Exploring Novel Clinical Trial Designs for Gene-Based Therapies Proceedings of a Workshop (2020) / Chapter Skim
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Appendix B: Speaker Biographical Sketches
Appendix B: Speaker Biographical Sketches
Pages 89-98
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From page 89... ...
Mr. Bartek's professional experience also includes 20 years of federal executive branch and legislative branch service in defense, foreign policy, and intelligence, including 6 years on the policy staff of the House Armed Services Committee; 4 years at the Department of State's Bureau of Politico-Military Affairs, including 1 year as a negotiator on the U.S.
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From page 90... ...
His clinical practice and research interests have focused on pediatric neuromuscular disorders, especially spinal muscular atrophy, Duchenne muscular dystrophy, inherited neuropathies, and neurometabolic
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From page 91... ...
Dr. Finkel has participated in numerous clinical trials, natural history studies, and the development of standard-of-care guidelines, and he has contributed to the development of outcome measures, clinical trial design, and biomarker identification for neuromuscular disorders.
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From page 92... ...
Dr. Kanter works on the development of novel therapeutics in sickle cell disease with expertise in clinical trial recruitment and trial design as well as in areas of improving access to care.
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From page 93... ...
. He has sat on various advisory bodies within the National Heart, Lung, and Blood Institute and the National Institute of Allergy and Infectious Diseases, served for four National Academies of Sciences, Engineering, and Medicine committees, and chaired the International Society of Stem Cell Research Guidelines for Stem Cell Research and Clinical Translation revision task force (2015–2016)
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From page 94... ...
In January 2019 he initiated a clinical trial of gene therapy for Pompe disease. His laboratory is developing gene editing with CRISPR for the glycogen storage diseases.
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From page 95... ...
Results from the clinical trials that he directed led to the first approved gene therapy drug for genetic disease worldwide and the first Food and Drug Administration–approved recombinant virus-based gene therapy product for a genetic disease.
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From page 96... ...
She is also working on clinical trials of lentivirus mediated gene therapy for X-linked SCID and Artemis-deficient SCID, the latter being a first-in-human trial. Tejashri Purohit-Sheth, M.D., is currently the director of the Division of Clinical Evaluation and Pharmacology/Toxicology in the Office of Tissues and Advanced Therapies (OTAT)
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From page 97... ...
Within the past year she has shared her story on several panels concerning sickle cell disease and clinical trials such as Rare Disease Day at NIH, the Howard University Cure Sickle Cell Symposium, and the Rare Disease Legislative Advocates congressional caucus briefing. Most recently she spoke to the House subcommittee on labor, health and human services, education, and related agencies.
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From page 98... ...
His group focuses on developing curative strategies for sickle cell disease through transplantation of allogeneic or genetically modified autologous bone marrow stem cells.
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