gene therapy

An artists depiction of DNA molecules made up of tiny particles
Biotech

RegenXBio links DMD gene therapy to improved outcomes

The updated phase 1/2 data provide evidence the pivotal gene therapy dose improves functional outcomes in older boys with the disease.
Nick Paul Taylor Jun 5, 2025 8:52am
Make a delay and accumulation of outstanding tasks Full stop

Rocket crashes as gene therapy patient dies, FDA imposes hold

May 27, 2025 9:49am
a lot of scissors

Gene editor shelves sole clinical asset, lays off 25% of staff

May 19, 2025 2:28pm
Close-up of human eye

Atsena gene therapy hits safety goals, closes retinal splits

May 19, 2025 12:59pm
gene therapy AAV adeno-associated viruses

Neurogene details death in Rett syndrome gene therapy trial

May 16, 2025 3:00pm
Rocket with heart on blue background

Rocket's gene therapy sets new efficacy bar in ph. 1 heart study

May 16, 2025 10:36am