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 ...

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

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

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 ...

Analysts appear optimistic for Intellia’s gene editor nex-z, which showed a greater serum TTR reduction than Alnylam’s ...

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 ...

AstraZeneca has selected a candidate to progress in the type 1 diabetes (T1D) Treg cell therapy programme, as per an 18 ...

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...

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