Transcript slides - The National Academies of Sciences, Engineering, and

NAS Public Meeting – February 11, 2016
Michael Werner
Executive Director
Alliance for Regenerative Medicine
ARM’s Role in the Sector
As the leading global advocate for the regenerative medicine and advanced therapies
sector, ARM enables acceleration of research, development, investment and
commercialization of transformational treatments and cures for patients worldwide.
• Drives regulatory, scientific and policy advancement
• Maximizes market access and reimbursement
• Enables sustainable access to capital
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Promise & Potential of Genome Editing in Somatic Cells
•
Genome editing technologies (including zinc finger nucleases, CRISPR/Cas9
and TALEN-based methods) represent a powerful new approach for targeting
and changing DNA sequences in somatic (non-reproductive) human cells
•
These technologies hold significant promise to durably treat and potentially
cure devastating diseases and conditions, several with currently high degrees
of unmet medical needs.
•
Such diseases include several forms of cancer; HIV/AIDS; ocular disorders;
hemoglobinopathies and other blood disorders; neurodegenerative disorders;
and various rare or orphan diseases, such as Duchenne muscular dystrophy,
sickle cell disease, lysosomal storage disorders and many more.
• The patient population could number in the hundreds of thousands,
possibly millions, worldwide
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ARM’s Position on Human Germline Editing
ARM encourages and promotes the use of genome editing technologies in somatic (nonreproductive) human cells to durably treat and potentially cure devastating human diseases and
conditions.
In our Nature commentary piece, published March 2015, ARM called for a temporary moratorium on
research involving the using of genome editing technologies and their use on human germline cells.
Following the December 2015 NAS summit on Human Gene Editing, ARM stated that until the many
unresolved safety, ethical and legal concerns surrounding the use of gene editing techniques to
manipulate or modify the human germline are adequately addressed, our position is that pursuing
this line of research is not appropriate at this time.
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Potential Commercial Landscape
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Somatic cell genome editing therapeutic products offer many potential commercial opportunities.
•
Given the sizeable patient community and the unparalleled therapeutic benefits these technologies stand to
offer, analysts estimate the global genome editing market could surpass $3.5B (USD) by the end of this
decade.1
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CRISPR and other genome-editing technologies also offer the promise of replacing less efficient and costlier
procedures, making drug discovery more targeted and addressing the root cause of disease, rather than
managing the symptoms, offering significant additional economic benefits for the healthcare system.
•
Given the promising scientific breakthroughs gene editing technologies represent, this field’s complex IP
landscape continues to evolve, the results of which will have significant impact on the field and future
commercial landscape.
“Genome Editing / Genome Engineering Market by Application (Cell Line Engineering, Animal & Plant Genetic Engineering), Technology (CRISPR,
Antisense, TALEN, Zinc Finger Nuclease), & End User (Biotechnology & Pharmaceutical, CRO) - Global Forecast to 2019”, marketsandmarkets – April 2015
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Present Commercial Landscape
The present commercial landscape for genome editing includes a number of different types of companies: product developers, tools
and reagent providers, pharmaceutical companies, contract research organizations and more.
This includes the following:
Abeona Therapeutics
Addgene
Advanced Analytical Technologies
American Type Culture Collection
AstraZeneca
Batu Biologics
Bayer Healthcare
Biocytogen
Biogen
BIO-RAD Laboratories
bluebird bio
Bristol Myers-Squibb
Caribou Biosciences
Cellecta
Celgene
Cellectis
Cibus
CRISPR Therapeutics
Cyagen
Dharmacon
Dow
Editas Medicine
GlaxoSmithKline (GSK)
Genecopoeia
Please note: this is not an exhaustive list of all the companies active in genome editing
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GE Healthcare
GenScript
Herabiolabs
Horizon Discovery
Intellia Therapeutics
Integrated DNA
Technologies
Juno Therapeutics
Merck
MirusBio
Novartis
Orig3n
Pfizer
Poseida Therapeutics
Recombinetics
Sangamo BioSciences
Shire
SigmaAldrich
ThermoFisher
Transpogen
Twist Bioscience
Vertex Pharmaceuticals
Therapeutic Developer Case Studies
bluebird bio
Type of genome editing technology:
• bluebird bio employs megaTALs / homing endonuclease-based technology for generating precise genetic alterations to target
cells.
Stage of development:
• bluebird’s genome editing technology is currently in pre-clinical development and is being investigated across a range of potential
applications (including T cells for oncology).
• The first publicly announced application of bluebird’s genome editing technology is in collaboration with Kite Pharma in the context
of a HPV-specific TCR-based T cell immunotherapy.
Disease focus areas:
• T cells for oncology and other yet-undisclosed applications
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Therapeutic Developer Case Studies
Sangamo BioSciences
Type of genome editing technology:
•
Proprietary technology based on the engineering of a naturally occurring class of DNA-binding zinc-finger proteins (ZFPs).
Stage of development:
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Phase 2 clinical program to evaluate the safety and efficacy of novel ZFP Therapeutics for the treatment of HIV/AIDS (SB-728)
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Phase 1/2 clinical program to evaluate safety and efficacy of its IVPRP approach for hemophilia B.
•
FDA cleared Sangamo’s IND application for its Hurler syndrome (MPS I) program at the end of 2015, with the goal of initiating a
Phase 1/2 clinical trial in the first half of 2016 (IND applications cleared Feb 2016)
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Goal to file six more IND applications to move its other preclinical programs into the clinic by the end of 2016, including IND
applications for Hunter syndrome (MPS II) and beta-thalassemia (in collaboration with Biogen) in the first half of the year, and
IND applications for its programs in hemophilia A, Gaucher, Fabry and sickle cell disease (Biogen), in the second half of 2016.
Disease focus areas:
•
Using its proprietary IVPRP, Sangamo is currently developing one-time, life-long therapies for blood and lysosomal storage
disorders, including hemophilia A and B; Hurler syndrome (MPS I); Hunter syndrome (MPS II); Gaucher disease; Fabry disease.
•
Sangamo is implementing its ex-vivo ZFN-mediated genome editing strategy to develop one-time treatments for
hemoglobinopathies and infectious diseases, including beta-thalassemia; sickle cell disease; HIV/AIDS.
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Supporting Genome Editing’s Therapeutic Potential
The potential of somatic cell genome editing for research
and therapeutic product development purposes is
extremely promising and progressing rapidly
These products fit within the overall existing regulatory
framework:
- No need for a unique oversight process
- Gene editing products must undergo the same level of
regulatory review as other biotech products, as
appropriate
- NIH-RAC – public discussion of novel technologies
- ARM has several proposals designed to streamline the
regulatory review process
It is ARM’s goal to ensure there is no legislative or
regulatory action taken that would hinder or delay the
therapeutic use of these techniques
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