects was 1-2 days. In this live project I had to gather the secondary data
based on the particular topic and analysis the company profiles (key player on that market).
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7. PROJECT DESCRIPTION
1. INTRODUCTION
1.1. OBJECTIVES OF THE STUDY
1. To understand the gene therapy Market
2. To understand the current scenario of Gene therapy in India
3. To understand the future prospects of gene therapy in India considering the covid-19 situation.
1.2. DEFINITION OF GENE THERAPY MARKET
1.2.1. WHAT IS GENE THERAPY
Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the
future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s
cells instead of using drugs or surgery. Researchers are testing several approaches to gene
therapy, including:
• Replacing a mutated gene that causes disease with a healthy copy of the gene.
• Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
• Introducing a new gene into the body to help fight a disease.
Although gene therapy is a promising treatment option for a number of diseases (including
inherited disorders, some types of cancer, and certain viral infections), the technique remains
risky and is still under study to make sure that it will be safe and effective. Gene therapy is
currently being tested only for diseases that have no other cures.
1.2.2. HISTORICAL DEVELOPMENT
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1.3. YEARC CONSIDERED –

We have considered 2015 – 2025 as a scope of the study. 2015 to 2018 are considered as a
historical year for analysis. 2019 is considered as a base year. The CAGR is calculated from
2020 – 2025.
1.4. CURRENCY –
n this report we have considered US Dollar as the base currency while calculating the market
size and understanding the competitive landscape. Any other currency is converted to US Dollar
with the base year as 2019. Conversion/ Exchange rate are considered according to the data
provided by IMF (International Monetary Fund). Further we have assessed other conversion rate
website such as OANDA, X-rates, XE Currency converter for data validation. All the forecasts
are done with the revenue and volume calculated under the standard assumption that is globally
accepted currency - the U.S. Dollar's value remains constant over the next five years.
1.5. LIMITATION OF THE STUDY –
• The research is based on the secondary research techniques.
• Secondary data is collected from the official government websites of the addressable
market. Further we have analyzed the company websites of the key market players, material
available in form of annual reports, trade data, news, and expert’s blogs. But the limitations of
secondary data cannot be neglected in this case, the conclusions and findings are drawn on the
basis of analysis of this data only so, any problems with these data can lead to huge changes in
the above said.
1.6. STAKEHOLDERS –
o Gene Therapy manufacturers
o Gene Therapy dealers, and suppliers
o Different vector manufacturing companies
o Raw material and component suppliers
o Government and research organizations
o Environmental and industrial associations
o Banks, venture capitalists, financial institutions, and other investors
o Market research and consulting firms
o Raw Material Distributors, Wholesalers
o Support service providers
o Technology Standards Organizations, Forums, Alliances and Associations
2. RESEARCH METHODOLOGY
2.1. DATA ANALYSIS –
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Data analysis is the method or methods that can be used to analyze data and the process of
analyzing it. There are many different forms of data, but people usually think of quantitative data
first. These are data such as census data or survey data. This projects is based on secondary data.
2.2. DATA VALIDATION –
Validation means using more than one method to collect data on the same topic. This is a way of
assuring the validity of research through the use of a variety of methods to collect data on the
same topic, which involves different types of samples as well as methods of data collection.
However, the purpose of triangulation is not necessarily to cross-validate data but rather to
capture different dimensions of the same phenomenon.
2.3. RESEARCH PROCESS
2.3.1. SECONDARY RESEARCH -
1. Sources for conducting secondary research:
Government or semi-government publications – These institutes collect data on a regular basis in
a variety of areas and publish it for use by members of the public and interest groups. Some
common examples are the census, vital statistics registration, labor force surveys, health reports,
economic forecasts and demographic information.
2. Regulatory Bodies –
o Annual Report – Data is collected from the annual reports or 10k Sec filings of Tier 1 and
Tier 2 companies.
o Market Research Repository – For some topics, an enormous number of research studies
that have already been done by others can provide you with the required information.
o Mass media – Reports published in newspapers, in magazines, on the Internet, and so on,
may be another good source of data
2.4. MARKET SIZE ESTIMATION
2.4.1. BOTTOM UP APPROACH –
A bottom-up approach is the piecing together of systems to give rise to more complex systems,
thus making the original systems the sub-systems of the emergent system. Bottom-up processing
is a type of information processing based on incoming data from the environment to form a
perception. In a bottom-up approach the individual base element of the system is first specified
in great detail.
2.4.2. TOP-DOWN APPROACH –
It is also known as step wise design. It is essentially the breaking down of a system to gain
insight into its computational sub-systems in a reverse engineering fashion. Top down approach
starts with the big picture. It breaks down from there into smaller segments
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2.5. RESEARCH ASSUMPTION –

The global economy has a direct impact on the Gene Therapy Market. Regarding the global
economy landscape, no recessions, downturns, economic collapses of any sort are considered
while forecasting any market in this research study. For niche market segments where accurate
data of the respective time line was not available, the data was calculated using trend line
analysis. In some instances, where mathematical and statistical models could not be applied to
arrive at the number, generalization of specific related trends to that particular –
o The market will be growing / increasing in developing and developed countries around
the globe.
o The demand and supply will be at par.
o The government policies will play an important role in the growth of the industry.
o The unstable relationship between different countries around the world, which may result
into war, may affect the industry
3. EXECUTIVE SUMMERY –
In 2019, the cell therapy segment accounted for a market share of over 53% in the global cell and
gene therapy market. The segment is expected to grow at a steady rate during the forecast period
due to the increase in the target population and the rise in the number of countries preferring cell
therapies in their patients. Increased therapeutic benefits are attracting several countries to invest
in this technology and conduct a high number of clinical trials.
In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene
therapy market. Oncology has been one of the targets of intense research for the gene therapy
procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting
cancer. The segment is expected to grow at a promising rate on account of the high prevalence of
cancer diseases, especially in low and middle-come countries. The market is growing at a
double-digit CAGR, which is expected to help the segment as many cell and gene therapy for
cancer are commercially available.
Source: https://finance.yahoo.com/
4. MARKET OVERVIEW
1.1. DRIVES
o HIGH INCIDENCE OF CANCER AND OTHER TARGET DISEASES –
The weight of chronic diseases has just been expanding since recent years. The cases for cancer,
for example, are developing quickly. As indicated by the American Cancer Society, from 2016–
2019, the quantity of new instances of cancer in the U.S. expanded from 1,685,210 to 1,762,450,
and the cancer mortality rose from 595,690 to 606,880 during a similar timeframe. Without a
doubt, various progressions have been made in the medical industry for building up a legitimate
solution for cancer and other rare chronic diseases.
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The progressions in regards to gene therapy however can significantly aid in getting closer to
dealing properly with several chronic diseases. Gene therapy is introducing genetic material into
cells for compensating for abnormal genes or for making a beneficial protein. In case a mutated
gene causes an essential protein to be faulty or non-existent, gene therapy may potentially be
able to introducing a new copy of the gene for restoring the function of the protein. Ascribed to
these reasons, the quality treatment showcase generated revenue of $3,407.5 million out of 2019
and is relied upon to progress at a 31.1% CAGR during 2020–2030.
o THE APPROVAL OF EARLY CAR-T (chimeric antigen receptor - T cell) BASED
GENE THERAPY PRODUCTS –
The endorsement of early CAR-T-based quality treatment items has made rewarding roads in
products endorsements over extended signs. Clinical preliminaries for T-cell treatments every
year has risen exponentially in the ongoing past. Steady improvements in CAR-T cell treatment
are perceived as one of the key driving components for showcase development.
The U.S. Food and Drug Administration gave a notable activity today making the First gene
therapy treatment accessible in the United States, introducing another way to deal with the
treatment of Cancer and other serious and life-threatening diseases.
The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and youthful grown-up
patients with a type of acute lymphoblastic leukemia (ALL).
Kymriah is a genetically-modified autologous T-cell immunotherapy. Each dose of Kymriah is a
customized treatment made utilizing an individual patient's own T-cells, a kind of white blood
cell known as a lymphocyte. After collecting the patient's T-cells it is sent to an manufacturing
center where they are genetically modified to include a new gene that contains a particular
protein (a chimeric antigen receptor or CAR) that guides the T-cells to target and kill leukemia
cells that have a particular antigen (CD19). When the cells are modified, they are infused back
into the patient to kill the cancer cell.
Advanced therapy medicinal products (ATMPs) or cell and gene therapy products (CGTPs), as
they are brought in the US, bear game-changing potential for the treatment of serious conditions
for which we have no suitable treatments today. A few ATMPs have been authorized throughout
the most recent decade in the European Union (EU) just as in the United States (US), and the
quantity of ATMPs arriving at the market is relied upon to develop throughout the following
decad.
Adoptive T-cell transfer (ACT), a subclass of ATMPs, is another part of transfusion medication.
Antitumor, antiviral, or anti-inflammatory affects are mediated by infusion of lymphocytes. It
has been a quick advancement from a promising type of immuno-oncology in preclinical models
to the ongoing promoting endorsements of chimeric antigen receptor (CAR)- T cells for
treatment of leukemia and lymphoma
CAR Ts have indicated immense reduction rates in clinical preliminaries, up to 94%, in severe
types of pediatric and young adult refractory hematologic B-cell malignancies. This is
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remarkable, as most CAR-T clinical trials recruit cancer patients that have not responded to
various other available treatments
1.2. RESTRAINTS
o HIGH COST ASSOCIATED WITH THE TREATMENT
Unlike other drugs regulated by the Food and Drug Administration, gene therapies are subject
not exclusively to the administrative structure of the FDA, yet in addition to the Office of
Biotechnology Activities, and the Recombinant DNA Advisory Committee. Exorbitant
administrative oversight makes a lengthened and costly course to endorsement.
By one estimate, an approved gene therapy drug costs nearly $5 billion (five times as high as the
average cost of FDA approval). Covering these costs is difficult because many of the diseases
gene therapy drugs treat are extremely rare.
o SIDE EFFECTS LIKE UNWANTED IMMUNE RESPONSES
The immune systems reaction to antigen depends on (i) the relative frequencies of responding T
and B cells and on the thresholds of binding affinity that their receptors display, (ii) the levels of
antigen present, and (iii) the period during which the antigen remains in secondary lymphoid
tissue, where primary immune responses are initiated.
In the peripheral immune system the key mechanisms that induce and maintain tolerance include
clonal deletion, anergy (absence of the normal immune response to a particular antigen or
allergen), ignorance, and suppression. Ignorance describes the situation whereby T cells fail to
respond to a specific antigen. This can be due to (i) low levels of antigen that are insufficient to
activate T cells, (ii) antigens that are physically separated from T cells (such as blood-brain
barrier). Antigens that are presented in the absence of co-stimulation signaling can induce
anergy, characterized by state of T-cell unresponsiveness. Deletion of T cells can occur when the
cell is activated in the absence of co-stimulation, or due to a lack of growth factors. Tolerance
induction by suppression is an active process by which a regulatory subset of T cells (Tregs, Tr1,
etc.) specifically suppresses the activity of T cells.
1.3. OPPORTUNITIES
o HIGH GROWTH
Global Gene Therapy Market is expected to register a CAGR of 40.7% during the forecast period
of 2019 to 2025 and was valued at USD 524 million in 2018. More than 150 investigational new
drugs applications were filed for gene therapy in 2018 alone. With this in mind, it is expected
that this market to grow significantly, with ten to 20 cell and gene therapy approvals per year
over the next five years.
1.4. CHALLENGES
The biggest challenge lies in the areas of process development, manufacturing and analytical
technologies. As was the case for biologic drugs 30 years ago, multiple methods for the
production of different viral vectors and cell therapies exist.
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o COMPLEX MARKET ACCESS DYNAMICS

Price points for recently launched gene therapies have fallen short of expectations. For example,
prior to the launch of Kymriah, industry participants projected a price of $600,000 to $750,000
per patient. However, the actual price of Kymriah at launch was $475,000 per acute
lymphoblastic leukemia patient. Spark’s Luxturna, which was able to achieve a relatively high
price of $850,000 for the treatment of both eyes, fell short of the $1 million+ price point
expected by the market. Payers are hesitant to pay high up-front costs for these therapies. This is
partly due to the fact that curative therapies do not yet have a long-term track record of sustained
efficacy.
o CHALLENGING GENE THERAPY FRANCHISE SUSTAINABILITY
The “bolus-like” revenue curve associated with a first gene therapy presents a challenge to
achieving sustainable growth given the lack of a stable base from which to build. Revenue will
wane naturally as the addressable population is depleted a few years after launch. The short
duration of meaningful revenue contributions from a single gene therapy product suggests the
timing of life-cycle management efforts and other product launches is critical. Achieving growth
would require launching another revenue stream (e.g., another gene therapy product) before the
revenue of the first gene therapy starts to wane. This would lead to a situation in which the
revenue peaks overlap, potentially resulting in substantial growth. However, maintaining growth
would require launching a product every few years. Beyond the question of whether a biotech
company would have the portfolio breadth or resources to launch several products within a few
years of each other, it would be challenging to optimally time the launches of subsequent gene
therapies.
2. INDUSTRY TRENDS
2.1. PORTER’S FIVE FORCES MODEL
2.1.1. THREAT OF NEW ENTRANT – LOW
The threats of new entrants is low for the Pharmaceutical industry. There are extensive costs
associated with establishing a manufacturing set up, research & development, marketing sales &
distribution. There is also a high risk of no return on investment if a company fails to produce the
required new drug on time, causing losses in million. The existing players have developed
economies of scale, leading to increase profits. Another reason is the lengthy process of approval
from regulatory authorities. Established brands and product differentiation are also a cause of the
low entry threat of new entrants. A new entrant will also have a tough time getting access to
distribution channels. Most buyers go for drug brands they have already tried. So, a new entrant
will have a tough time grabbing market share in this industry. Another reason is that most drugs
are patented so new entrant will have to start from scratch. Thus, the threat of new entrants is
low.
2.1.2. THREAT OF SUBSTITUTE – MODERATE/LOW
If the drug is still in its patent period, there is no substitute for that drug. However, once the
patent period expires, its generic productions begins and a number of substitute develop. The
22
second threat is alternative medicines and treatment to these drugs. This is more common in the
Easter nations. These include yoga, meditation, & various other therapies. Also, homeopathic,
and herbal treatments are also the other substitute for drugs made by pharmaceutical industries.
Promotion of a healthier lifestyle such as balanced diet, exercise, and other physical activities are
the substitute to many drugs. Thus, the threat of substitute is from low to moderate.
2.1.3. BARGAINING POWER OF BUYER – MEDIUM
Patents of new drugs last for around 20 years, allowing the manufacturer to dictate the prices for
this time period. After which, generic production begins and prices become more competitive.
Similarly, large customers such as hospitals do possess a certain bargaining power but the
individual customers have very little to none. Another factor is the brand name. That further
reduce the bargaining power of the buyers. Buyers today have access to the internet which allows
them to further do research on drugs in addition to the prescription of their doctors, giving them
more bargaining power. Thus, overall the bargaining power of the buyers or customers is
moderate.
2.1.4. BARGAINING POWER OF SUPPLIER – LOW
The pharmaceutical industry only requires the raw materials for the drugs as the drugs are
manufactured in house. The second requirement is technology for the manufacturing &
production plane. The third element the suppliers provide is the packaging material. All of these
are supplies that a number of suppliers are willing to provide. Thus, they are in no position to
bargain or attempt to influence the market price. Some of the suppliers include BASF
Corporation, Nexeo Solutions, 7 Wako Chemicals. Thus, the suppliers in the pharmaceutical
industry have very low bargaining power.
2.1.5. INTENSITY OF COMPETITIVE RIVALRY – HIGH
The pharmaceutical industry is one of the most competitive industries in the world. Most of the
players in the industry have been here for a long time and are well recognize globally. The profit
margins are high, there are a large number of small and large sized player, and strict government
regulations make it a very competitive industry. There is no room for making errors. Another
reason is the recent trend of mergers and acquisitions where large industries have absorbed the
small players. Technological advancement in biotech and generics have further increased the
competition as the major players are Saofi, AstraZenca, Pfize, Merck & GlaxoSmithKline. Thus,
the overall competitive rivalry in the Pharmaceutical industry is high.
2.5. CURRENT SCENARIO CONSIDERING THE IMPACT OF COVID-19
PANDEMIC –
Before Covid-19 –
Gene therapy market is characterized by close to 500 drug candidates that are currently under
development. According to a market report released last year, the gene therapy market was
estimated to be over USD 10 billion by 2030. 30% of clinical stage therapy candidates are being
designed for the treatment of oncological disorders; AAVs are the preferred vector types. $16+
23
bn has been invested by both private & public investors, till 2019; there have been 28IPOs, from
which companies claim to have raised over $2 billion.
Due to Covid-19 –
Some key players, such as bluebird bio have certainly been impacted by COVID-19. While some
other players, such as Pfizer have announced little impact on their gene therapy development
plans. A number of gene therapy developers are also re-shifting their focus to develop novel
vaccines for COVID-19. The revised short-term focus across these established developers is
likely to contribute significantly to the impact the gene therapy market.
Overall, the gene therapy market is likely to witnesses a short-term decline in the overall market
growth rate. The ability of the developers to adapt to novel trial designs, manage the supply
chain, and navigate regulatory environment is likely to play a critical role in minimizing the
impact of COVID-19.
4. GENE THERAPY MARKET SEGMENTATION BY VECTOR
3.1. VIRAL VECTOR

3.1.1. RETROVIRUSES
Retroviral vector-mediated gene transfer has been key to the advancement of gene therapy.
Retroviruses have a few particular points of interest over different vectors, particularly when
permanent gene transfer is the preferred outcome. The most important advantage that retroviral
vectors offer is their ability to transform their single stranded RNA genome into a double
stranded DNA molecule that stably integrates into the target cell genome. This implies retroviral
vectors can be utilized to permanently modify the host cell nuclear genome.
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3.1.2. LENTIVIRUSES
Insertional mutagenesis has been a significant disadvantage for the utilization of γ-retroviral
vectors in human gene therapy. Lentivectors additionally display insertional mutagenesis in
animal models and cell based frameworks inclined to oncogenesis, although overall they seem to
be less mutagenic than their oncoretrovirus partners (Hematti et al., 2004; Montini et al., 2009).
Actually, in the first approved human clinical trial using lentivectors for the treatment of HIV, no
abnormal cell expansion or enrichment of integration sites near proto-oncogenes have been
detected so far.
3.1.3. ADENOVIRUSES
Greater than 400 gene therapy trials had been or are being performed with human ad vectors [2].
Most of those trials are for remedy of cancer, even though some are to be used of ad vectors as
vaccines wherein the vector expresses a foreign antigenic protein or for gene remedy in which
the vector expresses a non-mutant protein to correct a genetic defect. Also, thousands and
thousands of military recruits has been and are being immunized effectively against acute
respiratory disease by way of gastrointestinal management of live ad serotype four (ad4) and ad7
vaccines inside the form of enteric covered drugs [6]. Ad vectors have many benefits: ads are
nicely studied and ad vectors may be grown into high titer stable stocks, they infect non-dividing
and dividing cells of different kinds, and they are maintained in cells as an episome.
3.1.4. ADENO ASSOCIATED VIRUS
Adeno-associated virus (AAV) vectors are currently among the most frequently used viral
vectors for gene therapy. At recent meetings of the American Society for Gene Therapy, nearly
half of the presentations involved the use of AAV. There are several concerns for any viral
vector. These include the capacity to connect to and enter the target cell, successful transfer to
the nucleus, the ability to be expressed within the nucleus for a sustained period of time, and a
general lack of toxicity. AAV vectors have been highly successful in pleasant all of those
standards. Furthermore, a variety of changes have served to enhance their application. Numerous
issues have guided the improvement of current AAV vectors, particularly the shortage of
pathogenicity of the wild-type virus and its persistence.
3.1.5. HERPES SIMPLEX VIRUS
Herpes simplex virus type 1 (hsv-1) is a common human virus, best referred to as the causative
agent of recurrent labial herpes (bloodless sores) [1]. The specific residences of hsv-1 have more
and more been exploited in vector layout, particularly for therapy of the central nervous system
diseases.
Herpes simplex virus (HSV) has a number of advantages as a vector for delivering precise genes
to the apprehensive device. These include its large size, wide host range, and its capability to
establish long-lived asymptomatic infections in neuronal cells wherein a selected location of the
viral genome continues to be expressed. Unluckily, the big size of this virus and issue in
manipulating it has led to its use as a vector lagging at the back of that of other, smaller viruses
along with the retroviruses. In addition, the virus's ability to replicate lytically inside the brain,
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under some circumstances, causing encephalitis, has led to fears about its potential safety for
ultimate use in humans.
3.1.6. POXVIRUS
Remedies with poxvirus vectors can have long-lasting immunological effect within the host, and
thus they were considerably studied to treat diseases and for vaccine improvement. More
importantly, the oncolytic properties of poxviruses have brought about their improvement as
most cancers therapeutics. Poxviruses, vaccinia virus (vacv) and myxoma virus (myxv), were
notably studied as virotherapeutics with promising consequences. The poxviruses (family
poxviridae) are large, double-stranded dna (dsdna) viruses which have had an oversized effect
upon human fitness and history, each positive and negative. Smallpox has been an epidemic of
humanity from prehistory until the close of the 20 th century, a tremendously contagious disease
that killed among one-fourth and one-third of its victims and automatically left survivors with
lifelong scarring
3.1.7. VACCINIA VIRUS
Vaccinia virus was widely used in the World Health Organization's smallpox eradication
campaign and is currently a promising vector for gene remedy as a result of its unique
characteristics. Vaccinia virus can selectively replicate and propagate productively in tumor
cells, ensuing in oncolysis. In addition, rapid viral particle production, huge host range, large
genome size (approximately 200 kb), and safe handling render vaccinia virus a suitable vector
for gene therapy
3.2. NON-VIRAL VECTORS
Non-viral vectors avert the primary concerns that come with the use of viruses: safety,
immunogenicity and manufacturing limits (yield, scaling-up and costs). Several non-viral
techniques were developed and are well worth bringing up: naked nucleic acid, physical-
mediated strategies (gene gun, electroporation, hydrodynamic) and chemical-based
nanoparticles. Even as these techniques are all utilized in gene delivery research accomplished in
small animal models (e. G., mouse), maximum of them are typically much less applicable for
systemic gene delivery in human beings, besides for naked nucleic acid and chemical-based
nanoparticles.
3.2.1. LIPOFECTION
Lipofection is a lipid-based transfection technology which belongs to biochemical methods
including also polymers, DEAE dextran and calcium phosphate.
Lipofection, also known as “lipid transfection” or “liposome-based transfection,” uses a lipid
complex to deliver DNA to cells. Lipids are a broad class of fat-soluble biomolecules, such as
fats, oils, and waxes. The cell membranes of animal cells are composed of a bilayer of
phospholipids with hydrophilic surfaces facing the cytoplasm and extracellular environment.
Lipofection technology uses tiny vesicular structures called liposomes that have the same
composition as the cell membrane. A scientist performs a simple reaction that forms a liposome
26
around the DNA sequence to be transfected. Depending on the liposome and cell type, the
liposome can be endocytosed or directly fuse with the cell membrane to release the DNA
construct into cells.
3.2.2. NAKED/PLASMID VECTORS
Increases in performance have made naked DNA gene transfer a feasible technique for gene
remedy. Intravascular delivery outcomes in effective gene transport to liver and muscle, and
provides in vivo transfection methods for basic and applied out gene therapy and antisense
techniques with oligonucleotides and small interfering RNA (siRNA). Delivery via the tail vein
in rodents provides an specially easy and powerful means for in vivo gene transfer.
Electroporation strategies drastically enhance direct injection of naked DNA for genetic
immunization. The availability of plasmid DNA expression vectors that allow sustained
excessive level expression, lets in for the development of gene therapies based on the delivery of
naked plasmid DNA.
3.2.3. OLIGONUCLEOTIDES
Antisense oligonucleotides (ASOs) have been first observed to influence RNA processing and
modulate protein expression over two decades ago; but, development translating those agents
into the clinic institution has been hampered by means of inadequate target engagement,
insufficient biological activity, and off-target toxic effects. Over the years, novel chemical
modifications of ASOs had been employed to address those issues. Those modifications, in
mixture with elucidation of the mechanism of action of ASOs and improved clinic trial design,
have furnished momentum for the translation of ASO-based strategies into therapies. Many
neurological conditions lack an powerful treatment; but, as research regularly disentangles the
pathogenic mechanisms of those diseases, they provide a really perfect platform to test ASO-
based strategies.
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5. GENE THERAPY MARKET SEGMENTATION BY APPLICATION
4.1. NEUROLOGICAL DISEASES

The nervous system is a complex and difficult organ system to study, and the brain is an organ in
which many of the most pervasive disease processes arise, for which the reason remains elusive.
These diseases encompass a broad spectrum of pathological states and can have global or local
effects on metabolism, and neural improvement and function. Drugs and neurosurgical
techniques have usually now not confirmed powerful in the treatment of those disorders owing to
the complexity and restricted information of the pathophysiology involved. Further, the blood–
brain barrier (BBB) limits the usage of systemic remedies as it impedes substantial delivery of
therapeutic agents to the CNS.
Adeno-associate viral (AAV) vectors are a rapidly emerging gene therapy platform for the
remedy of neurological diseases. In preclinical research, transgenes encoding therapeutic
proteins, microRNAs, antibodies or gene-editing machinery have been successfully delivered to
the relevant worried system with natural or engineered viral capsids through diverse routes of
administration.
4.2. HEPATOLOGICAL DISEASES -
Hematologic diseases are disorders which mainly affect the blood & blood-forming organs.
Hematologic diseases include rare genetic disorders such as anemia, HIV, sickle cell disease &
complications from chemotherapy or transfusions.
4.3. CARDIOLOGICAL DISEASES –
Cardiovascular disease (CVD) is basically a class of diseases that involve the heart or blood
vessels. CVD is mainly includes coronary artery diseases (CAD) such as angina and myocardial
28
infarction (commonly known as a heart attack). Other CVDs include stroke, heart failure,
hypertensive heart disease, rheumatic heart disease, cardiomyopathy, abnormal heart rhythms,
congenital heart disease, valvular heart disease, carditis, aortic aneurysms, peripheral artery
disease, thromboembolic disease, and venous thrombosis.
Cardiovascular diseases (CVDs) have now become the leading cause of mortality in India. A
quarter of all mortality is attributable to CVD. Ischemic heart disease and stroke are the
predominant causes and are responsible for >80% of CVD deaths. The Global Burden of Disease
study estimate of age-standardized CVD death rate of 272 per 100 000 population in India is
higher than the global average of 235 per 100 000 population. Thus there is a high chance of the
Gene Therapy market growth in this segment.
4.4. ONCHOLOGICAL DISEASES –

Oncology diseases, commonly known as cancer, typically start in one part of the body and then
spread to other areas in a process called metastasis. In all types of cancer, some of the body’s
cells begin to divide without stopping and spread into surrounding tissues. Cancer can start
almost anywhere in the human body, which is made up of trillions of cells. Normally, human
cells grow and divide to form new cells as the body needs them.
Estimated number of people living with the disease: around 2.25 million, every year, new cancer
patients registered: Over 11,57,294 lakh, Cancer-related deaths: 7,84,821. Total deaths due to
cancer in 2018 are 7,84,821, Men: 4,13,519 & women: 3,71,302 (Source:
http://cancerindia.org.in/)
A number of clinical trials conducted so far have shown promising results for the treatment of a
wide variety of cancers such as lung cancer, pancreatic cancer, prostate cancer, and malignant
melanoma. The FDA has approved gene therapies for the treatment of patients with certain types
of cancers. Current clinical trials include Rexin-G gene for pancreatic cancer, p53 gene for head
and neck cancer, the MDA-7 gene for melanoma, and TNF-α for pancreatic cancer. The Rexin-G
gene (for pancreatic cancer) is a cytocidal cyclin C1 construct that gets accumulated
preferentially in the cancer cells and blocks the action of cyclin G1 thus causing cell death. The
trial for this gene therapy is in Phase III.
4.5. OTHER DISEASES –

Gene therapy has a growing research potential particularly in the field of ophthalmic and retinal
diseases owing to three main characteristics of the eye; accessibility in terms of injections and
surgical interventions, its immune-privileged status facilitating the accommodation to the
antigenicity of a viral vector, and tight blood-ocular barriers which save other organs from
unwanted contamination.
5. GENE THERAPY MARKET SEGMENTATION BY TYPE
5.1. SOMATIC CELL GENE THERAPY –
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Gene therapy involves transferring good genes into cells to replace harmful genes. There are two
specific types: genes transferred into germ line cells (reproductive cells) and genes transferred
into somatic cells (body cells). In somatic gene therapy, altered genes are inserted into the
affected part of the body, or body cells are removed, treated with altered genes, and replaced.
People using this kind of therapy include patients with diseases such as cancer, hemophilia,
cystic fibrosis, and muscular dystrophy. Positive aspects patients may expect include health
benefits, safety, and less ethical concerns than germ line gene therapy, but negative aspects may
include lifetime treatments and problems with gene delivery.
5.2. GERMILINE GENE THERAPY –
Germline Gene therapy refers to the introduction or alteration of genes in germ cells. Gene
therapy is currently an upcoming field of treatment as it targets the cause of the disease. By
altering the genes responsible for a particular disease condition, it is possible to eliminate the
disease at an early stage.
6. GENE THERAPY MARKET SEGMENTATION BY DELIVERY METHOD
6.1. IN VIVO –
In vivo gene therapy is a technique which involves direct delivery of genes into the cells of a
particular tissue inside the patient’s body to treat genetic diseases. It can be applied to many
tissues of the human body including liver, muscle, skin, lung, spleen, brain, blood cells, etc. The
therapeutic genes are introduced by the viral or nonviral-based vectors into the patient. However,
30
the success depends on several factors such as efficient uptake of the therapeutic gene carrying
vectors by the target cells, intracellular degradation of the genes within the target cells and gene
uptake by the nucleus, expression ability of the gene, etc.
6.2. EX VIVO -
Ex vivo gene therapy is a type of gene therapy which involves exterior modification of a
patient’s cell and reintroduction of it to the patient. The cells are cultured in the labs (outside the
patient’s body), and genes are inserted. Then the stable transformants are selected and
reintroduced into the patient to treat the disease. Ex vivo gene therapy can be applied only to
certain cell types or selected tissues. Bone marrow cells are the cells frequently used for ex vivo
gene therapy.
7 COMPANY PROFILE
1.1. Spark Therapeutics
1.1.1. COMPANY OVERVIEW
INDUSTRY - Biotechnology Pharmaceutical
FOUNDED - 2013
HEADQUARTERS – Philadelphia, Pennsylvania
NO. OF EMPLOYEES – 368 (2019)
31
At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing

and delivering gene therapies, they challenge the inevitability of genetic diseases, including blindness,
hemophilia, lysosomal storage disorders and neurodegenerative diseases.
Founded in March 2013 as a result of the technology and know-how accumulated over two decades at
Children’s Hospital of Philadelphia (CHOP), our investigational therapies have the potential to provide
long-lasting effects, dramatically and positively changing the lives of patients with conditions where no,
or only palliative, therapies exist. Greater understanding of the human genome and genetic abnormalities
have allowed our scientists to tailor investigational therapies to patients suffering from very specific
genetic diseases. This approach holds great promise in developing effective treatments to a host of
inherited diseases. Their initial focus is on treating orphan diseases.
Mission –
Challenge the inevitability of genetic disease by discovering, developing and delivering treatments in
ways unimaginable – until now.
Vision –
A world where no life is limited by genetic disease.
1.1.2. PRODUCT PORTFOLIO

1. Gene therapy Research (using adeno-associated viral vector)
2. disease areas of focus –
 Mainly in Genetic diseases
 Neurodegenerative Diseases
 Others -
Learn more Learn More Learn More
3. Scientific platform & programs –
Cutting-edge vector State-of-the-art, in-house expertise in vector Strong commitment to

design expertise in vector manufacturing improve patient care
manufacturing
32
1.1.3. RECENT DEVELOPMENTS

Spark Therapeutics Wins Prix Galien USA Award - The Galien Foundation has awarded Spark
Therapeutics with the prestigious 2019 Prix Galien USA Award for "Best Biotechnology
Product".
Science Magazine Names Spark 2019 Top Biotech and Pharma Employer - Spark
Therapeutics ranks fifth on a list of 20 admired Top Employers in the field.
Spark Therapeutics Joins Roche Group - Definitive merger agreement closed; Spark
Therapeutics to continue operations in Philadelphia as an independent company within the Roche
Group.
Spark Therapeutics, a fully integrated, commercial gene therapy company dedicated to
challenging the inevitability of genetic disease, on 1 st June, 2020 announced the publication of
new research in the journal Nature Medicine demonstrating that treatment with immunoglobulin
G-degrading (IgG) enzyme of Streptococcus pyogenes (IdeS) resulted in rapid and transient
reduction of neutralizing anti-adeno-associated virus (AAV) antibodies and restored gene therapy
efficacy in controlled laboratory tests of animal models.
1.2. Alnylam Pharmaceuticals
INDUSTRY – Pharmaceuticals
FOUNDED – 2002
HEADQUARTER – Cambridge, Massachusetts, US
NO. OF EMPLOYEES – 1,323 (2019)
33
Alnylam is derived from “Alnilam,” the bright center star in the constellation Orion’s belt, which has
been used by navigators for thousands of years and symbolizes the passion for discovery.
Alnylam has led the translation of RNAi (RNA interference) from Nobl Prize-winning discovery into an
innovative, entirely new class of medicines. Their pioneering work has delivered the world’s first and
only approved RNAi therapeutics – ONPATTRO (patisiran) in 2018 and GIVLAARI (givosiran) in 2019.
They are advancing a deep pipeline of innovative RNAi-based medicines in four therapeutic areas:
genetic medicines, cardio-metabolic diseases, infectious diseases, and central nervous system (CNS) and
ocular diseases.
Mission –
Build a top-tier, independent biopharmaceutical company founded on RNAi.
Vision –
Harnessing a revolution in biology for human health.
1. ONPATTRO (patisiran)
Patients and healthcare Personalized support program

providers, for onpattro
2. GIVLAARI (givosiran)
Patients and healthcare Personalized support program

providers, for givlaari
1.2.3. RECENT DEVELOPMENTS

Alnylam's Response to COVID-19 - The COVID-19 pandemic is having a significant impact on
people and communities all over the world and will continue to do so for an indefinite period. At
Alnylam, we’re closely monitoring developments and guidance from governments and health
authorities on a daily basis. They are also working on a potential new treatment option to combat
SARS-CoV-2, the virus that causes the COVID-19 disease, with their partner Vir Biotechnology.
Alnylam Named #1 Biopharma Employer by Science Magazine - Alnylam has been named the
#1 science employer in biopharma for 2019 by Science Magazine and Science Careers. This is their
first appearance on the list.
34
John Maraganore in "Biotechnology in the Time of COVID-19: Commentaries from the Front
Line" – Covid-19 has fundamentally changed the way that people all over the world live and work.
In the midst of this pandemic, people and companies within the biopharmaceutical industry are rising
to the challenge of developing new vaccines and treatments, united by a common purpose to respond
to perhaps the greatest public health threat of our lifetimes. In a new e-book, dozens of biopharma
industry leaders, including the CEO of Alnylam, John Maraganore, share their perspectives from the
industry's unique vantage point on the front line.
1.3. ArrowHead Pharmaceuticals
3.1. COMPANY OVERVIEW
HEADQUARTER – Pasadena, California, U.S.
INDUSTRY – BIO-TECHNOLOGY
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that
cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead
therapies trigger the RNA interference mechanism to induce rapid, deep and durable knockdown of target
genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a
specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based
therapeutics leverage this natural pathway of gene silencing.
Pipeline Development Strategy -
Arrowhead is focused on developing innovative drugs for diseases with a genetic basis, characterized by
the overproduction of one or more proteins. The depth and versatility of our RNAi technologies enable us
to address conditions in virtually any therapeutic area and pursue disease targets that are not otherwise
druggable by small molecules and biologics.
3.2. PRODUCT PORTFOLIO

Novel Drugs to Treat Intractable Diseases –
 ARO-AATAlpha-1 Liver Disease

 ARO-APOC3 - Hypertriglyceridemia
 ARO-ANG3 - Dyslipidemia
 ARO-HSD - Liver Disease
 ARO-ENaC - Cystic Fibrosis
35
 ARO-Lung2 - COPD
 ARO-COV - COVID-19
 ARO-HIF2 - Renal Cell Carcinoma
 JNJ-3989 - Hepatitis B
 AMG 890 - Cardiovascular Disease
 ARO-JNJ1 – Undisclose
3.3. RECENT DEVELOPMENT

Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) on 17th May, 2020 announced precautionary
measures the company is taking to help protect the health and safety of its employees, partners, and
the healthcare workers and patients involved in ongoing clinical studies of Arrowhead’s
investigational medicines. Arrowhead does not expect these precautionary measures to materially
affect the projected timelines for its development programs.
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) on 3rd May, 2020 announced that it had dosed
the first patients in AROHSD1001, a Phase 1/2 clinical study of ARO-HSD, the company’s
investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with
alcohol related and nonalcohol related liver diseases, such as nonalcoholic steatohepatitis (NASH).
1.4. SQZ Biotechnologies

At SQZ they believe that cell therapies can revolutionize the way we treat diseases. The SQZ cell therapy
platform can be the foundation of a new generation of therapeutics.
36
By enabling engineering of almost any function into any cell type, the SQZ platform overcomes
fundamental barriers in the field and enables the creation of previously infeasible cell therapies. Their
current focus is on creating high-impact therapies for patients in the fields of oncology, infectious disease,
and auto-immunity. Ultimately, SQZ has the potential to create therapies addressing a broad range of
diseases.
The fundamental phenomenon underlying SQZ’s technology was discovered at MIT.

SQZ APCs –
Oncology:
 SQZ-PBMC-HPV - HPV+ Solid Tumors

 SQZ-PBMC-X - Solid Tumors
 Next Gen SQZ APC - Solid and Liquid Tumors
INFECTIOUS DISEASE:
 SQZ-ID1 - Chronic-Infectious Diseases

 SQZ-RRV - Rapid Response Vaccines
SQZ AACs –
Oncology:
 SQZ-AAC-HPV - HPV+ Solid Tumors
 SQZ-AAC-KRAS - KRAS mut Solid Tumors
 Next Gen SQZ AAC- Solid and Liquid Tumors
SQZ TACs –
Immune Tolerance -
 SQZ-TAC-AAV - Gene Therapy Repeat Dosing
 SQZ-TAC-T1D - Type 1 Diabetes
 Next Gen SQZ TACs - Multiple Diseases
1.4.3. RECENT DEVELOPMENT

SQZ Biotech snags $65M to push cancer vaccine, break into infectious disease (18 th May,
2020) – SQZ Biotech is looking to propel its lead asset, a cancer vaccine for HPV-positive
tumors, through phase 1 and expand its cell-squeezing technology beyond oncology into
infectious diseases. And it's reeled in $65 million to do it.
2020 Could be a Defining Year for SQZ Biotech (22ND JAN, 2020) – If 2019 was a
breakthrough year for Watertown, Mass.-based SQZ Biotech, 2020 could be a defining year as the
company tests is first antigen-presenting cell asset in the clinic to treat solid tumors that are HPV
positive.
37
1.5. AveXis
1. COMPANY OVERVIEW
INDUSTRY – Pharmaceutical
FOUNDED – 2015
HEADQUARTER – Chicago, United States
Rare diseases don’t feel rare to the families devastated by them. They create daily challenges that must be
faced head-on, often without the help of readily available treatment.
With cutting-edge technology, they are making progress in the treatment of rare and life-threatening
neurological genetic diseases. Their initial gene therapy product was approved by the U.S. Food and Drug
Administration for the treatment of pediatric patients less than 2 years of age with spinal muscular
atrophy (SMA). It is also being evaluated by health authorities outside of the U.S. They are also pursuing
gene therapies for Rett syndrome (AVXS-201), a genetic form of amyotrophic lateral sclerosis (ALS)
caused by mutations in the superoxide dismutase 1 (SOD1) gene (AVXS-301), and Friedreich’s ataxia
(AVXS-401).
2. PRODUCT PORTFOLIO
Our Gene Therapy Products & Pipeline –
 Spinal Muscular Atrophy IV – ZOLGENSMA
 Spinal Muscular Atrophy IT – AVXS-101
 Rett Syndrome – AVXS-201
 Amyotrophic Lateral Sclerosis (ALS) SOD1 – AVXS-301
 Friedreich’s ataxia – AVXS-401
 Undisclosed – AVXS- 501
 Undisclosed – AVXS-601
38
3. RECENT DEVELOPME
Like many organizations across the United States and around the world, AveXis is closely
monitoring the coronavirus disease (COVID-19) and its impact on patients, the SMA community,
healthcare providers, and their employees and partners.
On 27th March, 2020 AveXis receives positive CHMP opinion for Zolgensma®, the only gene
therapy for spinal muscular atrophy (SMA).
On 24th March, 2020 AveXis presents AVXS-101 IT data demonstrating remarkable increases in
HFMSE scores and a consistent clinically meaningful response in older patients with SMA Type
2.
1.6. Asklepios Bio-Pharmaceutical (AskBio)
1. COMPNY OVERVIEW –
INDUSTRY - Pharmaceutical
FOUNDED – 2001
HEADQUARTER – Research Triangle Park, North Carolina
At Asklepios BioPharmaceutical (AskBio), they are making history with every clinical advancement and
aspire to turn hope into cures by unraveling new possibilities for genetic medicine. They are
headquartered in Research Triangle Park, North Carolina, a thriving biotechnology hub with nearly 600
life science companies, and have additional research and development facilities in Edinburgh, Scotland,
and Paris, France, and gene therapy manufacturing in San Sebastian, Spain.
2. FOCUSED AREA –
 Genetic Diseases
 AAV Gene Therapy
 Pipeline
 Research Areas
3. RECENT DEVELOPMENT –
 On February 2, 2020 The first patient was dosed at The Christ Hospital in Cincinnati, and the
NAN-101 trial is actively enrolling patients.
 On DECEMBER 19, 2019, AskBio’s AAV gene therapy combined with ImmTOR could prevent
the formation of neutralizing antibodies and potentially enable re-treatment of patients with
Pompe disease.
39
1.7. Biogen (US)
1. COMPANY OVERVIEW –
FOUNDED – 1978
INDUSTRY – Bio-Technology
HEADQUARTER – Cambridge, MA
OWNERSHIP – Public
EMPLOYEES – 10,000
REVENUE –
Through cutting-edge science and medicine, Biogen discovers, develops and delivers innovative therapies
worldwide for people living with serious neurological and neurodegenerative diseases.
Founded in 1978, Biogen is a pioneer in biotechnology and today the Company has the leading portfolio
of medicines to treat multiple sclerosis, has introduced the first and only approved treatment for spinal
muscular atrophy, and is at the forefront of neurology research for conditions including Alzheimer’s
disease, Parkinson’s disease and amyotrophic lateral sclerosis. Biogen also manufactures and
commercializes biosimilars of advanced biologics.
With approximately 7000 people worldwide, they are truly a global organization, headquartered in
Cambridge, Massachusetts, which is also home to our research operations. Their international
headquarters are based in Zug, Switzerland and we have world-class manufacturing facilities in North
40
Carolina and Denmark. They offer therapies globally through direct affiliate presence in 30 countries and
a network of distribution partners in over 50 additional countries.
2. Focused Areas –
o Aducanumab (Aβ mAb) - Alzheimer’s disease
o BIIB067 (tofersen) - SOD-1-Amyotrophic lateral sclerosis (ALS)
o BIIB093 (IV glibenclamide) - Large hemispheric infarction
o BIIB111 (timrepigine emparvovec) – Choroideremia
o BAN2401 (Aβ mAb) - Alzheimer’s disease
o SB11 (biosimilar referencing LUCENTIS®) - Various ophthalmologic conditions
o BIIB054 (cinpanemab) – Parkinson's disease
o BIIB059 (anti-BDCA2) - Systemic lupus erythematosus
o BIIB074 (vixotrigine) - Trigeminal neuralgia
o BIIB074 (vixotrigine) - Small fiber neuropathy (SFN)
o BIIB092 (gosuranemab) - Alzheimer's disease
o BIIB093 (IV glibenclamide) - Brain Contusion
o BIIB104 (AMPA) - Cognitive impairment associated with schizophrenia (CIAS)
o BIIB112 (NSR-RPGR) - X-linked retinitis pigmentosa (XLRP)
o Dapirolizumab pegol (anti-CD40L) - Systemic lupus erythematosus
o Natalizumab - Epilepsy
o Opicinumab (anti-LINGO) - Multiple sclerosis (MS)
o TMS-007 (plasminogen modulator) - Stroke
o BIIB061 (oral remyelination) - Multiple Sclerosis (MS)
o BIIB076 (anti-tau mAb) - Alzheimer's disease
o BIIB078 (IONIS-C9Rx) - Amyotrophic lateral sclerosis (ALS)
41
o BIIB080 (IONIS-MAPTRx) - Alzheimer's disease
o BIIB091 (BTK inhibitor) - Multiple sclerosis (MS)
o BIIB094 (LRRK2 ASO) - Parkinson’s disease
o BIIB095 (Nav 1.7) - Neuropathic pain
o BIIB100 (XPO1 inhibitor) - Amyotrophic lateral sclerosis (ALS)
o BIIB110 (ActRIIA/B ligand trap) - Spinal muscular atrophy (SMA)
o BIIB118 (CK1 inhibitor) - Irregular Sleep Wake Rhythm Disorder (ISWRD) in

Parkinson’s Disease (PD)
July,2020 - Biogen Completes Submission of Biologics License Application to FDA for
Aducanumab as a Treatment for Alzheimer’s Disease
May 28, 2020 - Biogen and MIT Launch New Virtual Learning Lab for High School Students
Historically Underrepresented in Science
April 16, 2020 - BIOGEN, BROAD INSTITUTE OF MIT AND HARVARD, PARTNERS
HEALTHCARE LAUNCH CONSORTIUM TO BUILD AND SHARE A COVID-19
BIOBANK. Biogen employees, family members, and other close contacts can contribute blood
samples and medical data to aid efforts to rapidly understand and overcome COVID-19.
February 27, 2020 - Biogen and Sangamo Announce Global Collaboration to Develop Gene
Regulation Therapies for Alzheimer’s, Parkinson’s, Neuromuscular, and Other Neurological
Diseases.
42
1.8 Novartis AG (Switzerland)
1. COMPANY OVERVIEW –
FOUNDED – 1996
INDUSTRY – Pharmaceuticals
HEADQUARTER – Basel, Baselstadt
EMPLOYEES – 10,001+ (96,917 in Linkedln)
REVENUE –
Novartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines
company, they use innovative science and digital technologies to create transformative treatments in areas
of great medical need. In our quest to find new medicines, they consistently rank among the world’s top
companies investing in research and development. Novartis products reach nearly 1 billion people
globally and they are finding innovative ways to expand access to our latest treatments. About 125 000
people of more than 140 nationalities work at Novartis around the world.
2. PRODUCT PORTFOLIO –
 Disease Areas -
 Cancer
43
 Cardiovascular, Renal & Metabolism

 Immunology & Dermatology
 Ophthalmology
 Neuroscience
 Respiratory
Platforms and Technology -
 Data and Digital

 Cell & Gene Therapy
3. RECENT DEVELOPMENT -
Jul 07, 2020 - Novartis receives EC approval for Enerzair Breezhaler, including the first digital
companion (sensor and app) that can be prescribed alongside a treatment for uncontrolled asthma
in the EU.
Jul 02, 2020 - Novartis has finalized its previously disclosed agreement with the US Attorney’s
Office for the Southern District of New York, the New York State Attorney General, and relator
Oswald Bilotta resolving a civil suit challenging speaker programs and other promotional events
conducted from 2002 through 2011 by Novartis Pharmaceuticals Corporation in the US. As part
of this settlement, Novartis will pay USD 678 million and has agreed to new corporate integrity
obligations with the Office of Inspector General (OIG) of the US Department of Health & Human
Services that will change how the company delivers peer-to-peer programs in the US. As it has
done over the past several years, Novartis will continue to evolve its approach to peer-to-peer
medical education. The company fully provisioned for this settlement in July 2019.
Jun 29, 2020 - Novartis receives simultaneous approval for five new products from Japanese
Ministry of Health, Labour and Welfare, offering Japanese patients a broad range of novel
treatment options.
44
1.9 Orchard Therapeutics plc. (UK)
1. PRODUCT PORTFOLIO -
FOUNDED – 2015
HEADQUARTER – London, England
EMPLOYEES – 500
REVENUE –
Orchard Therapeutics is a leading global fully integrated commercial-stage company dedicated to

transforming the lives of patients with rare diseases through innovative gene therapies.
Orchard’s portfolio of autologous ex vivo gene therapy programs has demonstrated sustained clinical
benefit in over 150 patients across five disease areas. These programs include Strimvelis®, the first
autologous ex vivo gene therapy approved by the EMA in 2016, 3 programs in advanced registrational
studies in MLD (metachromatic leukodystrophy), WAS (Wiskott Aldrich syndrome) and ADA-SCID
(adenosine deaminase severe combined immunodeficiency), 2 other clinical programs in X-CGD (X-
linked chronic granulomatous disease) and beta-thalassemia, as well as an extensive preclinical pipeline.
The company is partnered with world-leading institutions in gene therapy, including University College
London, Great Ormond Street Hospital, the University of Manchester and Central Manchester University
Hospitals, the University of California Los Angeles and Boston Children’s Hospital, and Telethon
Institute of Gene Therapy/Ospedale San Raffaele.
NEUROMETABOLIC DISORDERS -
45
 METACHROMATIC LEUKODYSTROPHY (MLD

 MUCOPOLYSACCHARIDOSIS TYPE I (MPS-I)
 MUCOPOLYSACCHARIDOSIS TYPE IIIA (MPS-IIIA)
MUCOPOLYSACCHARIDOSIS TYPE IIIB (MPS-IIIB)
PRIMARY IMMUNE DEFICIENCIES -
 ADENOSINE DEAMINASE SEVERE COMBINED IMMUNODEFICIENCY (ADA-SCID

 WISKOTT ALDRICH SYNDROME (WAS)
 X-LINKED CHRONIC GRANULOMATOUS DISEASE (X-CGD)
BLOOD DISORDERS -
 BETA THALASSEMIA
July 19, 2020 - Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, and
MolMed S.p.A (MLMD.MI), one of the company’s principal contract development and
manufacturing partners, today announced that they have extended their collaboration –
initiated in April 2018 – for a period of five years through June 2025.
Jan 29, 2020 - Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today
announced that it has received orphan drug designation from the U.S. Food and Drug
Administration (FDA) for OTL-102, the company’s ex vivo autologous hematopoietic stem
cell (HSC) gene therapy being investigated for the treatment of X-linked chronic
granulomatous disease (X-CGD). The FDA may grant orphan designation to drugs and
biologics intended to treat a rare disease or condition affecting fewer than 200,000 persons in
the U.S.
1.10 Bluerock Therapeutics
2. PRODUCT PORTFOLIO -
46
FOUNDED – 2016
HEADQUARTER – Cambridge, Massachusetts
OWNERSHIP – Private
EMPLOYEES – 200
REVENUE –
BlueRock Therapeutics is an engineered cell therapy company with a mission to develop regenerative
medicines for intractable diseases.
BlueRock’s Cell+Gene platform harnesses the power of cells for new medicines across neurology,
cardiology and autoimmune indications. BlueRock’s cell differentiation technology recapitulates the
cell’s developmental biology to produce native cell therapies which are further engineered for additional
function. Utilizing these cell therapies to replace damaged or degenerated tissue brings the potential to
restore or regenerate lost function.
BlueRock was founded in 2016 by Versant Ventures and capitalized with one of the largest-ever Series A
financings in biotech history by Bayer AG and Versant.
BlueRock’s culture is defined by scientific innovation, the highest ethical standards and an urgency to
bring transformative treatments to all who would benefit. The company strives to be a top employer of
scientific talent, empowering every member of the team to make meaningful and lasting contributions to
the burgeoning field of regenerative medicine.
Over the next 12 months, BlueRock will be significantly expanding staff at all locations. Please contact
the company via LinkedIn or their corporate website to learn about rewarding career opportunities.
Area Diseases Cell Type Mechanism

Neurology Parkinson’s Disease Dopaminergic Neuron Authentic Cell
Replacement
Neurodegeneration Microglia Engineered Authentic
Cell
Demyelinating Oligodendrocyte Authentic Cell
Disorders Replacement
Hirschsprung’s Disease Enteric Neuron Authentic Cell
Replacement
Cardiology Heart Failure Cardiomyocyte Authentic Cell
Replacement
Immunology Tolerance/Fibrosis Macrophages Engineered Authentic
Cell
Graft vs Host Disease T-Reg Cells Engineered Authentic
47
Cell
January 21, 2020 - Ncardia and BlueRock Therapeutics Announce Collaboration Agreement and
Licensing of Process Development Technologies for the Manufacture of iPSC-derived
Cardiomyocytes
August 8, 2019 - Bayer AG and BlueRock Therapeutics today announced an agreement under
which Bayer will fully acquire BlueRock Therapeutics, a privately held US-headquartered
biotechnology company focused on developing engineered cell therapies in the fields of
neurology, cardiology and immunology, using a proprietary induced pluripotent stem cell (iPSC)
platform.
8. CASE STUDY – A SUCCESS STORY OF NOVERTIES WITH THE INNOVATIVE

IDEA OF JOE JIMENEZ, CEO OF NOVERTIES -
PROBLEM –
the company was launching a lot

of products, but many were
unsuccessful once they hit the
marketplace.
CHALLENGES -
To create an innovative &

successful molecule to compete
the market.
CASE –
When Jimenez joined Novartis as the CEO, the company was launching a lot of products, but many were
unsuccessful once they hit the marketplace. He felt the company was not thinking about the commercial
potential of company products in the pipeline. To better oversee the income potential of treatments,
Jimenez created a group called early commercial development, which he calls the commercial conscience
of research.
“That was a little nerve wracking,” he now says. “I was the only individual on the Innovation
Management Board with an equal vote who was not an MD or a PhD. This got our scientists to realize
that it's not good enough to create a molecule. You have to think about when you're going to launch that
molecule and what the competitive landscape is going to look like.”
48
When Jimenez made the move from Big Pharma to small biotech, he also learned about how quickly
companies can move. At Novartis, he remembers being in meetings that would last hours simply trying to
make a decision that should have taken five minutes. He is now an advisor for several small biotech
companies where those long meetings do not take place.
“Bureaucracy doesn't necessarily have to be a bad word,” he admits. “Bureaucracy can certainly mean red
tape, but if you have a huge pharmaceutical company without a clear decision making structure, what you
are left with is chaos.”
THE SOLUTIONS –
One successful strategy that was used at Novartis was in creating a Venture Group, which Jimenez
describes as a successful and independent effort. The company put money into a venture fund and told the
fund to do its thing. It had access to the resources of Novartis but was not linked to the company.
The company was the first to pursue a CAR T therapy. When Novartis invested in the therapy, it still
appeared to be science fiction.
They were discussing the concept of taking blood from a cancer patient, genetically engineering the T-
cells in that blood to recognize and go after the tumor, and then reinfuse it back into the patient. This was
in 2012, their oncology people coming into the office with stunned looks on their faces. They had just
become aware of Carl June's work at the University of Pennsylvania. His early work was with chronic
lymphocytic leukemia, and his CAR T therapy was being used on patients who had no other hope.
Novartis looked at June’s work and decided they had to be involved with it. At the time, no other large
pharma companies opted to make an investment. Kite and Juno were two biotech’s that emerged in the
space and were quickly purchased by Big Pharma.
RESULT –
Now that gene editing is a reality, Jimenez believes regenerative medicine holds huge hope for patients.
The U.S. population is aging, and Novartis has been investigating how treatments can increase life
expectancy and prolong vision, hearing, and muscle as humans age. These are areas where there is
significant degeneration.
Today around 40 percent of all investment is going into the area of immuno-oncology. Jimenez believes
that’s good, since patients will now have new treatment options that they never thought possible. He notes
there are around 3,500 immuno-oncology therapies currently being investigated. Still, he worries about
what that might mean for more traditional diseases that still have a large unmet medical need. For
example, he sees some companies almost completely abandoning respiratory disease, rheumatology,
dermatology, GI, and cardiovascular disease. While the news is good for oncology patients, the swing in
funding could leave other patients in need.
49
9. CASE STUDY - Drug Launch: Zolgensma

PROBLEM –
There were no treatments to offer the

families of babies born with a genetic
disease.
CHALLENGE –
To came up with a successful solution

for this disease when there were no
medicines & no other solution existed
for the degenerative condition called
spinal muscular atrophy, or SMA.
Another challenge was to get the approval.
CASE –
Up until three years ago, Vamshi Rao, a neurologist, didn't have any treatments to offer the families of
babies born with a genetic disease that meant they likely wouldn't live past the age of two.
No medicines existed for the degenerative condition called spinal muscular atrophy, or SMA. So Rao, a
neurologist at the Lurie Children's Hospital of Chicago, could only help manage expectations and day-to-
day care.
SUCCESS –
Approval was a major accomplishment in a field that had for decades held the potential to develop
curative fixes for devastating genetic disorders like SMA.
Rao’s role, and that of other physicians, changed dramatically in 2016, when Biogen's Spinraza became
the first drug to win U.S. approval for the condition, and then again this May, when the Food and Drug
Administration cleared a gene therapy called Zolgensma.
Rao can now give families options that represent a realistic shot at a longer, better life for babies with
SMA.
Between May and October, about 100 families facing the diagnosis opted for Zolgensma, just the second
gene therapy for an inherited disease to reach commercial markets in the U.S. Unlike Spinraza,
Zolgensma is designed to be a one-time treatment and, possibly, a cure.
RESULT -
About 50 other U.S. institutions have administered Zolgensma commercially, outside of clinical testing.
The promise of lifelong benefit was central to Novartis' justification for pricing Zolgensma at $2.1 million
per patient, more than any other drug before it.
50
While insurers initially balked at covering such a pricey, one-time treatment, Novartis says coverage is
now in place for 90% of eligible patients on commercial plans and 30% of those on Medicaid.
Spinraza costs $750,000 for the first year of treatment, and $375,000 thereafter — potentially making
$2.1 million more palatable if Zolgensma truly is a one-and-done treatment.
51
8. LEARNING/ FINDING
Learning -
1. From this internship I got a basic idea about the market research & consulting industry. I got to know
that what strategy they follow for brand awareness.
2. Also learnt that how to create Press Released Table Of Content and after that how to create the sample
research report paper.
3. Apart from this I got some idea about company profile listing in different platforms. And basic idea
about that how to create infographic for business perpous.
4. Through the live project I got to know the different strategies to gather the relevant secondary data and
also how to analysis those for the final research report.
Overall it was a great experience to work with this company. I learnt a lot as a fresher, & this helped me
to enhance my knowledge also.
Findings –
1. The growth rate of the consulting industry was increasing rapidly. It was around 30% but due to
COVID-19 the growth decreased. In 2019 the market size was US&170 bn but in 2020 It became
US$ 130bn.
2. The estimated growth rate of Gene therapy market was to grow from USD 3.8 bn in 2019 to USD
13.0 bn by 2024 but due to COVID-19 the growth in 2020 has been decreased.
10. REFERENCES
1. https://chromatus.com/tracker-studies/
2. https://www.statista.com/
4. https://www.researchgate.net/
5. https://www.editage.com/insights/how-to-write-the-rationale-for-research
6. https://www.moneycontrol.com/stocksmarketsindia/
7. https://www.bloomberg.com/
8. https://www.marketsandmarkets.com/
9. http://www.aceanalyser.com/Analyser.aspx?MenuCode=C3
10. www.indianchamber.org
11. https://www.ibef.org/
12. https://www.counterpointresearch.com/
13. https://www.openbusinesscouncil.org/consulting-industry-market-research/
53

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SUMMER INTERNSHIP

A STUDY ON GENE THERAPY & ITS FUTURE PROSPECTS IN INDIA

under the guidance of

VIJAY NATEKAR, Operation Head

BIBHAS B., Dean

CERTIFICATE FROM THE CROMATUS CONSULTING

(Certificate from the Institute- it should be in color. This header and

Signature of Internal Guide

Signature of Director / COE

1.9 Orchard Therapeutics plc. (UK)..................................................................................................................45

A Study on Gene Therapy & Its future

Student Name Priyanka Saha

Student Roll No 2019-2005-0001-0040

Project Guide Name (External Mentor) Mr. Vijay Natekar

Duration of project 60 days

3. OBJECTIVE OF THE STUDY

1. To get the overview of the industry

2. To understand the current market growth of the industry

3. To understand the covid-19 impact on the industry

4. To understand the new trends in that particular industry

5. To understand the gene therapy Market

6. To understand the current scenario of Gene therapy in India

4. ABOUT THE SECTOR

HISTORY OF THE SECTOR -

CURRENT INDUSTRY OVERLOOK -

Major (Strategy-Focused): Consulting geared towards high-level, corporate decisions. This category

Information Technology (IT): Consulting that focuses on Information Technology development in

Niche: Consulting that tends to focus on a specific technology, research, or analysis method, or is

HISTORICAL GROWTH OF THE SECTOR –

5. ABOUT THE COMPANY & DEPARTMENT

About the company -

Product Portfolio & Services –

1 . P RIMA RY MA RKE T RESEA RCH

CONSUMER Any marketer wants to have an understanding of the

to reach the end-users in a more innovative way and find a

building appropriate strategies. Chromatus conducts

SURVEY analytical tools and statistical techniques.

1.3. YEARC CONSIDERED –

2.5. RESEARCH ASSUMPTION –

o COMPLEX MARKET ACCESS DYNAMICS

3.1. VIRAL VECTOR

5. GENE THERAPY MARKET SEGMENTATION BY APPLICATION

4.1. NEUROLOGICAL DISEASES

4.4. ONCHOLOGICAL DISEASES –

4.5. OTHER DISEASES –

1.1.1. COMPANY OVERVIEW

INDUSTRY - Biotechnology Pharmaceutical

HEADQUARTERS – Philadelphia, Pennsylvania

NO. OF EMPLOYEES – 368 (2019)

At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing

A world where no life is limited by genetic disease.

1.1.2. PRODUCT PORTFOLIO

Learn more Learn More Learn More

3. Scientific platform & programs –

Cutting-edge vector State-of-the-art, in-house expertise in vector Strong commitment to

1.1.3. RECENT DEVELOPMENTS

1.2.1. COMPANY OVERVIEW

HEADQUARTER – Cambridge, Massachusetts, US

NO. OF EMPLOYEES – 1,323 (2019)

Build a top-tier, independent biopharmaceutical company founded on RNAi.

Harnessing a revolution in biology for human health.

1.2.2. PRODUCT PORTFOLIO

Patients and healthcare Personalized support program