Passage Bio Launches with $115.5 million series A funding for treating rare monogenic CNS diseases.

Passage is developing gene therapies for CNS-targeted rare diseases. It launches with $115.5 Mn in series A funding aimed at treating rare monogenic central nervous system (CNS) diseases.

Passage Bio launches with $115.5 Mn in series A funding, which will support the development of five programs aimed at treating rare monogenic central nervous system (CNS) diseases led by OrbiMed Advisors and joined by Frazier Healthcare Partners, Versant Ventures, New Leaf Venture Partners, Vivo Capital and Lilly Asia Ventures.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.

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.

“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.”

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.