- Passage Bio, a young but prominent company developing genetic medicines for brain and nervous system disorders, has added two more programs to its slate of research projects, including one that will investigate treatments for Huntington's disease.
- The program for Huntington's and the other for Canavan disease — also a rare and fatal genetic illness — are the latest development from an existing collaboration between the company and the University of Pennsylvania's Gene Therapy Program. The two parties have been closely linked since Passage was incorporated in 2017. Jim Wilson, a genetic medicine pioneer and director of the Gene Therapy Program, is one of Passage's founding scientists.
- In a Wednesday statement, Passage and the program said they have begun preclinical research that aims to simultaneously develop multiple approaches for targeting the mutated gene and corresponding defective protein that cause Huntington's.
Huntington’s was the first illness ever traced back to changes in a specific chromosome. And yet, almost 40 years since that discovery, no drug has been proven to alter a course of the disease. This year alone saw experimental genetic medicines from Wave Life Sciences and partners Roche and Ionis Pharmaceuticals fail in closely watched clinical trials. Another program, from Voyager Therapeutics, was shuttered as well.
The hunt for an effective medicine hasn't stopped, though. An analysis from the investment bank Piper Sandler found nine gene therapies for Huntington's in development as of September. While most were preclinical, one from the Dutch biotech UniQure has advanced into the early stages of human testing, with initial data expected late next year.
Huntington's is now on Passage's radar, too, adding to a list of research targets that spans from dementia and ALS to lesser-known neurodegenerative illnesses like Krabbe disease and GM1 gangliosidosis. Factoring in the Huntington's and Canavan programs, Passage has three therapies in human trials and another six in preclinical testing.
The Gene Therapy Program has been essential to that pipeline growth, given it's responsible for preclinical research and development activities as laid out by the collaboration with Passage, which was first signed in 2018 and then amended in 2020. Under the updated agreement, Passage provides $5 million in funding each year for five years, and in exchange gets exclusive rights to resulting technologies and drug programs targeting rare diseases of the central nervous system that are caused by mutations in a single gene.
"We are pleased with the continued expansion of our R&D partnership with Passage Bio in our joint quest to develop transformative therapies for patients with devastating CNS disorders," said Wilson, who also serves as Passage's chief scientific advisor.
Investors, though, have been harder to please. As of Wednesday, Passage shares were down 65% compared to the $22 they traded at when the company went public in early 2020.
Passage's share value rose about 1% to 2% following Wednesday's announcement, to trade around $7.70.