Angela Lek, VP of research, Muscular Dystrophy Association (Moderator)
Dr. Angela Lek is VP of Research at the Muscular Dystrophy Association (MDA) with expertise in muscular dystrophies and genetic therapies. She completed her PhD at the University of Sydney, studying Limb Girdle Muscular Dystrophy, followed by postdoctoral research at Boston Children’s Hospital and Harvard Medical School on Facioscapulohumeral Dystrophy. Previously, she led a translational research program at Yale. At MDA, she oversees research grants, venture philanthropy, and the scientific direction of the Kickstart program for ultra-rare gene therapy development. Dr. Lek also consults on gene therapy projects, driving research from bench to clinic.

Alan Beggs, Director of The Manton Center for Orphan Disease Research, Boston Children’s Hospital
Dr. Beggs is Director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital and Sir Edwin & Lady Manton Professor of Pediatrics at Harvard Medical School. Following undergraduate studies at Cornell University, he obtained his PhD in Human Genetics at Johns Hopkins University, with subsequent postdoctoral training in medical and molecular genetics at Johns Hopkins and Boston Children’s hospitals. Throughout his career, he has used the toolset of human molecular genetics to study normal muscle biology and pathophysiology in a variety of neuromuscular diseases with a particular focus on the congenital myopathies. In this role, and as founding director of The Manton Center, he has led the discovery of numerous novel rare and ultra-rare disease genes and has pioneered the development of gene replacement and other molecular therapies for neuromuscular disease.

Olivier Danos, EVP & CSO, REGENXBIO
Olivier Danos is Executive Vice President and Chief Scientific Officer at REGENXBIO. He is a pioneer in the field of gene therapy, and has dedicated his career to advancing the use of this technology to develop life-saving therapies for patients. Olivier joined REGENXBIO in 2017 from Biogen where he was a Senior Vice President in charge of Cell and Gene Therapy. Over the past twenty years, he has played leadership roles in cell and gene therapy as Director of the Gene Therapy Consortium of the University College of London, at the Necker Hospital – Enfants Malades in Paris, as Chief Scientific Officer of Genethon and as senior director of research at Somatix Therapy Corporation. He has held senior research positions in France at the Centre National de la Recherche Scientifique and at the Institut Pasteur. Olivier is the former President and a founding member of the European Society of Gene and Cell Therapy. Olivier received a Master’s in Genetics and Molecular Biology at University of Paris Orsay, and his Ph.D. at the Pasteur Institute and University of Paris Diderot.

Rachel Saltzman, CEO, Armatus Bio
Dr. Rachel Salzman is the CEO of Armatus Bio and a leader in drug development for rare diseases, where biology and business intersect to address unmet medical needs. Previously, she served as Executive Vice President at Alcyone Therapeutics and co-founded SwanBio Therapeutics, where she was CEO and later President and Chief Portfolio & Development Officer. In 2021, she founded UltraSquared Bio, a nonprofit dedicated to developing gene therapies for ultra-rare diseases. Prior to that, Dr. Salzman was Chief Science Officer at The Stop ALD Foundation, advancing therapies for X-linked adrenoleukodystrophy. She holds a B.S. in Animal Science from Rutgers University and a DVM in Veterinary Medicine from Oklahoma State University, bringing deep expertise in both science and business to her work.