Overview          Leadership          Board of Directors           Scientific Advisors           Investors

Scientific Advisors

Vineeta Agarwala, MD, PhD

Venture Partner, GV
Off-target Assessment

Joy Cavagnaro, PhD, DABT, RAC

President, Access BIO
Regulatory Affairs

Marie E. Egan, MD

Professor of Pediatrics and of Cellular And Molecular Physiology, Yale University

Cystic Fibrosis

Peter Glazer, MD, PhD

Robert E. Hunter Professor of Therapeutic Radiology and Professor of Genetics, Yale University

Triplex Gene Editing

George Kemble, PhD

CEO, 3-V Biosciences
Research and Development

Harry Lam, PhD

EVP Technical Operations, JW Therapeutics
Drug Product Development

 Mike Placke, PhD, DABT

Global Head of Nonclinical Development and US Research, Shire

Pre-clinical Development

W. Mark Saltzman, PhD

Goizueta Foundation Professor of Biomedical Engineering, Chemical & Environmental Engineering & Physiology, Yale University

Formulation Development

Jack Szostak, PhD

Professor of Genetics, Harvard Medical School

Genetics

Elliott Vichinsky, MD

Director of Hematology/Oncology, UCSF Benioff Children's Hospital Oakland

Clinical hematology, Sickle cell disease, Translational Science

Mark Walters, MD

Director, Blood and Marrow Transplantation Program 
Division of Hematology/Oncology, UCSF Benioff Children's Hospital Oakland 

Clinical hematology, Sickle cell disease, Translational Science 

 
Vineeta Agarwala, MD, PhD

Off-target Assessment

 

Vineeta Agarwala, MD, PhD is a Venture Partner at GV (Google Ventures). Dr. Agarwala has been engaged in multiple projects at the intersection between medicine and genomic data, including contributing to the development of a bioinformatic tool to predict genome-wide off-target sites for CRISPR/Cas9 at the Broad Institute and leading the development of national-scale real-world databases that integrate clinical and genomic data for oncology drug development and research at Flatiron Health. Previously, she has been a computational researcher at Cold Spring Harbor Laboratory and Lawrence Livermore National Laboratory, and a management consultant for biotech and pharmaceutical clients at McKinsey & Company in New York. She holds a BS in biophysics from Stanford University, and earned MD/PhD degrees from Harvard Medical School and MIT.

Joy Cavagnaro, PhD, DABT, RAC

Regulatory Affairs

 

Joy Cavagnaro, PhD is President of Access BIO. She consults on development strategies and therapeutic safety for novel drug, biologics and device combinations. Her career spans the CRO and biotechnology industries, academia and government. Formerly, she served at FDA/CBER, where she was appointed to the Senior Biomedical Research Service, served as FDA's topic lead for safety for the ICH initiative for seven years, and was rapporteur for the ICH S6 Guidance on Preclinical Safety Evaluation of Biotechnology-derived Pharmaceuticals. Currently, she is past Chair of the Clinical and Regulatory Affairs Committee and past member of the Translational Science & Product Development Committee of the American Society for Gene and Cell Therapy, is an advisor and member of the Grants Working Group of the California Institute of Regenerative Medicine, and Chair of CRRI, an independent IRB. Prior to joining FDA, Dr. Cavagnaro was principal study director for biotechnology products at Covance Inc. Dr. Cavagnaro is Past Chair of Regulatory Affairs Professional Society and the National Capital Area Chapter of Society of Toxicology. She serves on multiple SAB’s and consults and lectures internationally on translation and risk assessment of novel therapies. She has co-authored numerous white papers and chapters related to various aspects of preclinical safety assessment. The book she edited “Preclinical Safety Evaluation of Biopharmaceuticals A Science-Based Approach to Facilitating Clinical Trials”. She holds a BS in biology from the University of Miami, and earned her PhD degree from the University of North Carolina, Chapel Hill.

Marie E. Egan, MD

Cystic Fibrosis

 

Marie Egan, MD is Professor of Pediatrics (Respiratory Medicine) and of Cellular and Molecular Physiology at Yale University, and is Director of the Cystic Fibrosis Center, Interim Division Chief of Respiratory Medicine Allergy Immunology and Sleep Medicine and Vice Chair for Research in the Department of Pediatrics. Dr. Egan’s primary research interest is to understand the regulation of ion transport across the airway epithelia in health and disease. Dr. Egan has pioneered the use of triplex gene editing in multiple cell and animal models of cystic fibrosis for both the dominant CFTR mutation, F508del, as well as other mutations observed at lower population frequency. Dr. Egan currently serves on the Drug Monitoring Safety Board at the CF Foundation, and multiple study and review boards at Yale, ATS and NIH. Dr. Egan earned her BA from the University of Pennsylvania and MD at Mt. Sinai and trained in Pediatrics and Pediatric Pulmonary at the Johns Hopkins Hospital.

Peter Glazer, MD, PhD

Triplex Gene Editing

 

Peter Glazer, MD, PhD is Professor and Chair of Therapeutic Radiology and Professor of Genetics at Yale School of Medicine, and also serves as Chief of Radiation Oncology at Yale-New Haven Hospital. Dr. Glazer’s research career has focused on gene targeting and gene therapy, genetic instability in cancer, DNA repair, and cancer therapy. Over the past decade, he has pioneered the design and use of peptide nucleic acids for triplex-mediated gene editing. Dr. Glazer holds his BA from Harvard, MS from Oxford, and MD/PhD from Yale.

George Kemble, PhD

Research and Development

 

George Kemble, PhD is Chief Executive Officer at 3-V Biosciences, where he was previously Chief Scientific Officer, responsible for directing the medicinal chemistry, research, translational and CMC groups. 3-V’s programs range from anti-infectives, oncology to liver diseases. Working closely with external collaborators, Dr. Kemble’s teams have filed 2 INDs and progressed a lead candidate to Ph2 trials in NASH. Prior to joining 3-V, Dr. Kemble was with Medimmune, Inc., a subsidiary of Astra-Zeneca, PLC where he served as Senior Vice President of R&D and Head of Research. During his tenure, Dr. Kemble was responsible for a large group of scientists dedicated to research and development of programs across a number of therapeutic areas, including the successful launch of FluMist. During his career, he has lead programs utilizing multiple technologies including small molecules, large molecules and vaccines from research into the clinic. Dr. Kemble received his BS from the University of Santa Clara and a PhD from Stanford University and did his post-doctoral training at UCSF.

Harry Lam, PhD

Drug Product Development

 

Harry Lam, PhD is Director of Technical Operations, Therapeutics Division, at 23&Me and oversees all aspects of process development, manufacturing, quality control and supply chain. Prior to joining 23&Me, he was at Affinita Biotech where he was Chief Technology Officer and Vice President of CMC Development. Previously, he was VP, Head of Biologics Manufacturing at Sanofi, and before that VP, Manufacturing and Process Development, at Shire Regenerative Medicine. Harry has over 30 years of experience in biopharmaceutical process development and manufacturing, including 17 years at Genentech, where he held a number of positions; including, Director of Manufacturing & Technology, Genentech Singapore Pte Ltd; Senior Director, Global Biologics Drug Substance Manufacturing Science & Technology; and ultimately Roche Global Head of Contract Manufacturing Operations, Commercial Drug Substance. Prior to joining Genentech, Harry spent 11 years at Pfizer in the Bioprocess R&D department. He received his BS in Chemical Engineering from the University of Birmingham, UK and his PhD in Chemical Engineering from Rensselaer Polytechnic Institute.

Mike Placke, PhD, DABT

Pre-clinical Development

 

Mike Placke, PhD is Global Head of Nonclinical Development and US Research at Shire. He has over 30 years of experience in pharmaceutical drug development, including scientific and operational responsibilities for multi-disciplinary pharmaceutical development operations, including Drug Safety and Metabolism (DSM) and Chemical Product Development (CPD) Divisions in large pharmaceutical and biopharmaceutical companies. Prior to his current role at Shire, he was Senior Vice President of Nonclinical Development at Alnylam Pharmaceuticals. Before joining Alnylam, he was President & CEO of Ricerca Biosciences, a privately held contract research company, providing integrated non-clinical drug development services to the biopharmaceutical industry. Prior to Ricerca, Dr. Placke was VP, Drug Safety at Wyeth Pharmaceuticals (and Pfizer post-acquisition). He has experience in developing a wide array of drug product modalities; including, small molecules, biologics, siRNA therapies, gene therapy, vaccines, and specialty drug products. He is formally trained in experimental pathology and toxicology, earning BS and MS degrees from The Ohio State University and his PhD from the University of Connecticut. He is board certified in general toxicology and was a director and officer of the American Board of Toxicology.

W. Mark Saltzman, PhD

Formulation Development

 

W. Mark Saltzman, PhD is Goizueta Foundation Professor of Chemical and Biomedical Engineering at Yale University, where he was the Founding Chair of the Biomedical Engineering Department. He has pioneered formulations development for the delivery of PNAs for triplex gene editing. Dr. Saltzman’s research has broadly impacted the fields of drug delivery, biomaterials, nanobiotechnology, and tissue engineering, and he is the recipient of multiple awards and honors, including election to the US National Academy of Medicine and the US National Academy of Engineering. Prior to Yale, he has been professor of chemical engineering at Johns Hopkins University and Cornell University. Dr. Saltzman holds a BS in Chemical Engineering from Iowa State University and PhD in Medical Engineering from Massachusetts Institute of Technology.

Jack Szostak, PhD

Genetics

Jack Szostak, PhD is an Investigator of the Howard Hughes Medical Institute, professor of genetics at Harvard Medical School, and the Alex Rich Distinguished Investigator in the Department of Molecular Biology and the Center for Computational and Integrative Biology at Massachusetts General Hospital. Dr. Szostak’s early research on telomere structure and function, and the role of telomere maintenance in preventing cellular senescence, was recognized by the 2006 Albert Lasker Basic Medical Research Award and in 2009 the Nobel Prize in Physiology or Medicine, shared with Drs. Elizabeth Blackburn and Carol Greider. He served as an early advisor to Genetics Institute and Gilead Sciences, Inc, and is a co-founder and Chair of the SAB of Ra Pharmaceuticals, a public company. He is a member of the SAB of Elysium Health, Chair of the SAB of Moderna Therapeutics, and an advisor to ORI Capital, Hong Kong. In the 1990s Dr. Szostak and his colleagues developed in vitro selection as a tool for the isolation of functional RNA, DNA, and protein molecules from large pools of random sequences. Dr. Szostak’s current research interests are in the laboratory synthesis of self-replicating systems and the origin of life. Dr. Szostak is a member of the National Academy of Sciences and a fellow of the American Academy of Arts and Sciences and the American Association for the Advancement of Science. He holds a BS in cell biology from McGill University, and earned his PhD degree at Cornell University.

Elliott Vichinsky, MD

Clinical hematology, Sickle cell disease, Translational Science

Elliott Vichinsky, MD is Division Chief, Hematology/Oncology, at UCSF Benioff Children’s Hospital Oakland. Dr. Vichinsky diagnoses and treats children with blood disorders, cancers and tumors. He founded and directs the largest hemoglobinopathy center of sickle cell disease and thalassemia in North America. He has been instrumental in implementing newborn screening programs for blood diseases in California and throughout the world. He is working with the World Health Organization in studying the worldwide public health problem of thalassemia and sickle cell disease. Dr. Vichinsky was responsible for the development of the bone and marrow transplantation program, which has cured hundreds of children who had sickle cell anemia, thalassemia, and various cancers. He has developed techniques that make blood safer for chronically transfused patients, and is a leader in investigating novel drugs that turn on hemoglobin-producing genes which enable sickle cell disease and thalassemia patients to produce their own healthy blood. He has authored and co-authored more than 300 articles in peer-reviewed journals, four books, and is Editor-in-Chief of the journal Pediatric Hematology/ Oncology. He has been the principal investigator on more than 40 major research projects. Dr. Vichinsky earned his MD at SUNY Downstate and trained in Pediatrics at University Hospital/Children’s Orthopedic Hospital and Medical Center, Washington.

Mark Walters, MD

Clinical hematology, Sickle cell disease, Translational Science
 

Mark C. Walters, MD, is the Jordan Family Director of the Blood and Marrow Transplantation Program at UCSF Benioff Children’s Hospital, Oakland and Professor of Pediatrics at UCSF. He is Program Director of the CIRM alpha stem cell clinic at UCSF. Dr. Walters received his A.B. with honors in Genetics from the University of California, Berkeley and his MD from the University of California, San Diego. He completed pediatric residency training at the University of Washington and hematology/oncology fellowship training at the University of Washington and the Fred Hutchinson Cancer Research Center in Seattle. He was a junior faculty member in Seattle before matriculating to Oakland in 1999. He has been active in cooperative clinical transplantation trials and has led several NIH-supported investigations of hematopoietic cell transplantation for sickle cell anemia and thalassemia. He has authored or co-authored many publications with a focus on hematopoietic cell transplantation for hemoglobin disorders, and he has a research interest in extending transplantation to young adults with hemoglobin disorders and other novel cellular therapies for hemoglobin disorders. Currently, research interests are focused on genomic editing and gene addition therapies as a strategy to extend curative therapy in all patients who inherit a clinically significant hemoglobinopathy.

 
 
 
 
 
 
 
 
 
 
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