The Centers for Medicare and Medicaid Services (CMS) has selected 35 participants for its new, voluntary Cell and Gene ...

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a new gene therapy for Amyotrophic Lateral ...

Paris: Sanofi has received fast track designation from the US Food and Drug Administration (FDA) for SAR446597, a one-time ...

The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...

Ultragenyx Pharmaceuticals RARE announced that the FDA has issued a Complete Response Letter (CRL) for its biologics license ...

Nanoscope Therapeutics begins rolling submission of BLA to US FDA for gene-agnostic therapy, MCO-010 to treat retinitis pigmentosa: Dallas, Texas Wednesday, July 16, 2025, 14:30 H ...

Ultragenyx Pharmaceuticals took another hit when the FDA rejected its gene therapy for patients with a rare, genetic disorder.

Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer ...

Groundbreaking new research shows that it is possible to use gene therapy to restore hearing in both children and adults.

This is the first time such results have been achieved in both children and adult patients born with a specific type of ...

In his first 100 days as commissioner of the Food and Drug Administration, Marty Makary has made a handful of splashy ...

A single shot of gene therapy restored hearing in people born deaf, with most showing improvement within weeks and some ...