Trump fingers Robert F. Kennedy Jr. to lead the HHS, lupus and ATTR-CM dominate headlines this week, bluebird bio has a cash gap to leap and RegenxBio eyes Sarepta in Duchenne muscular dystrophy.

REGENXBIO Inc. (NASDAQ:RGNX – Get Free Report) has received an average recommendation of “Moderate Buy” from the twelve brokerages that are covering the firm, MarketBeat reports. One research analyst ...

Researchers described a promising new approach for using gene therapy to treat sickle cell disease in the journal Human Gene ...

Metagenomi is preparing to initiate IND-enabling activities that seek to support MGX-001’s transition from preclinical to ...

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

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

Talks with the FDA haven’t gone according to plan. Seeking a streamlined pathway, Neurogene submitted a Regenerative Medicine ...

Regenxbio’s investigational gene therapy improved muscle function for patients with Duchenne muscular dystrophy (DMD) in a phase 1/2 study. | Regenxbio’s investigational gene therapy improved muscle ...

The agency's openness to a targeted pivotal study shows it’s still willing to consider accelerated clearance for Duchenne ...

Regenxbio reported improved muscle function in boys with Duchenne muscular dystrophy using their gene therapy RGX-202. The ...

Wall Street Analysts Think Regenxbio Could Surge 234.37% ... Approves Expanded Use For Sarepta's Rare Muscular Dystrophy Gene Therapy The FDA has approved Sarepta Therapeutics' Elevidys for ...