A team at the Centro Nacional de Investigaciones Cardiovasculares (CNIC) has developed an innovative gene-therapy strategy ...

Investigators have developed STITCHR, a new gene editing tool that can insert therapeutic genes into specific locations without causing unwanted mutations. The system can be formulated completely as ...

Maria Lowe, Ph.D., associate vice president of Pharmaceutical Intelligence at Institute for Clinical and Economic Review ...

Investigators from Mass General Brigham and Beth Israel Deaconess Medical Center have developed STITCHR, a new gene editing tool that can insert therapeutic genes into specific locations without ...

A recent study demonstrated the potential of gene therapy to restore motor capacity in an ultra-rare disease, megalencephalic ...

As the countdown to the launch of its first therapeutic continues, Rocket Pharmaceuticals is fueling up its commercial team.

Gene therapy, a technique that is revolutionizing the treatment of multiple genetic conditions, including eye and muscle diseases and blood disorders, requires efficient and specific delivery of the ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved ...

Marks’ resignation leaves the field without a regulator many view as “integral” to its progress over the last decade.

A team at CNIC has developed an innovative gene therapy to treat arrhythmogenic right ventricular cardiomyopathy type 5 ...