Neurogene Halts Development of Batten Gene Therapy NGN-101 Following FDA Denial

FDA Denial:
Neurogene has discontinued the development of NGN-101, a gene therapy for Batten disease subtype CLN5, after the FDA denied its application for regenerative medicine advance therapy designation13.

Rationale:
The company considered this designation crucial for continued investment in the product’s development1.

Impact:
This decision reflects the challenges in developing treatments for rare diseases like Batten disease, which has seen several gene therapy programs discontinued recently13.

Parallel Developments:
Neurogene is continuing to develop NGN-401, a gene therapy for Rett syndrome, despite encountering a serious adverse event in a high-dose cohort, which has raised concerns12.

Future Plans:
Neurogene plans to enroll more patients in the NGN-401 trial and expects to report updated data in the second half of 2025, with plans for a registrational clinical trial also in the works1.

Sources:

1. https://www.cgtlive.com/view/neurogene-rett-syndrome-gene-therapy-ngn-401-demonstrates-efficacy-in-low-dose-cohort-but-serious-adverse-event-in-high-dose-group-raises-concerns

2. https://ir.neurogene.com/news-releases/news-release-details/neurogene-provides-update-ngn-401-gene-therapy-clinical-trial

3. https://www.fiercebiotech.com/biotech/neurogene-stops-batten-gene-therapy-after-fda-denies-request-dealing-double-dose-bad-news