firstwordpharmaMay 25, 2021
Tag: Decibel Therapeutics , gene therapy , molecular genetics
Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, today announced the appointment of leading experts in gene therapy, cochlear development and molecular genetics to its Scientific Advisory Board (SAB).
"These additions to our SAB bring a deep understanding of the genetic roots of hearing loss and balance disorders, and we look forward to working closely with them as we progress our pipeline of gene therapies and regenerative medicines," said Joe Burns, Ph.D., Vice President, Discovery.
"We value their insights and tremendous experience in the translational considerations for the development of gene therapies as Decibel works to address the vast unmet needs in hearing and balance disorders," said John Lee, Chief Development Officer.
Connie Cepko, Ph.D., is the Bullard Professor of Genetics and Neuroscience at Harvard Medical School and a Howard Hughes Medical Institute Investigator. She trained in virology with Dr. Phillip Sharp at MIT for a Ph.D., and later with Dr. Richard Mulligan at the MIT Whitehead Institute. She helped develop retroviral vectors for transduction into the central nervous system (CNS) for lineage analysis and for studies of gene function in vivo. Her laboratory has focused on the mechanisms of cell fate determination in the CNS with focus on retina through the analysis of progenitor and stem cells. More recently, she has been studying the mechanisms of photoreceptor death in diseases that cause blindness, such as retinitis pigmentosa and macular degeneration, and is developing gene therapies to avert photoreceptor death in order to prevent vision loss.
Guangping Gao, Ph.D.,is an internationally recognized researcher who played a key role in the discovery and characterization of a new family of adeno-associated virus (AAV) serotypes to advance the gene therapy field. He has published extensively in the field, with more than 300 papers, and holds more than 191 patents, with hundreds more pending. The Penelope Booth Rockwell Professor in Biomedical Research at the University of Massachusetts Medical School, Dr. Gao is an elected fellow of both the U.S. National Academy of Inventors and the American Academy of Microbiology. He is the Past President of the American Society of Gene and Cell Therapy. Dr. Gao co-founded Voyager Therapeutics, Adrenas Therapeutics and Aspa Therapeutics to develop AAV-based gene therapies for rare diseases.
Matthew Kelley, Ph.D., directs the Laboratory of Cochlear Development in the Intramural Program at the National Institute on Deafness and Other Communication Disorders, National Institutes of Health. A widely published and well-respected research scientist, he focuses on the cellular and molecular development of the mammalian cochlea. Dr. Kelley has long been an active member and is the past President of the Association for Research in Otolaryngology (ARO).
Glenn Pierce, M.D., Ph.D.,is Entrepreneur-in-Residence at Third Rock Ventures. He has 30 years of experience in drug discovery and development—with a particular focus on tissue regeneration, gene therapy and hematology—and has contributed to the development of six marketed products.As the former Chief Medical Officer, Hemophilia Therapeutic Area at Biogen, he led work culminating in multiple regulatory approvals for hemophilia therapeutics. Dr. Pierce has served in multiple leadership roles for the National Hemophilia Foundation as well as on advisory boards for the U.S. Food and Drug Administration and the U.S. Department of Health and Human Services. He has co-authored more than 150 scientific papers and has received more than 15 patents. Dr. Pierce serves on the Boards of Directors of Global Blood Therapeutics and Voyager Therapeutics.
Dinah Sah, Ph.D., is an accomplished drug developer and R&D leader with over 25 years of experience in research and drug development in the biotechnology industry. Most recently, she was Chief Scientific Officer at Voyager Therapeutics, joining soon after its start in 2014. Prior to Voyager, Dr. Sah was Vice President of Research at Alnylam, overseeing many of the research programs during her seven-year tenure. She has successfully led multiple research and preclinical programs toward and into clinical development across new modalities, including the CNS-focused AAV programs at Voyager and the groundbreaking novel class of RNAi therapeutics developed at Alnylam.
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