rare diseases

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

Clinical setbacks and controversial pricing may have put a dampener on the gene therapy sector, but one of the industry’s pioneers is looking for a way forward.

James Waldron Oct 7, 2025 10:00am

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

end of the road road block shut down closed blocked

FDA snubs Fortress' Zydus-partnered Menkes disease candidate

Oct 1, 2025 12:00pm

Applied Tx's stock drops on non-update for rare disease asset

Sep 29, 2025 10:57am

Ionis rare disease candidate ekes out phase 3 win

Sep 22, 2025 10:46am