DUX4 siRNA Optimization for the Development of an Antibody-Oligonucleotide Conjugate (AOC™) for the Treatment of FSHD
Optimization of AOC 1001, an antibody-oligonucleotide conjugate targeting the underlying cause of myotonic dystrophy type 1
DUX4 siRNA Optimization for the Development of an Antibody Oligonucleotide Conjugate (AOC™) for the Treatment of FSHD
Elizabeth joined Avidity in January 2020 and serves as SVP Clinical Development. Ms. Ackermann brings extensive experience in research and development of RNA therapeutics with a focus on neurology and rare diseases. Most recently Ms. Ackermann served as Vice President of Clinical Development at Otonomy Inc. and prior to Otonomy served as the Chief Science Officer at the Myotonic Dystrophy Foundation. Ms. Ackermann worked at Ionis Pharmaceuticals for over 8 years holding various leadership positions including Vice President of Clinical Development. At Ionis she led the development of Tegsedi® (an approved treatment for TTR Amyloidosis with Polyneuropathy) and several other rare disease programs. Ms. Ackermann holds a Ph.D in biochemistry from UC San Diego and completed a NIH postdoctoral fellowship in Pharmacology at UC San Diego.
In January 2021, Joe Johnston joined Avidity Biosciences as Senior Vice President, Regulatory Affairs and Quality. Mr. Johnston brings to Avidity over 30 years of pharmaceutical industry experience, including extensive experience in the development of novel oligonucleotide and small molecule therapies for neuromuscular, rare and pediatric diseases. Most recently, Mr. Johnston was Senior Vice President at Catabasis Pharmaceuticals in Boston where he advanced multiple programs including a pivotal program for the treatment of Duchenne Muscular Dystrophy. Prior to Catabasis, Mr. Johnston was at Ionis Pharmaceuticals (formerly Isis Pharmaceuticals), a biopharmaceutical company in RNA-targeted drug discovery and development, where he served as Vice President, Regulatory Affairs and Quality. At Ionis, Mr. Johnston held a series of roles with increasing responsibility starting from 1990, when he was part of the original scientific team.
Mr. Johnston holds a M.B.A. from University of California Irvine and a M.S. in Pharmacology and a B.A. in Biology, both from the University of Pennsylvania.
Sarah Boyce brings to Avidity extensive experience in the life sciences industry, where she has built global organizations and brought to patients a number of innovative therapies, including Tegsedi®, Waylivra®, Soliris®, Gleevec® and Tasigna®. She most recently served as President and a member of the board of directors of Akcea Therapeutics, where she led commercialization of the company’s rare disease products. Prior to joining Akcea in April 2018, Ms. Boyce held multiple executive-level roles at leading pharma and biotech companies including Ionis Pharmaceuticals, Forest Laboratories, Alexion Pharmaceuticals, Novartis Oncology and Roche.
Tamar Thompson is a distinguished health policy strategist, government affairs leader and market access executive with a diverse background across multiple healthcare sectors and therapeutic categories including rare disease, immunology, oncology and cardiovascular disease. She has more than twenty years of leadership experience in health care, including a focus on developing strategic and tactical recommendations to ensure optimal reimbursement and market access for rare disease products. Ms. Thompson currently serves as the Vice President, US Government Affairs and Policy for Alexion Pharmaceuticals, Inc. and as the Chair of the Board of Alexion’s Charitable Foundation. Prior to joining Alexion, Ms. Thompson served as head, federal executive branch strategy and state government affairs for Bristol-Myers Squibb Company. She also served as a strategic policy advisor and consultant for premiere Washington, DC based firms, including ADVI, Kimbell and Associates and Avalere Health. Ms. Thompson holds an M.S. in Health Sciences with a concentration in Public Health from Trident University in Cypress California.
Eric Mosbrooker has over 20 years of experience in the global commercial operations space with demonstrated leadership and experience in global pricing, marketing, distribution, product launches, and compliance. In addition to his commercial experience, Mr. Mosbrooker has expertise in gene therapy, rare metabolic diseases, additional orphan conditions and oncology. Mr. Mosbrooker currently serves as the Chief Operations Officer for Cognoa, leading the commercial, program management, product, and business operations functions. Prior to joining Cognoa, he was the Chief Commercial Officer at Audentes Therapeutics overseeing the gene therapy business unit. Mr. Mosbrooker also served as the Senior Vice President of the Global Orphan Business Unit at Horizon Pharmaceuticals. He holds a B.S. in Industrial Engineering from the University of Wisconsin – Madison.
Jean Kim is a recognized healthcare investment partner with more than twenty years of biotechnology experience and leadership on Wall Street. Ms. Kim served as a Partner at Deerfield Management Company LP from August 2006 to July 2020 where she provided extensive research and analysis on individual companies operating in the healthcare industry, with a particular focus on rare and orphan diseases. In addition, Ms. Kim incubated and founded a new gene therapy portfolio company at Deerfield Management with a novel incubator company structure focused on rare orphan monogenic diseases. Prior to joining Deerfield, she was a healthcare investment professional for six years with Merrill Lynch Ventures and a Financial Analyst in Merrill Lynch’s investment banking department. Ms. Kim received her Bachelor of Arts in English Literature and a Bachelor of Science in Biology from Stanford University. She also holds an MBA from Harvard Business School and a Master of Science degree from the Massachusetts Institute of Technology through the Biomedical Enterprise Program and was a Fulbright Scholar. Ms. Kim serves on the Board of Directors of Amplo Biotechnology, a gene therapy company.
Edward Kaye, M.D., has served as CEO and a director of Stoke Therapeutics since October 2017. Dr. Kaye previously served as CEO and a director of Sarepta Therapeutics, where he led the successful push to win FDA approval of Exondys 51, a pioneering drug for Duchenne Muscular Dystrophy. Before taking on the CEO role at Sarepta, he served as the company’s Chief Medical Officer for six years, leading the development of Exondys 51. Earlier in his career, Dr. Kaye spent 10 years at Genzyme Corporation, most recently as Group Vice President of Clinical Development, where he supervised clinical research in programs focused on lysosomal storage disease and genetic neurological disorders. Dr. Kaye currently serves as a member of the boards of directors at Cytokinetics, Inc. and the Massachusetts Biotechnology Council. Prior to entering the biotech industry, Dr. Kaye trained in pediatrics, pediatric neurology and biochemical genetics. He was on the research staff at Massachusetts General Hospital and Tufts University Medical Center and was the chief of biochemical genetics at the Children’s Hospital of Philadelphia. He remains on the pediatric neurology staff at Boston Children’s Hospital. Dr. Kaye earned his B.S. in biology/chemistry from Loyola University and earned his M.D. at the Loyola University Stritch School of Medicine.