University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse ...

Lilly inks gene therapy deal with Seamless Therapeutics to develop programmable recombinase treatments targeting genetic hearing loss worldwide.

Fractyl Health is developing a shot that would program the body to make more of the GLP-1 hormone naturally, a risky bet that it can provide a longer-lasting benefit than blockbuster weight-loss drugs ...

The new method is designed to focus specifically on pain-related signals, without interfering with normal activity in other parts of the brain. A new preclinical study has identified a gene therapy ap ...

Gene therapy approaches include gene replacement, suppression, and editing, each matched to specific genetic mechanisms in hereditary hearing loss. Preclinical studies in rodent models show promise, ...

US biotech firm Fractyl Health is developing Rejuva, an experimental gene therapy that may mimic GLP-1 drugs for years after ...

Potency assays are central to proving a therapy activity and safety, yet for advanced therapies like cell and gene therapies ...

For years, cell and gene therapies were mainly associated with rare and orphan diseases, those that impact small patient groups and lack effective treatment options. However, recent developments in ...

Few companies stand better poised than Beam Therapeutics to reap the first fruits of the FDA’s promised flexibility toward cell and gene therapies. | Riding a regulatory win for its base editing ...