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

By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...

Under the agreement, participating states will receive “discounts and rebates” from the drugmakers if the treatments don’t ...

After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing ...

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 ...

A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...

Precision DNA editing gene therapy achieves deep remissions in aggressive leukemia previously considered incurable.

Doctors have delivered gene therapy during heart bypass surgery for the first time, aiming to strengthen blood vessels ...

OCU410 controlled lesion growth 46% better than placebo in dAMD-related GA.

"Regulatory flexibility must be tailored for cell and gene therapies," commented FDA Commissioner Marty Makary. "These are common-sense reforms that will address the unique characteristics of cell and ...

Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for a progressive muscle‑wasting disorder showed sustained benefit in ...