Keyword: Sarepta Therapeutics
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.
Sarepta expects to resume dosing in a U.K. trial of its DMD drug golodirsen pending MHRA approval.
Targeting "clusters" of mutations with CRISPR could lead to a treatment that could work for up to 60% of patients with DMD.