Ultragenyx dives into Duchenne with $40M Solid Bio deal

06 November 2020


Duchenne

Solid Biosciences has had a roller coaster couple of years, with its gene therapy for Duchenne muscular dystrophy enduring multiple FDA holds due to safety concerns and manufacturing issues. Now, the biotech is getting a hand with its Duchenne work through a partnership with Ultragenyx worth $40 million upfront but could net it another $255 million in milestone payments.


Under the deal, the duo will work on gene therapies for Duchenne and other diseases stemming from a lack of the dystrophin protein, with Solid Bio contributing its microdystrophin construct and Ultragenyx bringing its gene therapy manufacturing platform. Ultragenyx picks up an exclusive license to any treatments that come out of the collaboration, but Solid Bio may choose to co-fund programs once they achieve proof of concept, the companies said in a statement.

Solid Bio holds on to the rights to other uses of its microdystrophins, including its troubled Duchenne program, SGT-001. The FDA lifted its latest clinical hold on the program earlier this month.

Duchenne is caused by mutations in the dystrophin gene that stop it from producing the dystrophin protein. Without it, muscle fibers, including those in the heart, eventually weaken and die. Treatments like SGT-001 and Sarepta’s SRP-9001 are designed to give patients a gene that makes microdystrophin—a truncated version of dystrophin—to make up for patients’ lack of dystrophin. Some companies are taking this route because the dystrophin gene is too big to fit into AAV vectors.

“Ultragenyx has a demonstrated track record of success in developing and commercializing innovative therapies for rare diseases,” said Solid Bio CEO Ilan Ganot in the statement. “We believe it is the partner of choice for exploring new gene therapy opportunities for patients with Duchenne.”

The partnership marks Ultragenyx’s entry into muscular dystrophy. The company already had two gene therapies in its pipeline, picked up in its 2017 acquisition of Dimension Therapeutics: DTX401, a treatment for glycogen storage disease type Ia, in which the body’s cells accumulate the sugar glycogen, and DTX301, a treatment for ornithine transcarbamylase deficiency, in which ammonia builds up in the blood. Ultragenyx posted promising data for both in May this year.


Published by fiercebiotech.com on October 23, 2020

Image by Shutterstock