Sarepta Therapeutics will cut 500 jobs and add a serious warning on the label of its muscle-disorder gene therapy Elevidys, the drugmaker said on Wednesday, following the recent deaths of two patients ...

While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...

Ultragenyx's UX111 gene therapy faces FDA delay due to facility issues; analysts adjust price targets amid uncertain ...

Indian American chemist receives prestigious CAREER award for research on synthetic DNA ‘programmable’ nanoparticles ...

BETHESDA, Md. (July 16, 2025) – Today, the Cystic Fibrosis Foundation announced an additional investment of up to $24 million ...

The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech ...

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

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

Just days before South African scientists were set to launch clinical trials of a promising HIV vaccine, their progress was ...

Alfie Clatworthy and Angus Powell are two of only three children in the UK living with CRELD1 and there is currently no cure.

Prof. Jiangfan Yu's team at the School of Science and Engineering, The Chinese University of Hong Kong (Shenzhen), has ...

Joint Press release from Bambino Gesù Children’s Hospital, Rome; Department of Biochemistry and Gurdon Institute, University ...