Topic: Duchenne muscular dystrophy (DMD)
Avidity Biosciences is on a roll—after inking an R&D deal with Eli Lilly and hiring a new CEO, the company is reeling in $100 million.
A new clinical hold is the latest setback for Solid Biosciences and the development of its gene therapy for Duchenne muscular dystrophy.
Sarepta’s chief Doug Ingram has come out against accusations that the FDA is biased against the company after its recent drug rejection.
The FDA rejected the New Drug Application for golodirsen, the follow-up to Exondys 51, Sarepta’s first treatment for Duchenne muscular dystrophy.
Sarepta’s stock plunged 12% on the news that a patient in a study testing its Duchenne gene therapy had been hospitalized for a serious illness.
One of the first six people to receive the treatment was hospitalized with acute renal injury, leading analysts to talk down Pfizer’s chances.
The outlay will give Vertex owenership of Exonics and expand its deal with CRISPR, setting it up to use gene editing to treat DMD and DM1.
The first patient treated with the higher dose of DMD gene therapy SGT-001 suffered a serious adverse event, wiping 35% off Solid’s stock price.
Sarepta licensed five gene therapies for limb-girdle muscular dystrophy from Myonexus last May. Now it's buying Myonexus outright for $165 million.
A Duke lab at the center of a research pact with Sarepta is reporting promising results from a trial of CRISPR gene editing in mouse models of DMD.