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

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

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

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 ...

- VY1706 is the fifth neuro gene therapy development candidate nominated leveraging Voyager’s IV-delivered, CNS-penetrant TRACER capsids - ...

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

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.

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 ...

Pfizer's Hympavzi has become the first once-weekly subcutaneous treatment for people living with severe haemophilia B in the ...

A new type of therapy that "edits" a gene in patients with a rare heart condition has been shown to be safe and effective, ...

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