Belite Bio's, Tinlarebant, targets eye diseases STGD1 and GA, showing promising results but with delayed phase 3 trial data.

We recently compiled a list of the 10 Best Genomics Stocks To Buy Right Now. In this article, we are going to take a look at ...

On Monday, Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) presented long-term safety and efficacy results from the Phase 1 study ...

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 ...

Rocket Pharmaceuticals (RCKT – Research Report), the Healthcare sector company, was revisited by a Wall Street analyst today. Analyst Gil Blum ...

The firm's chief scientific officer called the achievement "a major milestone for Spirovant and for the cystic fibrosis ...

Failing to receive the RMAT designation from the FDA for its early-stage Batten disease gene therapy, Neurogene tells ...

The U.S. Food and Drug Administration (FDA) has announced a groundbreaking approval of Kebilidi (eladocagene exuparvovec-tneq ...

Ocugen has announced positive Phase 1 trial results for OCU410, a one-time gene therapy for geographic atrophy secondary to ...

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