FDA is investigating Sarepta's Elevidys after two deaths from liver failure in non-ambulatory DMD patients, raising safety ...

Sarepta stock dropped again Wednesday after the FDA announced it's investigating the deaths of two patients who received Elevidys.

The biotech will work on molecular glue degraders with Gilead in a new deal. Existing partner Sanofi changed its focus to a ...

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

Compass Pathways' Phase 3 COMP-360 data met primary endpoints but disappointed vs analyst expectations. See why investors ...

The FDA is assessing the need for “further regulatory action” on Sarepta's Duchenne muscular dystrophy gene therapy in the ...

In a functioning system, the kind of placebo-controlled vaccine trials that Secretary Kennedy is calling for would never get past an IRB, much less progress to an IND application. Vaccines are being ...