The funding will be used to develop a portfolio of five therapeutic candidates to treat rare monogenic CNS diseases. These programs are being developed under a research, collaboration and license agreement with the University of Pennsylvania (Penn) and its Gene Therapy Program (GTP) together with the Penn Orphan Disease Center (ODC), with the support of co-founder James M. Wilson, M.D., Ph.D.  Passage Bio also has an option to fund the preclinical development of up to seven additional rare monogenic CNS indication programs at the GTP and license new intellectual property arising from these programs from Penn.

Co-founder and interim chief executive officer at Passage Bio, Stephen Squinto, Ph.D., is a venture partner at OrbiMed Advisors with more than 25 years of biotechnology industry experience. Dr. Squinto was a co-founder of Alexion Pharmaceuticals Inc. and recently served as its executive vice president and chief global operations officer. While at Alexion, Dr. Squinto was involved in the discovery, development and commercialization of Soliris, one of the world’s most successful orphan drug products for patients with paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome and generalized myasthenia gravis.

“Passage Bio’s development portfolio presents an unparalleled opportunity to transform the lives of patients with rare monogenic CNS diseases,” said Dr. Squinto. “We look forward to continuing progress in this exciting field of therapeutics and advancing our lead programs in GM1 gangliosidosis and frontotemporal dementia into the clinic in early 2020.”

Co-founder and Chairman of the Board at Passage Bio, Tachi Yamada, M.D. is a venture partner at Frazier. Prior to joining Frazier he was chief medical and scientific officer at Takeda Pharmaceuticals and has served as chairman of Research and Development at GlaxoSmithKline.

“We co-founded Passage Bio with a shared commitment to serving patients with high unmet needs. Both Dr. Wilson and I have been working in the gene therapy field for over 30 years, and this deep understanding will be instrumental as we grow and advance this new world-class gene therapy company,” said Dr. Yamada. “This financing will allow us to continue to build out our industry-leading team and advance our therapies into the clinic as rapidly as possible.”

Co-founder and chief scientific advisor at Passage Bio, James M. Wilson, M.D., Ph.D., is director, Gene Therapy Program; Rose H. Weiss Professor and Director, Orphan Disease Center; Professor of Medicine and Pediatrics, Department of Medicine at the Perelman School of Medicine at the University of Pennsylvania. Dr. Wilson’s laboratory has made seminal contributions to the technology of gene transfer and has paved the way for translation of these technologies into the clinic. He has published over 550 papers and is named on over 110 patents. 

“We believe this is a truly unique partnership, which gives Passage access to certain Penn AAV technologies developed at the GTP, our strong preclinical translational science capabilities and orphan drug development know-how,” said Dr. Wilson. “Our team at Penn is extremely experienced and has been on the cutting edge of AAV research for over 20 years. We are confident in this team’s ability to move new treatments for rare CNS monogenic diseases through clinical development in an effort to one day provide new treatment options for patients with chronic unmet needs with high mortality.”