Overview and Industry Trends:

Gene Therapy in US
Himani Chhabra
2111114
IIM Udaipur
 Introduction of Gene Therapy
Introduction
• Developed to treat diseases by altering genetic information
• Used to fix a faulty gene in order to cure a specific disease or to help the body fight diseases more effectively
• Many target diseases, including as cancer, Alzheimer's disease, and others, are becoming more common, and some gene therapies are now on the
market to treat them
• WHO estimates, that 17.9 million people died, which 35% of total deaths because of cardio vascular diseases, of which many can be cured by gene
therapy

Gene Therapy and Viral Vector

• In recent years, viral vector applications have made a promising new start in gene therapy
• Viral vector-based therapy has advanced to the forefront of modern medicine because to significant advancements in vector engineering, delivery, and
safety
• Immunotherapy has recently advanced, allowing for both preventative and therapeutic methods.
• Furthermore, gene silencing with a reversible effect has emerged as a viable option that is well-suited to viral vector distribution.

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 Size and Growth Forecast
Global Market Outlook

According to Grand View Research :

• In 2021, the Gene Therapy Market worth USD 3.42 billion, expected
Gene Therapy Growth Forecast (Global)
to reach USD 20 billion by 2028
• Expected CAGR is 20.4 % from 2021 to 2028
• Largest Market: North America and Fastest Growing: Asia Pacific 20.00
• Key Players: Biogen, Novartis, Gilead Sciences, AnGes, Pfizer and

USD Billions
many more 15.53
12.07
9.38
USA Market Outlook 7.29
5.66
4.40
According to PSmarketresearch North America constitutes the total 3.42
of 58% of total market share which is nearly USD 2.0 billion 2021 2022 2023 2024 2025 2026 2027 2028
USA will also nearly follow the world trend with CAGR of 20.4 % Year
The fastest growing market is Asia Pacific and there are some
countries which may constitutes more than the mentioned CAGR,
particularly India and China
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 Effect of Covid-19
Negative Effects
• Certain Gene Therapy companies have been hit particularly hard due to their Supply Chain of Gene Therapy
sophisticated production and distribution mechanism, as well as their funding
model
• Some 40 percent of survey respondents report disruption to their operations,
reflecting a more complex manufacturing and delivery model
• As a result of the delays, some CGT companies are having difficulty raising
further funds. In the next 12 to 24 months
• 43 percent of small to midcap public CGT companies in the United States are
projected to require new capital
• 45% respondents revealed that there will be delay in development programs
because of disruptions in R&D activities in Cell and Gene Therapy
• Man power was one of the major reason for the less efficiency of the sector, as
they were not working as per their full capacity
Source: McKinsey and Company

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 Effect of Covid-19
Positive Effects
• The application of gene and cell therapy techniques in the creation of
Working of Gene Therapy
COVID-19 vaccines is likely to be widespread
• Adeno-associated viral vectors are being used by the researchers to carry
lab-made antibodies into the body. These advancements are projected to
have a beneficial impact on market growth
• In animal models for the treatment of COVID-19, vaccine candidates
produced at Mass General Brigham and made using gene-therapy
technology elicited powerful immune responses.
• The vaccine candidate was named AAVCOVID and the researchers
received a USD 2.1 million grant to further develop the technology from
the Bill & Melinda Gates Foundation
• The vaccine which is being developed is likely to be far more convenient
than the ones being sold in the market currently, as it has single-dose and
can be stored at room temperature
Source: www.nature.com

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 Key Trends
• Various gene therapy strategies are now being used in the treatment of cancer.
• Anti-angiogenic gene therapy, pro-drug activating suicide gene therapy, gene therapy-based immune modulation, oncolytic virotherapy, gene
correction/compensation, antisense, genetic manipulation of apoptotic and tumour invasion pathways, and RNAi techniques are only a few examples.
• These medicines have been used to treat cancers such as brain, lung, breast, pancreas, liver, colorectal, prostate, bladder, head & neck, skin, ovarian,
and renal cancer.
• One of the most difficult parts of modern gene therapy has been transferring genes into cancer cells.
• Researchers are looking on ways to fix the flaws in this technique. Genes are delivered to cancer cells via a vector, which is a type of carrier.
• Viruses are the most popular carriers used in gene therapy because they can infiltrate cells and carry genetic material.

Source: Globocan 2020

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 Future Prospects
• Gene therapies are currently applied to diseases caused by only one genetic mutation
and to more accessible regions of the body, such as the liver or eyes, due to technical
limitations
• This is the goal of a new generation of gene therapy, which aims to use a variety of
methods to put genetic material into a patient that can subsequently be turned on or
off, and the strength dialled down or up
• Patient organisations are becoming more active in the development of gene therapies.
Leaders in the gene therapy industry discussed how the role of patients in gene
therapy development is shifting in a recent panel discussion at Bio-Europe Spring
2021
• The gene therapy profession saw a shift in stakeholders as its popularity expanded
• Data sharing would be something to focus on in the future, it would be very beneficial
for the future generations