Passage Bio emerged early last year with a sizable war chest, five gene therapy programs from the University of Pennsylvania and the option to license seven more. Now, the company is doubling down on that partnership, promising the university $5 million a year in exchange for access to more programs and new technologies that come out of the deal.
Under the expanded deal, Passage may license five more programs from Penn’s Gene Therapy Program for a total of 17. It also adds three years to the period in which Passage must pull the trigger on those programs, extending the deal from 2022 to 2025. But the jewel in the crown is the chance to tap new advances—such as better treatment delivery—from the lab of Jim Wilson, M.D., Ph.D., the gene therapy pioneer who co-founded Passage.
The original agreement included some of those advancements, like new capsids—the protein shell of a virus—for delivery, but the expansion gives Passage exclusive rights to technology that comes out of Penn’s discovery program, including capsids, formulation improvements and technologies to make gene therapies safer.
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“We have an expanded opportunity to incorporate advances that are happening at Penn—research advances that will help us [beyond] the initial programs we had options to,” Passage CEO Bruce Goldsmith, Ph.D., told FierceBiotech.
“We want to be able to partner with Jim and his group on leading those advances and incorporating them into programs as appropriate … and on an ongoing basis,” he added.
Passage Bio launched in February 2019 with $115.5 million and wasted no time raking in capital, from a $110 million series B round seven months later to a $216 million Wall Street debut in February of this year. Along the way, the company hired Chief Medical Officer Gary Romano, M.D., Ph.D., from Johnson & Johnson's Janssen and recruited Goldsmith to take over from founding CEO Stephen Squinto, Ph.D. It has also licensed the first of seven potential programs included in the original deal.
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It’s aiming to bring its lead program, a treatment for rare childhood disease GM1 gangliosidosis, into the clinic by the end of the year. Gene therapies for Krabbe disease and frontotemporal dementia will follow in the first half of 2021. Its earlier-stage programs target metachromatic leukodystrophy, a specific subset of patients with amyotrophic lateral sclerosis and patients with Charcot-Marie-Tooth hereditary neuropathy type 2A stemming from a particular mutation.
Passage isn’t waiting for the programs it already has to hit particular goals before it taps Penn for more.
“We’re focused on taking things that are compelling and the way we define compelling is really thinking about patient needs,” Goldsmith said. Any program selections it makes will hinge upon those needs, intersecting with the “evolution of the science” and Penn’s ability to “deliver transformative aspects” in its new gene therapy programs.