A developer of gene therapies for rare monogenic CNS diseases—whose co-founders include pioneer researcher James M. Wilson, MD, PhD—has launched with $115.5 million in Series A financing.

Passage Bio said it will use proceeds from the financing to develop a portfolio of five therapeutic candidates that incorporate adeno-associated virus (AAV) technologies developed by Wilson and colleagues at the University of Pennsylvania (Penn) and its Gene Therapy Program (GTP).

The gene therapy candidates will be developed through a research, collaboration, and license agreement with Penn and GTP, together with the Penn Orphan Disease Center (ODC), with the support of Wilson, who is director of both GTP and ODC, professor of medicine and pediatrics at Penn’s Perelman School of Medicine, and the company’s chief scientist advisor.

“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,” Wilson said in a statement.

Passage Bio retains 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.

“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,” stated Stephen Squinto, PhD, co-founder and interim CEO of Passage Bio.

GM1 gangliosidosis is an autosomal recessive genetic disorder, caused by an inactivating mutation of the lysosomal enzyme β-galactosidase (GLB1), which is required for the degradation of GM1 ganglioside and keratan sulfate.

Frontotemporal dementia (FTD) typically appears in patients who are in their 60s and 70s with progressive impairment of executive function, language, and social interaction. The symptoms are associated with a characteristic pattern of neurodegeneration affecting the frontal and temporal cortices. In 5–10% of FTD patients, according to Passage Bio, pathogenic loss-of-function mutations can be identified in the gene encoding progranulin, a ubiquitous lysosomal protein.

Blockbuster background

Squinto is also a co-founder of Alexion Pharmaceuticals, where he most recently served as EVP and chief global operations officer. At Alexion, he was involved in the discovery, development, and commercialization of the blockbuster orphan drug Soliris® (eculizumab), indicated for patients with paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, and generalized myasthenia gravis. Soliris generated $3.563 billion in sales in 2018, up 13% from $3.144 billion in 2017.

Squinto is now a Venture Partner at OrbiMed Advisors, which led the Series A financing, joined by Frazier Healthcare Partners, Versant Ventures, New Leaf Venture Partners, Vivo Capital, and Lilly Asia Ventures (LAV).

LAV originated in 2008 as a corporate venture subsidiary of Eli Lilly, but later spun off and became an independent investment management company with more than $1.2 billion of committed capital under management.

A venture partner at Frazier, Tachi Yamada, MD, is another co-founder of Passage Bio and serves as its chairman of the board. Before joining Frazier, he was chief medical and scientific officer at Takeda Pharmaceuticals, and has served as chairman of research and development at GlaxoSmithKline.

Wilson’s lab has pioneered vector technology for nearly three decades, including the discovery and development of novel serotypes of AAV, and has contributed to the development of a variety of nonviral and viral platforms. He led the research team in the 1999 clinical trial in which a patient who suffered from a liver enzyme deficiency, 18-year-old Jesse Gelsinger, died four days after treatment with gene therapy. Gelsinger received the highest dose in the trial, 38 trillion virus particles.

Wilson has published more than 550 research papers, and is named on over 110 patents. After launching his faculty career in the Howard Hughes Medical Institute at the University of Michigan, he moved to Penn in 1993. Wilson completed his training in Internal Medicine at Massachusetts General Hospital, followed by a postdoctoral fellowship at the Whitehead Institute, where he began his work in gene therapy.

Passage Bio disclosed that Wilson and three Penn-associated shareholders—the Wilson Family Trust, Julie Johnston, and Monique Molloy—hold founder shares in the company. Penn and GTP will receive sponsored research funding from Passage Bio, while Wilson and Penn may receive additional future financial benefits under the license as an inventor of the licensed technology.

Penn also holds an equity interest in Passage Bio, and Wilson is compensated for his scientific advisory position, the company also acknowledged.

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