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Managing for resilience: Passage’s ups and downs in gene therapy

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When Passage Bio was founded, it was a sort of comeback story for gene therapy pioneer Dr. James Wilson.

After making a name for himself as a researcher, Wilson became head of the Institute for Gene Therapy at the University of Pennsylvania. Then in 1999, a patient in a clinical study died after being dosed with a gene therapy created in Wilson’s lab. The tragic and well publicized event brought the field to a screeching halt. And in 2005, an FDA investigation into the patient’s death led to a five-year ban from clinical trials for Wilson.

Throughout that time, however, Wilson kept at his work outside the clinical arena. In 2002, he published research showing that that gene therapies could be more safely delivered using adeno-associated virus vectors. He also eventually got into the world of startups, first launching Regenxbio in 2009 and a decade later, Passage Bio, which got off the ground with $115.5 million in funding and a handful of promising gene therapy candidates.

Now, just a few years later, Passage is looking for a comeback of its own.

In March 2022, the clinical-stage company announced it was cutting about 13% of its workforce. After a 2021 IPO, the biotech’s market cap had soared to over $1 billion, but within about a year, shares for Passage plummeted about 90% amid a major downturn in the market that saw values tumble across the sector.

“The goal with these changes so soon after I started with Passage was to make all the hard decisions as soon as possible so that we can move into a rebuilding phase.”

Dr. William Chou

CEO, Passage Bio

Then in June, Passage’s CEO abruptly stepped down, opening the door for new leadership. A few months later, Dr. William Chou took the reins as Passage’s new CEO — a job he knew wasn’t going to be easy.

More job cuts followed about a month after Chou came on board along with news that Passage would have to slash its R&D efforts. The restructuring left the company with about 100 employees, two drug candidates in phase 1/2 trials — and a clear impetus among leadership to help Passage bounce back.

“I came in with eyes wide open,” Chou said. “But I had a belief in the science and in the data that has been published preclinically on these programs, so you have to believe in the fundamentals.”

There’s a lot on the line — not just for Chou and Passage, but also for patients.

One of Passage’s two remaining therapies targets GM1 gangliosidosis, a rare inherited condition that attacks nerve cells in the brain and spinal cord, and in its type 1 form, appears in babies where it is fatal before the age of 2. Right now, Passage’s gene therapy, PBGM01, is the only candidate in clinical trials for the disease.

The company’s other lead asset, PBFT02, targets frontotemporal dementia (FTD), a rare genetic mutation that can trigger early onset dementia.

Passage also has three therapies in discovery and is hoping to license out rights for two assets it had to discard — one in Krabbe disease and the other for metachromatic leukodystrophy.

“Stopping our programs had nothing to do with the science and what we think they can deliver to patients,” Chou clarified. “It was a matter of prioritizing the most advanced programs.”

After scaling back its workforce and pipeline, Chou said Passage is at the right size to move forward. The company has a cash runway that should carry it into 2025 and a unique platform developed by Wilson, who now serves as the company’s chief scientific officer.

And Chou, who was formerly the CEO of gene therapy and rare disease specialist Aruvant Sciences, as well as the vice president, global disease lead for Novartis’ cell and gene therapy unit, might be just the right leader to chart its new course.

Here, Chou discusses Passage’s critical milestones for 2023 and his tips for leading through tough times.

This interview has been edited for brevity and style.

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