Neurogene (NGNE) has released an update.
Neurogene Inc. has received RMAT designation from the FDA for their investigational gene therapy NGN-401, aimed at treating Rett syndrome, based on promising preliminary clinical trial results. This designation, along with inclusion in the FDA’s START Pilot Program, could lead to accelerated approval pathways, underscoring the therapy’s potential to meet the unmet needs of patients with this condition.
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