Gene therapies have long held promise for rare diseases — many of which are monogenic — as an elegant cure that corrects the singular underlying genetic defects. The execution, though, has been anything but elegant, spawning headaches about safety issues and delivery methods.
Penn professor James Wilson knows the roller coaster ride all too well. And as the field gains steam following the historic approval of the first gene therapy with several more lined up at the FDA, he is teaming up with a rare disease expert and an industry vet to develop new therapeutics using a technology that he’s helped perfect.
Emerging out of a research, collaboration and license agreement with Penn’s Gene Therapy Program and Orphan Disease Center, Passage Bio has packed its list of supporters with marquee biopharma investors: OrbiMed Advisors, Frazier Healthcare Partners, Versant Ventures, New Leaf Venture Partners, Vivo Capital and Lilly Asia Ventures.
They are collectively devoting $115.5 million to Passage Bio’s plan of developing five AAV-based therapies for rare monogenic diseases of the central nervous system, taking over once its academic partners finish the IND-enabling preclinical work. Under the pact with Penn, the biotech might also nab a few more preclinical programs and license new IP later.
Passage Bio will start with two indications with an eye for clinical entry in early 2020: GM1 gangliosidosis (GM1), a neuron-destroying disorder most common in infants; and frontotemporal dementia, which overwhelmingly affects the elderly.
Alexion co-founder and OrbiMed partner Stephen Squinto is running the shop as interim CEO, while Tachi Yamada — a VC at Frazier formerly of GlaxoSmithKline and Takeda — takes on the chairman’s role.
As Xconomy notes, Yamada was an old mentor of Wilson’s who offered crucial support in the immediate aftermath of a clinical trial that led to the death of a teenager and sent the gene therapy field into a harsh winter.
Wilson has since co-founded Regenxbio and advised Solid Bio, a biotech developing a gene therapy for Duchenne muscular dystrophy. A year ago, however, he abruptly resigned from Solid Bio’s board and subsequently sounded an alarm on high-dose AAV studies based on toxic reactions in monkeys.
There was no mention of that in the statement about Passage Bio Thursday. Instead, Wilson heralded the “truly unique partnership.”
“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.”
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