We are pleased to advise that Neurogene Inc. have announced that enrollment is now complete for its Phase 1/2 investigational trial of NGN-101 Gene Therapy for the treatment of CLN5 Batten disease, with a total of six patients (Clinical trial ID: NCT05228145). Neurogene Inc. unveiled this news in its Second Quarter 2024 Financial Results and Highlights Recent Updates on August 9.
According to the report, Neurogene plans to provide interim clinical data and a regulatory update in the first quarter of 2025.
“Given the rarity of CLN5 Batten disease, FDA alignment on a streamlined registrational pathway will be critical for continued investment in the program,” the report noted.
The BDFA is pleased that this study has hit this milestone and has assured Neurogene of our continuing support and collaboration.
As global patient advocacy organisations for the Batten disease community, we look forward to providing further updates on this study and continue our global collaboration to keep the Batten community informed and to advocate for treatments and cures for Batten disease.
You can read Neurogene Inc.’s full report by clicking here.
If you have any questions about this trial or any other matter relating to research and clinical trials, please contact Dr. Joanna Nightingale, BDFA Head of Scientific Affairs, joannanightingale@bdfa-uk.org.uk
