rare diseases

On target clinical trial success bullseye score
Biotech

Sanofi's $1.7B bet beats CSL's Zemaira in phase 2 face-off

Efdoralprin alfa beat the standard of care on primary and secondary endpoints, positioning the drugmaker to talk to regulators about the next steps.
Nick Paul Taylor Oct 22, 2025 10:35am
purple lightning tornado

Electra shines with $183M fundraise for rare disease trial

Oct 22, 2025 8:26am
graphic art of a right hand signing something

Hansoh, Chugai ink separate and lucrative licensing agreements

Oct 17, 2025 11:04am
An artists impression of DNA The DNA appears to be made up of glowing orbs and appears on a blue background

James Wilson's GEMMABio launches new gene therapy biotech

Oct 7, 2025 10:00am
Tiny green sprout growing from soil

Chiesi fertilizes Arbor's gene therapy with $2B biobucks deal

Oct 6, 2025 7:00am
Space shuttle takes off on background of blue sky

Rocket aborts mission to get rare disease gene therapy approved

Oct 3, 2025 9:30am