Keyword: Sarepta Therapeutics
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.
At 24 weeks, Summit's ezutromid raised utrophin levels and reduced muscle damage in a phase 2 trial.
We catch up with Doug Ingram, chief at rare disease biotech Sarepta, and discuss how he aims to remove the term “controversial” in front of the company’s name.
Sarepta has been signing a series of new R&D pacts since its controversial DMD drug approval last fall, and today it’s penned a deal for one of the hottest research areas around: CRISPR.
Catabasis is looking to test its experimental Duchenne drug edasalonexent in a late-stage trial.
Allergan CFO Hilado and Gilead COO Young will retire from their positions and Rubius has poached a GSK SVP as CMO.
The startup has secured the support of the ex-Sarepta CEO’s Xontogeny and $10 million to take a LANCL2-targeted IBD drug into the clinic.
Sarepta CEO Doug Ingram aims to raise $250 million in a public stock offering as the company looks beyond its first product, DMD therapy Exondys 51.