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Advancements in Gene Therapy, Synthetic Biology, Diagnostic Assays, Imaging Tracers, and Biopharma - Life Sciences, Health & Wellness Techvision Opportunity Engine
Advancements in Gene Therapy, Synthetic Biology, Diagnostic Assays, Imaging Tracers, and Biopharma - Life Sciences, Health & Wellness Techvision Opportunity Engine
D759-TV
January 18, 2019
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Table of Contents
Powerful topics in life sciences
Tau PET Imaging Tracer, APN-1607 Licensed for use in Tauopathy Clinical Studies 14
Prognostic and Diagnostic Cancer Screening Assays 15
®
Encouraging Interim Clinical Results of GoCAR-T Cell Therapy Candidate 16
Industry Relations 17
Exploring the Potential of Novel Platforms in Life
Sciences
D759-TV 3
Exploring the Potential of Novel Platforms in Life Sciences
Growth Opportunities in Life Sciences Research
1
Cyrus Bench™
12 Cyrus
Controllable Biotechnology 2
CAR-T On/Off Synbio
candidate Switches
11
Cancer Bellicum Synvivia 3
Screening Pharma On-Point
Assays Solutions
Contextual Elemental
Genomics Machines
10
Tau Imaging 12
Tracer Disruptive 4
APRINOIA Life Sciences Extremozymes
Therapeutics Stories iXpressGenes
9
Allogenic Cell
Therapy 5
Fate IQVIA CORE
Therapeutics 8 6 IQVIA
Biopharma Homologous
Development Recombination
Batavia Homology
7
Synthetic Biology Biosciences Viral Vector Medicines
Cyrus Biotechnology Cyrus Bench can be Cyrus Bench comprises an Cyrus partners with Fortune
launched Cyrus Bench, a accessed on the cloud, optimal array of molecular 500 pharmaceutical firms,
modern version of the well- where Rosetta’s capabilities modeling software, including 10 of the top 20
proven Rosetta molecular are provided completely, including sequence and companies. Cyrus also
modeling and protein design including the associated structure alignment. provides technologies for mid-
software package. array of bio-molecular Protein modeling, which sized biotechnology firms and
Cyrus Bench leverages computation tools. conventionally requires tens startups.
Rosetta’s powerful protein Cyrus developed a custom- or hundreds of thousands of Among the well-renowned
structure prediction, designed Rosetta’s graphical computational processors, customers, Cyrus provides
computational protein design, user interface (GUI) to enable are accessible on the cloud. support to Janssen
protein binders, and the automated attachment of Workflow is intuitive, Pharmaceuticals, BASF,
antibody-like affinity different sets of standard automated, and simplified. Illumina, and Harvard Medical
modeling. user procedures. School.
5
On/Off Switches for Genetically Modified Organisms
Synvivia, Berkeley, CA, US
• Synvivia excels in the synthetic biology space with the • Synvivia’s genetically encoded biocontainment system uses
development of organisms capable of being controlled via small programmable, self-replicating, engineered organisms to be
molecules. applied across many industries and sectors such as life
• Organisms’ cells can be either On or Off; the small molecule science therapeutics and diagnostics, advanced agriculture,
based control system decides about the organism activity by and molecular chemistry.
switching these binary parameters. • Synvivia’s technology makes use of synthetic biology by
• These On/Off switches are installed in the organism, so that facilitating the growth, spread, and modification of the
Synvivia can engineer these organisms to facilitate their control environment, taking into consideration all potential risks.
by using small molecules. • While natural organisms have evolved over billions of years,
• This technology is based on Synvivia’s lead product, a synthetic organisms are relatively new. Hence, synthetic
genetically encoded biocontainment system that can be applied organisms must be strictly controlled outside the lab.
to therapeutic organisms.
Technology Transfer
Commercially Available Synthetic Biology Developed at UC Berkeley and financially supported by NSF and
DARPA, Synvivia's technology has found a market niche for its
commercial synthetic biology product.
Synvivia launched the first Synvivia emphasizes the
commercially available relevance of developing Business Model
genetically encoded high-performance control • Synvivia holds the exclusive license for this technology to be
biocontainment system. This systems in genetically applied in all fields. Synvivia is applying for funding to Y
system enables organism engineered organisms to Combinator and the National Institutes of Health, to optimize the
control through specific small guarantee the safe use of market penetration of its genetically encoded biocontainment
molecules, especially designed synthetic biology outside of technology to diversify it in a complete suite of products.
by Synvivia. the laboratory.
Source: Frost & Sullivan
6
On-Point Solutions for a Smart Lab
Elemental Machines, Salem, New Hampshire, US
Artificial Intelligence (AI) meets life sciences by introducing the concept of ‘smart lab’.
Elemental Machines provides on-point solutions to achieve this goal by leveraging AI-based technology
platforms to enhance pre-clinical outcomes and laboratory compliance.
7
Extremozymes for Therapeutics Development
iXpressGenes, Huntsville, AL, US
iXpressGenes is a synthetic biology company located at the HudsonAlpha Institute for Biotechnology, focused on
protein and genetic engineering research in extreme conditions.
Seeing Beyond the Ecosystem
Deep Sea
(high
pressure and
Extreme low
Environment temperature)
Micro-
Gravity Unique Research
(near-zero Tool:
gravity) Trace Fluorescent
Salted Labeling
Complex Media (high
Genome solute
Evolution concentrati
on)
According to Frost & Sullivan’s research, iXpressGenes is revolutionizing therapeutics design through the discovery of
proteins and enzymes from genomes subjected to extreme environments, which have special qualities that can be used in
synthetic biology approaches to engineering and metabolic reactions.
8
Leveraging Human Data Science and Advanced Analytics for Clinical
Genomics
IQVIA
9
Leveraging Homologous Recombination for Gene Editing
Homology Medicines
Homology Medicines Poised to Transform the Gene Editing and Gene Therapy Landscape through
Natural Homologous Recombination-based Technology
10
Advancing the Manufacture of Viral Vectors
Vigene Biosciences
Vigene is a gene therapy company/CDMO that can help gene • Vigene’s value proposition is that they are only focused on
therapy developers from a gene therapy conception stage all the viral vectors and offer unprecedented expertise in that space.
way to the commercialization stage. The company has multiple The company has virologists, biomedical engineers,
business units and offers research grade production for proof-of- mechanical engineers and hence, the company can
concept animal studies. Vigene’s research grade business unit is manufacture viral vectors with high potency and consistency.
the largest commercial company in the world to distribute research The focus and technology capabilities in the viral vector space
grade biovectors. The company is the only licensee for AB is the key competitive differentiation that Vigene has to offer in
Biosciences and is the only distributor for Michael J Fox Foundation the global biomanufacturing landscape.
of Parkinson’s Disease for viral vector research reagents.
• The company’s key focus • Key Vector Types: • Vigene acquired Omnia Biologics (Rockville) in March 2017 for
areas comprise three • Plasmid, Adenoviral, an undisclosed sum. The purchase also included Omnia’s
areas of viral vector Lentiviral and AAV 7,000-square-feet cGMP facility, which houses three
production: manufacturing suites and a process development laboratory for
• 1) Gene therapy viral vector production.
• Key AAV serotypes: • Vigene Biosciences announced a collaboration with Virovek to
viral vector,
including AAV, • AAV1, AAV2, AAV5, make high yield cGMP AAV production technology accessible
and the lentivirus AAV7, AAV6, AAV8, and affordable.
AAV9 & AAV-DJ
• 2) Viral Vaccine Frost & Sullivan’s Verdict: Vigene has successfully addressed
production key industry challenges related to payload, scalability, and
• 3) Plasmid based • Key Cell Types: flexibility across the global gene therapy CDMO landscape and
is a leader of both research-grade and cGMP-grade AAV,
production • C19, SF9
lentivirus, plasmid, and adenovirus-based production platforms.
Source: Frost & Sullivan
11
Accelerating the Development of Biopharmaceutical Products
Batavia Biosciences
Batavia offers an unprecedented range of technology and services for enabling the development of a
wide range of biopharma candidates.
12
Promising Preclinical Data for off-the-shelf Cell Therapy
Fate Therapeutics, San Diego, CA, US
FT500 is the acronym assigned by Fate Therapeutics for its off-the-shelf immunotherapy for the
treatment of advanced solid tumors. FT500 in combination with an immune checkpoint blocker at the
preclinical investigation stage has shown good results with the company having landed an IND approval
from the FDA.
13
Tau PET Imaging Tracer, APN-1607 Licensed for Use in Tauopathy Clinical
Studies
APRINOIA Therapeutics, Taipei City, Taiwan
APRINOIA Therapeutics has provided a nonexclusive license to Celgene for the use of the company’s [18F]-labeled
investigational imaging tracer, APN-1607 in Tau PET imaging. APN-1607 was awarded the orphan drug designation
for progressive supranuclear palsy in 2017.
Celgene intends to employ APN-1607 for selection of patient population and measurement of efficacy in its tauopathy
clinical studies globally.
APN-1607
Diagnostic
Imaging
Frost & Sullivan’s comments: Celgene’s interest in utilizing APN-1607 indicates the strength of APRINOIA Therapeutics’
technology and the versatile applications of the Taiwan-based company’s imaging tracers in the Tau disease modality.
14
Prognostic and Diagnostic Cancer Screening Assays
Contextual Genomics, Vancouver, BC, Canada
Contextual Genomics has successfully launched an improved version of its molecular hotspot detection assay
with enhanced mutation detection panel and also a new liquid biopsy assay to help track disease progression.
The first version of the Find It The screening panel is similar to the The quality control of the next-
panel was launched in 2016. Find It panel, but the liquid biopsy generation sequencing assays is
The assay has a screening assay screens for mutations in the completely automated. The NGS
panel for 146 somatic genome cell-free circulating tumor DNA found assays are equipped with the patented
alterations, and 23 exons in 30 in blood plasma. While the Find It Quality Nexus platform that oversees
cancer-associated genes. The panel serves as a preliminary computational quality and facilitates
molecular hotspot assay would investigational tool, the Follow It detection of mutations by using a large
offer insights, enabling panel would serve as a monitoring number of proprietary molecular
precision treatment for cancer tool that can highlight tumor burden barcodes. The platform also can help
patients. For example, and any observed drug resistance. It identify true mutations from molecular
identification of specific gene can help highlight disease diversity indices through a machine
mutations can reveal drug progression for a better treatment learning approach focused on single
resistance, immunodeficiency outcome for patients, especially nucleotide variants and molecular
and so on. ones with metastatic cancers. barcoding analysis.
Frost & Sullivan’s Analysis: The next-generation sequencing assays are great point-of-care diagnostic aids that can help patients
receive the best care as they enable doctors to provide a customized treatment approach.
Bellicum Pharmaceuticals’ much anticipated GoCAR-T® candidate, BPX-601 has displayed positive initial
results with respect to the safety and activity of the therapy. The therapy is being assessed for its
effectiveness in the treatment of solid tumors.
Outcome: The treatment control provided by GoCAR-T could possibly provide unique treatment benefits. Solid tumors have
been the most difficult forms of tumors to treat but the GoCAR-T therapy provides encouragement, as the adenocarcinoma
disease modality is in a critical need for better treatments. On the safety aspect of the study, no adverse event was reported,
including no CRS or neurotoxicity that occur as a result of CAR-T cell therapy. Other adverse effects that are experienced by
cancer patients usually undergoing chemotherapy or immunotherapy were observed though.
Source: Frost & Sullivan
16
Industry Relations
D759-TV 17
Industry Relations
Contact Dashboard
Contact Company/Details
1 Cyrus Biotechnology Dr. Lucas Nivon, CEO. 500 Union St. Suite 320, Seattle, WA 98101.
Phone: +1-206-258-6561. E-mail: lnivon@cyrusbio.com URL: https://cyrusbio.com/
2 Synvivia Gabriel Lopez, Ph.D., CEO, Founder. Address 2341 Grant St Unit B Berkeley, CA 94703-
2570. Phone: +1-571-306-5201. E-mail: glopez@synvivia.com.
URL: https://www.synvivia.com/
3 Elemental Machines John Morgan, Director of Marketing. Salem, New Hampshire, 03079.
Phone: + 1-978-289-7738. E-mail: morgan@elementalmachines.io.
URL: https://elementalmachines.io/
5 IQVIA Tor Constantino, Media Relations, 4820 Emperor Blvd. Durham, NC 27703.
Phone: 484-567-6732. E-mail: tor.constantino@iqvia.com, URL: https://www.iqvia.com/
6 Homology Medicines Theresa McNeely, SVP, Corporate Communications & Patient Advocacy, One Patriots Park,
Bedford, MA 01730. Phone: +1-781-691- 3510. E-mail: tmcneely@homologymedicines.com,
URL: https://www.homologymedicines.com
18
Industry Relations
Contact Dashboard
Contact Company/Details
7 Vigene Biosciences Jeffrey Hung, Chief Commercial Officer, Suite 105, 9430 Key West Ave, Rockville, MD 20850.
Phone: 301-251-6638. E-mail: jhung@vigenebio.com, URL: https://www.vigenebio.com/
8 Batavia Biosciences Bioscience Park Leiden, Zernikedreef 16 2333 CL Leiden, The Netherlands.
URL: https://www.bataviabiosciences.com/
9 Fate Therapeutics Scott Wolchko, President & CEO. 3535 General Atomics Court, Suite 200, San Diego, CA 92121.
Phone: 858-875-1800. E-mail: info@fatetherapeutics.com URL: https://fatetherapeutics.com/
10 Aprinoia Te-Yu Chen, Business Development Associate. 17F, No. 3, Park Street, Nangang District, Taipei
Therapeutics City 11503, Taiwan. Phone: 86-512-8777-8382. E-mail: tychen@aprinoia.com.
URL: https://www.aprinoia.com/
11 Contextual Celia Courchene, President. Suite 204 Donald Rix Building, 2389 Health Sciences Mall,
Genomics Vancouver, BC V6T 1Z3 Canada. E-mail: info@contextualgenomics.com
URL: https://contextualgenomics.com/
12 Bellicum Rick Fair, President & CEO. 2130 West Holcombe Boulevard, Suite 800, Houston, TX 77030.
Pharmaceuticals Phone: 832-384-1100. E-mail: info@bellicum.com URL: http://www.bellicum.com/
19
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Vice President Vice President
California, USA Chennai, India
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Practice Leader Chennai, India
Innovation Services
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Senior Research Analyst Global Practice Leader
Chennai, India Innovation Services
Cecilia Van Cauwenberghe, PhD Frost & Sullivan, TechVision
Senior Industry Analyst Phone: +1 650-776-7413
Associate Fellow E-mail: sbasu@frost.com
Buenos Aires, Argentina URL: www.frost.com/techvision
20