Philly's Passage Bio, with relationship to Penn, raises $115.5 million in a Series A for rare diseases

Tom Paine

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James M. Wison / Penn Today

Philadelphia-based Passage Bio has raised $115.5 million in a Series A to develop cures for rare diseases , led by OrbiMed Advisors.

The funding will bd used to develop five therapeutic compounds to treat rare monogenic diseases of the central nervous system (CNS). Passage Bio has a license agreement with the University of Pennsylvania and its Gene Therapy Program along with the Penn Orphan Disease Center (ODC). James M. Wilson, a professor of Medicine at Penn's Perelman School of Medicine, is the company's co-founder and chief scientific advisor. Wilson is a pioneer in the gene therapy field.

Under the terms of their agreement, UPenn conducts the preclinical research, while Passage is responsible for clinical testing and eventual commercialization.

Last year’s approval US approval of the first viral-based gene therapy, Spark Therapeutics’ Luxturna, opened the door to others in the field.

“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 in a statement. “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.”

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— Tom Paine (@phillytechnews) February 17, 2019