Regenxbio has revealed plans to submit a biologics licence application (BLA) for RGX-202, its investigational gene therapy ...

Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for ...

A small trial of an experimental vaccine has shown promising results for patients with the most aggressive type of breast ...

In mice with muscular dystrophy, the treatment restored large therapeutic muscle proteins to normal levels, the researchers ...

Additionally, Sarepta’s expertise in Duchenne Muscular Dystrophy (DMD), coupled with its extensive patient, payer, and prescriber network, reinforces its market leadership. The company’s gene therapy ...

The company expects the trial to support the submission of a biologics license application with the US Food and Drug Administration in 2026.

In a report released yesterday, Tazeen Ahmad from Bank of America Securities maintained a Buy rating on Sarepta Therapeutics (SRPT – Research ...

Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic ...

PTC Therapeutics has received FDA approval for a gene therapy to treat AADC deficiency, a rare genetic disorder that impairs ...

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