genetic diseases

Person lacing up running shoe
Research

Atalanta’s RNA epilepsy asset suppresses seizures in mice

Mice with a rare form of epilepsy given a single injection of the drug candidate had 84% fewer seizures than control mice over the next six months.
Darren Incorvaia Apr 10, 2025 5:30am
lighthouse in stormy night

Gene therapy biotech secures $150M for eye disease candidates

Apr 2, 2025 7:30am
spiral stairs downward end dark black

UPDATE: Another NIH leader is out amid Trump admin shake-up

Mar 21, 2025 10:44am
Ship sailing through stormy seas

Arbor sails into rocky gene editing waters with $74M

Mar 18, 2025 6:30am
Gene editing CRISPR gene therapy

Beam gene therapy passes early safety test in AATD

Mar 10, 2025 12:22pm
cows

Gene therapy spares mice and calf from maple syrup urine disease

Mar 4, 2025 5:15am