As part of the mini-symposium entitled  Shark Tank-UMass Spin-out Life Sciences Start-ups,  Dr. Gao discusses an example of UMass life sciences technology that has been spun out into a start-up company -- Voyager Therapeutics from UMass Worcester

Gao, Guangping

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University of Massachusetts Medical School eScholarship@UMMS UMass Center for Clinical and Translational Science Research Retreat 2016 UMass Center for Clinical and Translational Science Research Retreat May 20th, 11:15 AM Voyager Therapeutics - A Spinout from UMass Gene Therapy and RNAi Technologies Guangping Gao University of Massachusetts Medical School Let us know how access to this document benefits you. Follow this and additional works at: https://escholarship.umassmed.edu/cts_retreat  Part of the Genetics and Genomics Commons, Pharmaceutics and Drug Design Commons, Therapeutics Commons, and the Translational Medical Research Commons Gao G. (2016). Voyager Therapeutics - A Spinout from UMass Gene Therapy and RNAi Technologies. UMass Center for Clinical and Translational Science Research Retreat. Retrieved from https://escholarship.umassmed.edu/cts_retreat/2016/program/15 Creative Commons License This work is licensed under a Creative Commons Attribution-Noncommercial-Share Alike 3.0 License. This material is brought to you by eScholarship@UMMS. It has been accepted for inclusion in UMass Center for Clinical and Translational Science Research Retreat by an authorized administrator of eScholarship@UMMS. For more information, please contact Lisa.Palmer@umassmed.edu. VOYAGER THERAPEUTICS  - A SPINOUT FROM UMASS  GENE THERAPY AND RNAI TECHNOLOGIES   Guangping Gao, PhD CCTS Mini Symposium  May 20, 2016  GENE THERAPY STRATEGIES: AN OVERVIEW DISCLOSURE 1. G. Gao is a cofounder of Voyager Therapeutics, a biopharmaceutical company and holds equity in the company.    2. G. Gao is an inventor on patents with potential royalties licensed to Voyager and other biopharmaceutical companies.  GENE THERAPY STRATEGIES: AN OVERVIEW Cost: $3 Billions GT GT drug development: - starts with a specific therapeutic agent - skips the drug discovery phase Cost: $ 0.6 Billion DRUG DEVELOPMENT  - CONVENTIONAL VERSUS GENE TX  First human gene therapy for ADA Via retrovirus  (Sept 14,1990) First lentivirus  against HIV (2003) Clinical trial for X-linked ALD (2014) CAR-T-cell therapy (2011) Jesse Gelsinger  (Sept 17,1999) First adenovirus for cystic fibrosis (1993) First AAV for CFTR(November 1995) Discovery of AAV8 (2002) Glybera approved by EMA (2012) AAV8 to treat hemophilia B (2011) AAV2 gene transfer to treat LCA (2008) Gao G entered  The field (1994) PROGRESS IN GENE THERAPY - DRIVEN BY VECTOR PLATFORM DEVELOPMENT Gene Therapy  -The path to today & beyond……  More Gene Tx companies founded and went public  More and more investment $ into Gene Tx Ye ar  2 01 3Ye ar  2 01 4Q1 , 20 15Ye ar  2 01 5 Pr oj ec ti on051 01 5GT Investment $ in billionCOMMERCIALIZATION OF GENE THERAPY  - RAPID TRANSFORMATION IN THE PAST FEW YEARS  AAV companies go to IPO fast r & faster   More than 200 gene therapy companies globally WHY UMMS? - A WORLD LEADER IN RNAI, DEGENERATIVE NEUROLOGICAL  DISEASE RESEARCH & AAV GENE THERAPY • Our faculty have made key advances – World leading scientists in RNAi research (Mello, Ambros, & Zamore) – World leading physician scientists in neurodegenerative disorders   Brown for Amyotrophic Lateral Sclerosis  Aronin for Huntington Disease – World leading rAAV gene therapy scientists   First to discover novel primate AAVs for efficient and stable gene transfer (Gao, 2002)  First to use AAV gene therapy in patients with 4 INDs (Flotte 1995) • Horae Gene Therapy Center – A >12,000 Ft2 state-of-art research facility on the 6th floor of  Albert Sherman Building – Research home for 15 faculty and affiliated faculty members, > 60 trainees and staff    IMPORTANT MILESTONES IN  FOUNDING & GROWING OF VOYAGER   | | | | | | 2009 2012 2013 2014 2015 2016 Voyager  IPO  $225 M cash Initially approached by  3rd Rock Venture Re- engagement  by 3rd Rock Venture Continued discussions w/t 3RV on “AAVian” Voyager signed license agreement  with UMMS for RNAi and rAAV technology platforms $45 M series A funding by 3RV & formally launching of Voyager   $845 M committed  by Sanofi/Genzyme for strategic collaboration w/t Voyager in CNS gene therapy   Voyager secured $60 M series B funding from  “Crossover” investors Company Founders 10 © Voyager Therapeutics  Voyager was founded by world leaders in the fields of AAV gene therapy, expressed RNA interference and neuroscience.  Krystof Bankiewicz, M.D., Ph.D. Kinetics Foundation Chair in Translational Research and Professor in Residence of Neurological Surgery and Neurology, University of California at San Francisco  Guangping Gao, Ph.D. Director, University of Massachusetts Medical School (UMMS) Gene Therapy Center & Vector Core; Scientific Director, UMMS-China Program Office; Professor of Molecular Genetics and Microbiology, UMMS  Mark Kay, M.D., Ph.D. Dennis Farrey Family Professor, Head, Division of Human Gene Therapy, Departments of Pediatrics and Genetics, Stanford University School of Medicine  Phillip Zamore, Ph.D. Howard Hughes Medical Institute Investigator; Gretchen Stone Cook Chair of Biomedical Sciences, Professor of Biochemistry and Molecular Pharmacology, and Co-Director of the RNA Therapeutics Institute, University of Massachusetts Medical School (UMMS) Company Highlights 11 © Voyager Therapeutics Robust product engine to engineer, optimize, manufacture and deliver AAV gene therapies Pipeline of five programs for severe CNS diseases Lead program, VY-AADC01, for advanced Parkinson’s disease with human POC expected in H2:2016 Strategic collaboration with Genzyme — gene therapy “know-how” Strong financial position with ~$225 million of cash following IPO in November 2015, no debt Management team and scientific founders that have pioneered significant advances in AAV gene therapy and neuroscience, and have extensive CNS drug development expertise (Steve Paul, MD, CEO)  Treating Severe CNS Diseases with AAV Gene Therapy • Significant unmet medical need • Genetically-validated targets • Targeted delivery to regions of the brain & broader delivery to the spinal cord is achievable  • Durable transgene expression as CNS cells are terminally differentiated • Immune-privileged site Why CNS? • Ability to target a variety of tissue & cell types within the CNS • >1,300 patients (200 in CNS) treated, no AAV-related SAEs to date • AAV does not readily integrate into the target cell genome, reducing potential for oncogenesis • Ability to manufacture at commercial quality and scale Why AAV? 12 © Voyager Therapeutics Product Engine Driving Pipeline of AAV Gene Therapies 13 © Voyager Therapeutics Product Engine Pipeline of 5 Programs VY-AADC01 Advanced Parkinson’s  Disease VY-SOD101 Monogenic Form of ALS VY-FXN01 Friedreich’s Ataxia VY-HTT01 Huntington’s Disease VY-SMN101 Spinal Muscular Atrophy U.S. Ex-U.S.  (option to worldwide) (option to  co-U.S.) Genzyme (option) Genzyme Genzyme (option) Genzyme (option) Genzyme Phase 1 Preclinical Commercial Rights Program Process R&D & Manufacturing Vector  Engineering  & Optimization Dosing &  Delivery Techniques ® ® ® ® • Process R&D center at Voyager’s headquarters • Research grade baculovirus / Sf9 production system • Up to 250L bioreactor capacity  • Proprietary reagents for new capsids and constructs • Collaboration with MassBiologics • cGMP baculovirus / Sf9 production system • Up to 1,000L bioreactor capacity  • Voyager retains IP and key process know-how Commercial Scale AAV Manufacturing Capabilities • Collaboration with UMass Medical School • Research grade baculovirus / Sf9 production system • Up to 500L bioreactor capacity Process R&D Large Scale Research Capacity Commercial Scale cGMP Capacity 14 © Voyager Therapeutics Robust Strategic Collaboration with UMMS    © Voyager Therapeutics 15 • Pilot grant program focused on understanding & optimizing AAV vectors for therapeutic use  • Licenses and sponsored research focused on novel AAV technology • Opportunities for UMMS participation in Voyager clinical trials • Postdoctoral training program • Sponsorship of annual lecture series on AAV-mediated gene therapy • Opportunities for Voyager to support graduate fellowships in the area of central nervous system AAV gene therapy • AAV vector supply from the UMMS Gene Therapy Vector Core to support Voyager research projects • Partnership with MassBiologics to  advance AAV process development & GMP production for Voyager product programs Research Collaboration Production & Manufacturing Educational  Support Voyager and UMMS collaborate under a broad strategic partnership to advance AAV gene therapy research, manufacturing and education 

Voyager Therapeutics - A Spinout from UMass Gene Therapy and RNAi Technologies

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Voyager Therapeutics - A Spinout from UMass Gene Therapy and RNAi Technologies

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