Keyword: Sarepta Therapeutics
Lysogene is licensing its Sanfilippo syndrome gene therapy to Sarepta for $15 million upfront and up to $125 million in potential milestones.
Two months after Sarepta was hit with a clinical hold by the FDA, the rare disease biotech has seen this lifted after sorting out a quality blip.
Another gene therapy alliance shows that Sarepta won’t allow an issue with its lead gene therapy candidate to dampen its enthusiasm for the field.
Despite posting promising early data, Summit Therapeutics' Duchenne muscular dystrophy drug missed its primary and secondary endpoints in a phase 2 trial.
The data, taken from three patients, showed a 38% increase in a muscle protein that boys with DMD usually lack.
At BIO, we chatted with Doug Ingram about Sarepta's pipeline, its latest hires and how the healthcare landscape must change.
Celgene's shake-ups continue with new CFO, Zai Lab grabs top AZ exec, Axovant brings on Spark co-founder as CTO, and Gilead promotes its top dealmaker.
Sarepta brought on Biogen’s VP of late-stage programs, Gilmore O’Neill, as chief medical officer, after their previous hire left after 8 months.
The reorg will cost about $1 million but will save the company about $3.3 million a year.
The FDA has extended its partial hold on Solid Bio's phase 1/2 DMD trial to a full hold following a patient's hospitalization.