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

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

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

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

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

The OUTREACH Study – Demonstrating that CAR-T therapies can be effectively administered in community cancer settings: In the ...

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

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

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

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

Nanoscope Therapeutics submitted the first modules of a biologics license application for MCO-010, an investigational gene ...