Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...

The hold followed Intellia’s voluntary pause in response to a grade 4 liver event—a case of Hy’s Law—in the ATTR-CM trial. Shortly after the regulatory hold was implemented, Intellia disclosed that ...

By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease progression and showed sustained benefit in patients' ability to control and ...

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 trial is funded through Abu Dhabi’s Healthcare Research and Innovation FundClinical development activities will commence ...

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

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

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

The global cell and gene therapy market is experiencing explosive growth. Key growth factors include rising chronic/genetic disease prevalence, advancements in gene editing (CRISPR) and viral vectors, ...

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

For those born with certain types of congenital deafness, the cochlear implant has been a positive and enabling technology.

The market size of CGT products is expected to continue to rise by 2031 and reach $63 billion. CGTs have yielded commercial gains for several pharma companies. For example, some of the top-selling CGT ...