A lower-than-expected increase in dystrophin over baseline in the first and lowest-dose cohort of a phase I/II study of ENTR-601-44 in Duchenne muscular dystrophy (DMD) caused shares of Entrada Therapeutics Inc. to plunge more than 57%, despite the cohort meeting the safety and tolerability primary objective.

Apertura Gene Therapy LLC and the TSC Alliance have established a collaboration to advance gene therapy programs designed to treat tuberous sclerosis complex (TSC).

Elaaj Bio, a wholly owned subsidiary of the nonprofit Loulou Foundation, has entered into a partnership with Viralgen Vector Core SL to advance a gene therapy program for CDKL5 deficiency disorder.