The Charcot-Marie-Tooth Association (CMTA) has announced a $300,000 investment in a groundbreaking gene editing project aimed ...

Childhood-onset striatonigral degeneration is a rare genetic disorder that robs children of the ability to walk and speak by ...

Intrathecal treatment with a vector-based gene therapy was associated with a greater improvement in motor function at 52 ...

The HFpEF Summit is a biannual, two-day meeting bringing together internationally recognized leaders in the clinical, biological, and translational study of HFpEF. This summit focused on the latest ...

Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...

Here is our list of the 11 Best Gene Therapy Stocks to Buy According to Analysts.

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

The arc of biotechnology is one of breakthroughs and struggles, writes Tim Hunt, CEO of the Alliance for Regenerative ...

Three patients living with hypertrophic cardiomyopathy have had their disease classification downgraded following a single ...

Durham-based Precision BioSciences Inc. has received investigational new drug (IND) clearance from the U.S. Food and Drug Administration (FDA) for PBGENE-HBV, an in vivo gene editing program designed ...

Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...

CROSS-SPECIES BBB-PENETRANT IV-DELIVERED AAV GENE THERAPY PROVIDES BROAD AND ROBUST CNS TAU LOWERING IN TAUOPATHY MOUSE AND ...