Session Abstract – PMWC 2023 Silicon Valley


Track 1, January 25

Track Co-Chairs:
Yael Weiss, Mahzi
Peter Marks, FDA

  • Welcome and Opening by Track Chairs: Yael Weiss, Mahzi & Peter Marks, FDA
  • Patient Driven Therapies: The Challenges and Successes (PANEL)
    Chair: Yael Weiss, Mahzi
    - Allyson Berent, Angelman Foundation
    - Terry Pirovolakis, CureSPG50
  • From Bench to Bedside - Biomarkers, Natural History Studies, Animal Models (PANEL)
    Chair: Peter Marks, FDA
    - Emil Kakkis, Ultragenyx
    - Alysson Muotri, UCSD
    - Nasha Fitter, Invitae
  • Commercialization - Moving from Rare to Common (PANEL)
    Chair: John Maraganore, Alnylam Pharmaceuticals
    - Shankar Ramaswamy, Kriya
    - Rahul Singhvi, Resilience
    - Andrew Lo, MIT
    - Mark Harrison, University of British Colombia
  • Future Modalities (PANEL)
    Chair: Shannon Muir, CIAPM
    - Nadav Ahituv, UCSF
  • Bioethics & Ethical Implications in Cell and Gene Therapies
    - Hank Greely, Stanford

 Session Chair Profile

M.D., Ph.D., Director, CBER, US FDA

Biography
Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching and caring for patients and in industry on drug development. He joined the FDA in 2012 as Deputy Center Director for CBER and became Center Director in 2016.

Talk
Facilitating development of gene therapy


 Session Chair Profile

Ph.D., Co-Director of Precision Medicine, CA Governor's Office

Biography
Dr. Muir co-directs the California Initiative to Advance Precision Medicine and heads the Initiative’s efforts to address cancer disparities and depression. She previously served as the Director of Research Proposal Development Services at the UC San Diego Office of Research Affairs, and as a Senior Program Associate and Science and Technology Policy Fellow in the Senate Health Committee for the California Council on Science and Technology.

Talk
Session: Participant-centered cell therapy
Session Description: Clinical trial enrollment for cell-based therapies can be hampered by logistical, financial, social, and cultural factors. Designing trials and developing targeted outreach strategies with participants at the center can increase enrollment and retention for research into cell-based therapies.


 Session Chair Profile

PHD, Advisory Board, Alnylam Scientific

Biography
Dr. John Maraganore served as the founding CEO and a Director of Alnylam from 2002 to 2021. He continues to serve on the Alnylam Scientific Advisory Board. Prior to Alnylam, he was at Millennium Pharmaceuticals, Inc., before Millennium, he held scientific and business roles at Biogen, Inc. Previously, he was a scientist at ZymoGenetics, Inc. and the Upjohn Company. He is currently a Venture Partner at ARCH Venture Partners, a Venture Advisor at Atlas Ventures, an Executive Partner at RTW Investments, and Senior Advisor for Blackstone Life Sciences. He is also member of the Board of Directors for Agios Pharmaceuticals, Beam Therapeutics, Kymera Therapeutics, ProKidney Corp., Takeda Pharmaceuticals, Aerium, Hemab, Aera, and Versanis, and the Biotechnology Innovation Organization. In addition, he serves on the Board of the Termeer Foundation and as Chair of the n-Lorem Foundation Advisory Council on the Advisory Board of Ariadne Labs and as an advisor to Nucleate.


 Session Chair Profile

MD, PHD, CEO, Mahzi Therapeutics

Biography
Yael Weiss Is currently CEO of Mahzi Therapeutics, a company focused on the development of therapies for ultra-rare genetic neurodevelopmental disorders. Mahzi works closely with patient foundations to support their journey towards drug development and bring programs into Mahzi once pre-clinical proof of concept is established. Yael completed her MD at Hadassah Medical School at the Hebrew University in Jerusalem and her PhD at the Weizmann Institute of Science in Rehovot, Israel. She has over 20 years of industry experience in medical/clinical and business development roles at Genzyme, Merck and Ultragenyx. Yael is a member of the NIH driven Bespoke Gene Therapy (BCTG) consortium, ASGCT translational committee, N=1 collaborative and is a 2022 Termeer Fellow. Board member/advisor to ADNP and FOXG1 foundations.


 Speaker Profile

JD, Professor of Law; Professor by couretsy of Genetics, Stanford University

Biography
Henry T. (Hank) Greely specializes in ethical, legal, and social issues arising from the biosciences. He is a founder and a past President of the International Neuroethics Society; chairs the California Advisory Committee on Human Stem Cell Research; chairs the Ethical, Legal, and Social Issues Committee of the Earth BioGenome Project; and serves on the NIH BRAIN Initiative’s Multi-Council Working Group while co-chairing the Initiative’s Neuroethics Work Group. He is the author of THE END OF SEX AND THE FUTURE OF HUMAN REPRODUCTION (Harv. Univ. Press 2016) and CRISPR PEOPLE: THE SCIENCE AND ETHICS OF EDITING HUMANS (MIT Press 2021). Greely graduated from Stanford in 1974 and Yale Law School in 1977. He clerked for Judge John Minor Wisdom of the Fifth Circuit Court of Appeals and Justice Potter Stewart, then served in the Departments of Defense and Energy in the Carter Administration. He joined the Stanford faculty in 1985.

Talk
Bioethics & Ethical Implications in Cell and Gene Therapies


 Speaker Profile

Ph.D., Associate Professor, UBC

Biography
Mark Harrison is an Associate Professor with the University of British Columbia’s Faculty of Pharmaceutical Sciences and leads a health economics program within the Collaboration for Outcomes Research and Evaluation (CORE) group. His program of research considers patient-physician decision-making, understanding the role of preferences in treatment decisions, and evaluating the impact of policy interventions on patient and health care system outcomes. Increasingly Mark is looking to study the access to health care of marginalized populations and the relationship between health/health care and the environment.


 Speaker Profile

Sc.D., Co-founder & CEO, Resilience

Biography
Rahul is a global leader in the Life Sciences industry. Most recently, he was an Operating Partner at Flagship Pioneering, where he was responsible for founding and operating companies launched from Flagship’s innovation foundry, Flagship labs. Before joining Flagship, Rahul was the Chief Operating Officer of Takeda’s Vaccine Business Unit where he was responsible for worldwide vaccine CMC and manufacturing operations. Before Takeda, Rahul was President and CEO of Novavax, Inc. where he transformed the company from a specialty pharmaceutical business to a vaccine development company. Rahul’s career began at Merck & Co in 1994, where he held several positions in R&D and manufacturing. Rahul serves on the Executive Advisory Board of the Leonard Davis Institute of Health Economics at the University of Pennsylvania and on the Scientific Advisory Board of the anti-microbial resistance research group at the Singapore MIT Advance Research and Technology program.


 Speaker Profile

DVM, DACVIM, Chief Science Officer, FAST, NPO

Biography
Dr. Berent is a veterinary internal medicine specialist/interventionalist who graduated from Cornell University and completed her residency at the University of Pennsylvania, where she served as an Adjunct Assistant Professor before joining the Animal Medical Center in NYC. She is the Director of Interventional Endoscopy, focusing on clinical trials researching medical devices particularly for ureteral and biliary obstructions. In 2014 Dr. Berent’s daughter, Quincy, was diagnosed with a rare neurogenetic disorder called Angelman syndrome. In 2015 she joined the Board of Directors for the Foundation for Angelman Syndrome Therapeutics (FAST), becoming the Chief Science Officer. Dr. Berent serves as the co-director of the Angelman Syndrome Biomarker and Outcome Measure Consortium. Dr. Berent co-founded GeneTx Biotherapeutics, a company focused on advancing an antisense oligonucleotide therapy, where she was the Chief Operating Officer. GeneTx was acquired in 2022 by Ultragenyx Pharmaceuticals, after launching the Phase 1/2 clinical trial.

Talk
The Parents' Journey Through Drug Development
After her little girl was diagnosed with AS Dr. Berent saw the impossible become possible. Through passion, scientific, and medical understanding, this incredible foundation (FAST) and a bold scientific team, advanced numerous treatment options for children with AS, paving the way for what so many other rare disease organizations are working so hard to do.


 Speaker Profile

Ph.D., Charles E. and Susan T. Harris Professor, MIT

Biography
Dr. Los healthcare-related research interests include: new financial engineering tools and business models for drug and device development and healthcare delivery; statistical methods for incorporating patient preferences into the drug approval process; predicting clinical trial outcomes via machine learning techniques; and novel funding and reimbursement models for creating a robust gene therapy ecosystem. He is a co-founder and director of BridgeBio Pharma, a director of AbCellera, Atomwise, and Roivant Sciences, a co-founder and chairman of QLS Advisors, and a member of the advisory boards of NCATS and the American Cancer Society’s BrightEdge Impact Fund.


 Speaker Profile

None of the above, Founder, CureSPG50

Biography
Terry Pirovolakis is the founder of CureSPG50, a charity that is actively working on building a cure for a neurodegenerative disease called Spastic Paraplegia Type 50 (SPG50). Terry started the charity after his youngest son Michael was diagnosed with the disease in 2019. Since then he has put together a team of scientific professionals from around the world that are working on a Gene Therapy for SPG50, which they hope will positively change the lives of children affected by this terrible disease. In addition to Terry’s charity work he is also a Director of Contact Centre Strategy at CIBC in Toronto Canada.

Talk
Journey To Cure Michael
I will take the audience through our families journey to cure my son Michael of an ultra-rare disease called SPG50.


 Speaker Profile

Ph.D., Professor, UC San Diego

Biography
Dr. Muotri earned a BSc in Biological Sciences from the State University of Campinas in 1995 and a Ph.D. in Genetics in 2001 from the University of Sao Paulo in Brazil. He moved to the Salk Institute as Pew Latin America Fellow in 2002 for a postdoctoral training in the fields of neuroscience and stem cell biology. He has been a Professor at the School of Medicine, University of California in San Diego since late 2008. His research focuses on modeling neurological diseases, such as Autism Spectrum Disorders, using human induced pluripotent stem cells and brain organoids. He has received several awards, including the prestigious NIH Director’s New Innovator Award, NARSAD, Emerald Foundation Young Investigator Award, Surugadai Award from Tokyo University, Rock Star of Innovation from CONNECT, NIH EUREKA Award, among others.


 Speaker Profile

MBA, Head, Data Platforms, Invitae

Biography
Nasha Fitter is a leader in the rare disease space through her work on utilizing real world evidence to accelerate treatments. She is also the mother of a child with the rare neurological condition, FOXG1 Syndrome, and co-founded and leads the FOXG1 Research Foundation. Nasha serves on the board for the ACMG Foundation for Genetic and Genomic Medicine and has an MBA from the Harvard Business School.


 Speaker Profile

M.D., Co-Founder, Chairman & CEO, Kriya Therapeutics

Biography
Dr. Ramaswamy was part of the early foundational team at Roivant Sciences, where he was responsible for helping to identify and evaluate assets across therapeutic areas and launch its first public subsidiary company. He went on to be the Chief Business Officer of Axovant Gene Therapies. Dr. Ramaswamy received his MD from Brown University and his undergraduate degree in economics from Harvard University.

Talk
Commercialization – moving from rare to common
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 Speaker Profile

Ph.D., Professor, UCSF

Biography
Nadav Ahituv is a Professor in the Department of Bioengineering and Therapeutic Sciences and the Interim Director of the Institute for Human Genetics at the University of California, San Francisco. He received his PhD in human genetics from Tel-Aviv University working on hereditary hearing loss. He then did his postdoc, specializing in functional genomics, in the Lawrence Berkeley National Laboratory and the DOE Joint Genome Institute. His current work is focused on identifying gene regulatory elements and linking nucleotide variation within them to various phenotypes including morphological differences between species, drug response and human disease. His lab was one of the co-developers of massively parallel reporter assays (MPRAs) that allow for high-throughput functional characterization of gene regulatory elements and pioneered the use of gene regulatory elements as therapeutic targets. Finally, the lab also utilizes short DNA sequences for diagnostic purposes.