GAAP revenue of $14.2 million for Q2 2025 beat analyst estimates and Revenue more than doubled year over year in the second quarter, beating analyst estimates. Net loss per share (GAAP) narrowed to ...

India has over 180 children with Hunter Syndrome, but many people are not even aware of this genetic disorder which mostly ...

Sarepta did not hold an investor call for its second-quarter earnings report or provide an updated full-year revenue outlook.

The FDA's approval of the first CRISPR-Cas9–based gene therapy marked a major milestone in biomedicine, validating genome ...

A 26-year-old Downriver man seen at Children’s Hospital of Michigan in Detroit since infancy for a rare bleeding disorder in ...

Sarepta Therapeutics (SRPT) stock in focus as report says company hired a Trump-linked lobbying firm after deaths linked to ...

Uppsala University Hospital-led investigators report that gene-edited donor islet cells survived 12 weeks inside a man with ...

Dr Vinay Prasad was asked to step down as the FDA's top vaccine official following several controversies during his brief tenure.

Connecticut is now participating in a new federal program aimed at expanding treatment access for patients with sickle cell ...

A combo of cancer drugs letrozole and irinotecan has been identified — via a complex AI-driven study — to reverse memory ...

At only 6 months, Gerard and Kaitlin Norton’s daughter Madeline was diagnosed with PKAN, a rare neurodegenerative disorder ...

Ocugen says it is on track to reshape the market for gene therapies against eye disorders over the next three years, by ...