News

US FDA declines to approve Ultragenyx's gene therapy for rare genetic disorder
The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...
Cystic Fibrosis Foundation8h
Cystic Fibrosis Foundation Commits Up to an Additional $24 Million for Prime Medicine to Develop Gene Editing Therapy
BETHESDA, Md. (July 16, 2025) – Today, the Cystic Fibrosis Foundation announced an additional investment of up to $24 million ...
BioPharma Dive2d
FDA rejects Ultragenyx gene therapy over manufacturing concerns
While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...
STAT2d
FDA declines to approve Ultragenyx gene therapy
In his first 100 days as commissioner of the Food and Drug Administration, Marty Makary has made a handful of splashy ...
Clinical Trials Arena on MSN6d
FDA approves Purespring’s application for Phase I/II trial of IgAN treatment
The trial is scheduled to commence in the fourth quarter of this year, with subject recruitment in both Europe and the US.
Promising leap for cancer therapies: Gene-editing breakthrough rewires T cells to attack tumors safely
T cell-based immunotherapy, a form of cancer treatment, harnesses a patient’s own T cells — white blood cells that help fight disease — by modifying or enhancing them to target and destroy cancer ...
Precision Medicine Online2d
FDA Declines to Approve Ultragenyx's Gene Therapy for Sanfilippo Syndrome Type A
The firm said the agency cited chemistry, manufacturing, and controls issues in its complete response letter, but had no concerns about product quality.