With 5 Gene Therapies Licensed From Penn, Passage Bio Launches With $115.5 Million Series A

Philadelphia-based Passage Bio launched with a Series A financing round worth $115.5 million. It was led by OrbiMed Advisors and joined by Frazier Healthcare Partners, Versant Ventures, New Leaf Venture Partners, Vivo Capital and Lilly Asia Ventures.

The funds raised will be used to advance a portfolio of five therapeutic compounds to treat rare monogenic diseases of the central nervous system (CNS). They are currently being developed under a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program along with the Penn Orphan Disease Center (ODC) with the support of company co-founder James. M. Wilson.

The company’s co-founder and chief executive officer is Stephen Squinto, former co-founder of Alexion Pharmaceuticals, now a venture partner at Frazier Healthcare Partners. Wilson is the company’s chief scientific advisor. Tachi Yamada is the company’s chairman and co-founder. He is a Venture Partner at Frazier. Before joining Frazier he was chief medical and scientific officer at Takeda Pharmaceuticals. He also served as chairman of Research and Development at GlaxoSmithKline.

“Passage Bio’s development portfolio presents an unparalleled opportunity to transform the lives of patients with rare monogenic CNS diseases,” stated 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.”

GM1 gangliosidosis (GM1) is an autosomal recessive genetic disorder. It is the result of a mutation of the lysosomal enzyme beta-galactosidase (GLB1). The infantile type of GM1 is the most common and most severe type of disease. It usually presents with gait abnormalities by four months and developmental regression by six months. Most quickly regress with mortality by about two years of age. There are currently no disease-modifying therapies.

Frontotemporal dementia (FTD) is very similar to Alzheimer’s disease. It typically shows up in patients in their 60s or 70s. In about 5 to 10 percent of FTD patients, loss-of-function mutations can be identified in the gene for progranulin, a ubiquitous lysosomal protein. There is typically average survival of eight years from onset of symptoms.

In addition to the five compounds optioned from UPenn, it has an option to fund and license another seven. Under the terms of the collaboration, UPenn conducts the preclinical research, then turns the gene therapies over to Passage for clinical testing and eventual commercialization.

Xconomy writes, “The financing comes at a time of great momentum for gene therapy, which has taken decades to begin to fulfill its promise. The idea is to use a modified virus or some other ‘vector’ to deliver healthy genes into a cell to replace faulty or missing ones. In theory, this could attack the root cause of a patient’s disease and provide a long-lasting, if not permanent treatment through a single procedure.”

“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,” Wilson stated. “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.”