(2022) Day 1: Pediatric Gene Therapy: State of the Field and Future Directions скачать в хорошем качестве

(2022) Day 1: Pediatric Gene Therapy: State of the Field and Future Directions

Moderator: Dr. Alison Bateman-House, PhD, MPH, MA Title: Assistant Professor, Department of Population Health, NYU Division of Medical Ethics, Grossman School of Medicine; Co-chair, PGTME BIO: Dr. Bateman-House is an assistant professor in the Department of Population Health at NYU Langone. Within the department’s Division of Medical Ethics, she co-chairs its Working Group on Compassionate Use and Preapproval Access (CUPA), whose members study ethical issues concerning access to investigational medical products. Dr. Bateman-House also co-chairs, with Lesha Shah MD, the Pediatric Gene Therapy and Medical Ethics (PGTME) working group, which includes academics, patient advocates, biopharmaceutical industry representatives, and a wide array of clinical and research professionals. Panelists: Dr. Terence R. Flotte, MD Title: Provost and Executive Deputy Chancellor; Dean; Celia and Isaac Haidak Professor and Professor of Pediatrics, University of Massachusetts T.H. Chan School of Medicine BIO: Dr. Flotte currently is the Provost/Dean and Celia and Isaac Haidak Professor at University of Massachusetts Chan Medical School, with faculty appointments in the Department of Pediatrics and the Horae Gene Therapy Center. Dr. Flotte is a pediatric pulmonologist and has been the principal investigator of multiple clinical gene therapy trials, including the first-ever human use of AAV vectors, a trial in cystic fibrosis patients that began in 1995, and recent trials in alpha-1 antitrypsin deficiency and Tay-Sachs disease. His research has been continuously NIH funded since 1993. Since 2015, he has also been Editor-in-Chief of his field’s oldest journal family, Human Gene Therapy. Dr. Louise Rodino-Klapac, PhD Title: Head of R&D, Chief Scientific Officer, Sarepta Therapeutics BIO: Dr. Klapac joined Sarepta in June 2018 and was appointed executive vice president, chief scientific officer in December 2020. Prior to this role, she served as Sarepta’s senior vice president of gene therapy. She became head of R&D in November 2021. She has led the design of most of Sarepta’s late-stage gene therapy candidates, has built and led Sarepta’s Genetic Therapies Center of Excellence (GTCOE) in Columbus, Ohio, and has oversight for Sarepta’s Gene Editing Innovation Center (GEIC) in Durham, N.C. Dr. Klapac is a current Board member of the Alliance for Regenerative Medicine, as well as a member of the American Society for Gene and Cell Therapy, and the American Academy of Neurology. Dr. Phillip J. Brooks, PhD Title: Acting Director, Division of Rare Diseases Research Innovation, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health BIO: Dr. Philip J. (P.J.) Brooks is the acting director of NCATS’ Division of Rare Diseases Research Innovation. Brooks represents NCATS in the NIH-wide Gene Therapy Working Group, the Regenerative Medicine Innovation Project, and the International Rare Diseases Research Consortium (IRDiRC). He also is the working group co-coordinator for the NIH Common Fund program on Somatic Cell Genome Editing, one of the leaders of the Platform Vector Gene Therapy (PaVe-GT) pilot project, and the co-chair of the Bespoke Gene Therapy Consortium. Brooks received his doctorate in neurobiology from The University of North Carolina at Chapel Hill. After completing a postdoctoral fellowship at The Rockefeller University, he became an investigator in the NIH intramural program, where he developed an internationally recognized research program focused on two distinct areas: the molecular basis of alcohol-related cancer and rare neurologic diseases resulting from defective DNA repair, including xeroderma pigmentosum, Cockayne syndrome, and Fanconi anemia.