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

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

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

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

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

Researchers at Children's Hospital of Philadelphia (CHOP) announced encouraging results from the first ever gene therapy ...

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

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

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 ...

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

The FDA has released a draft of new guidance addressing common FAQs about the development of potential CGT treatments.

The key benefit of zerlasiran’s approach is its ability to directly target and silence the gene responsible for Lp(a) ...