Partnership established to develop and scale production of ATL-105, a novel recombinant version of alpha-1 antitrypsin ...

Amid a season of regulatory and scientific advances, experts reveal a culture of data hoarding among cell and gene therapy ...

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

On an investor call Friday, analysts grilled the company over its apparent lack of transparency on the matter.

RARE faces regulatory setback as FDA issues a CRL for UX111, delaying approval of the MPS IIIA gene therapy until 2026.

The regulator had asked Sarepta Therapeutics to halt all shipments of its therapy, Ele­vidys, after three patients died from ...

The head of the US Food and Drug Administration said the agency will “take a hard look” at a gene therapy from Sarepta Therapeutics Inc. after two patient deaths, adding pressure to the biotech ...

Danaher, a United States-headquartered life sciences conglomerate, announced a strategic partnership in Shanghai on Monday between its operating company Cytiva and CorrectSequence Therapeutics, a ...

From Wall Street to real estate to a “big, ugly” pharma building, Mayo Venture Partner Audrey Greenberg reflects on a career ...

Scientists have successfully restored hearing in children and young adults with congenital deafness using gene therapy targeting the OTOF gene, marking a groundbreaking step toward curing inherited ...

Manufacturing issues are the latest problem for Ultragenyx Pharmaceutical Inc. to solve after last week’s disappointment in a phase III study to treat brittle bones. The U.S. FDA gave the company a ...