There's a fresh push to edit the genes of human embryos to prevent diseases and enhance characteristics that parents value.

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

Novo Nordisk will advance its acquired ATTR amyloidosis with cardiomyopathy monoclonal antibody (mAb) to Phase III trials.

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

In this week’s edition of InnovationRx, we look at a biotech entrepreneur treating a rare genetic disease, new tech aimed at ...

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 ...

Key Points Non-GAAP earnings per share reached $0.74, surpassing estimates by $0.03 (non-GAAP), but GAAP revenue of $181.1 million missed expectations and rose 1.7% (GAAP) compared to the second ...

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 ...