Neurogene Inc. has made significant progress in its clinical-stage development of NGN-401 gene therapy for Rett syndrome. The therapy has received RMAT designation from the FDA, recognizing its potential to address unmet medical needs. Additionally, NGN-401 has been selected for the FDA’s START Program, which aims to accelerate its development.
Interim efficacy data from Cohort 1 of the NGN-401 trial is expected to be released in the fourth quarter of 2024, with further data from both low-dose and high-dose cohorts anticipated in the latter half of 2025.
Neurogene also highlighted advancements in developing NGN-101 gene therapy for CLN5 Batten Disease, under Phase 1/2 trials. The company expects to release data from the ongoing trial in the second half of 2025.
Rachel McMinn, Neurogene’s CEO, emphasized the importance of the RMAT and START designations, which provide opportunities for enhanced collaboration with the FDA to expedite NGN-401’s development.
Neurogene has continued to advance the NGN-401 program by dosing the first patient in Cohort 2 and reported favorable preliminary safety profiles. Low-dose NGN-401 has also been well-tolerated by patients in Cohort 1.
Looking ahead, Neurogene anticipates sharing additional clinical data and updates on its pipeline of gene therapy candidates in the coming months and years.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
