Passage Bio to tackle CNS diseases with $115M and UPenn gene therapy programs

Philadelphia city skyline
Specifically, the collaboration is between Passage Bio and the University of Pennsylvania, its Gene Therapy Program and its Orphan Disease Center. (Pixabay Creative Common CC0)

Passage Bio reeled in a $115.5 million series A on Thursday, which will support the development of five programs aimed at treating rare monogenic central nervous system (CNS) diseases. Passage Bio is working on those five under a research, collaboration and license agreement with the University of Pennsylvania and could, in the future, choose to fund preclinical work for to seven more. 

Specifically, the collaboration is between Passage Bio and Penn, its Gene Therapy Program, and the Penn Orphan Disease Center. Of the seven preclinical programs that Passage Bio could potentially fund, the Philadelphia-based company has the option to license IP that comes out of them, according to a statement. 

“Passage Bio’s development portfolio presents an unparalleled opportunity to transform the lives of patients with rare monogenic CNS diseases,” said Stephen Squinto, Ph.D., the CEO of Passage Bio and a venture partner at OrbiMed Advisors, which led the financing. Frazier Healthcare Partners, Lilly Asia Ventures, New Leaf Venture Partners, Versant Ventures and Vivo Capital also chipped in. 

The company plans to push its lead programs, in GM1 gangliosidosis and frontotemporal dementia, into the clinic in early 2020, Squinto said in the statement. 

RELATED: Alector raises $133M in 5th VC round to push neuro-immunology drugs into Alzheimer’s trials 

“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 James Wilson, M.D., Ph.D., the gene therapy pioneer who wears several hats at Penn. He directs the university’s Gene Therapy Program and Orphan Disease Center and is a professor of medicine and pediatrics at its Perelman School of Medicine. 

“Our team at Penn is extremely experienced and has been on the cutting edge of AAV research for over 20 years,” he said. “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.” 

RELATED: New year, new Axovant: CEO Cheruvu on the company's gene therapy pivot 

Passage Bio isn’t the only company looking to treat the lysosomal storage disorder GM1 gangliosidosis. Others include Axovant and Lysogene. Last year, Axovant pivoted from Alzheimer’s disease to gene therapies, officially rebranding as Axovant Gene Therapies and picking up treatments for GM1 and GM2 gangliosidosis, as well as for oculopharangeal muscular dystrophy. It expects data from two early-stage trials soon, including in Parkinson’s disease and GM2 gangliosidosis.