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 ...

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

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

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

The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...

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

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

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

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

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

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