Neurogene has dropped plans to keep studying the high dose of its Rett syndrome gene therapy because of an adverse event that ...

Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...

In another sign of biopharma's waning interest in the traditional ex vivo gene therapy approach, CSL Behring is shutti | CSL ...

Regenxbio reported improved muscle function in boys with Duchenne muscular dystrophy using their gene therapy RGX-202. The ...

In an update posted today, Neurogene said that the "emerging treatment-related serious adverse event (SAE)" was a case of ...

Neurogene is proceeding with a scaled-back study of its proposed NGN-401 gene therapy for the rare genetic neurological disorder Rett syndrome after a serious adverse event left a study participant in ...

Intellia presented data from its Phase I study of NTLA-2001 at the 2024 American Heart Association scientific meeting in ...

Wilson celebrates the first U.S. patient to complete gene therapy for sickle cell, but says advocacy must continue.

Researchers have developed a dual-gene therapy approach to treat Usher syndrome type 1F, a rare condition causing deafness and progressive blindness.

At Neurogene, the developer of a gene therapy for Rett syndrome, expediency seems to be the priority, @adamfeuerstein writes.

Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...

The treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have ...