Passage Bio, a new start-up cofounded by University of Pennsylvania professor James Wilson, has raised $115.5 million in series A financing to develop gene therapies for rare genetic diseases of the central nervous system.
Wilson’s research on adeno-associated viruses (AAVs), which are used to deliver DNA into cells, has been instrumental in catalyzing a gene-therapy boom. In 2009, Wilson helped found the AAV-based company Regenxbio. It now licenses AAVs to 11 other companies, including Novartis, which expects its experimental therapy Zolgensma to soon become the second gene therapy ever approved in the US.
Wilson wanted to start a new company that he could be intimately involved with, explains Stephen Squinto, a partner at the venture capital firm OrbiMed Advisors and current CEO of Passage. After visiting Wilson’s lab at Penn, Squinto devised a unique arrangement for the new start-up.
“He runs his gene-therapy center like you’d run a very efficient company,” Squinto says. “Why not use the world’s most proficient AAV lab to do your preclinical work?” Wilson, Squinto, and Tachi Yamada—a partner at Frazier Healthcare Partners—decided that Passage would pay Wilson’s lab to create AAVs and run experiments in monkeys. Passage will take over once it’s time for experiments in humans.
“It will be a drug development organization without a research and discovery group because we are leveraging the expertise of Penn for that purpose,” Squinto says.
Passage is focusing on five rare diseases that Wilson’s lab has already been studying, including a disease called GM1 gangliosidosis that affects and often kills infants and a disease called frontotemporal dementia that affects adults.