CLN5 Program Update
Neurogene has completed enrolment of Cohorts 1 and 2 in the ongoing Phase 1/2 clinical trial for CLN5 Batten disease, and interim clinical data are expected in the second half of 2024. Neurogene is currently enrolling a final higher dose cohort. In the fourth quarter of 2023, Neurogene completed a positive meeting with the FDA regarding the future potency assay. The FDA accepted Neurogene’s proposed potency assay strategy and provided alignment with the testing approach, which will allow release of all future NGN-101 batches.
To enable a go/no-go decision to advance the program into a registration study, Neurogene is collecting and analysing natural history data for CLN5 Batten disease and planning to request a clinical/regulatory strategy meeting with the FDA in the second half of 2024. The focus of this meeting will be to align with the FDA on the expected clinical requirements to support a streamlined registration pathway, which will be necessary to move this program forward into a pivotal clinical trial.
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