DB18594InvestigationalGene Therapies
NGN-401 is a recombinant serotype 9 adeno-associated virus (AAV) encoding an intron encoding a miRNA generating sequence, complementary miRNA binding sites and a full-length human methyl cytosine binding protein 2... Developed by Neurogene Inc., it is being investigated for the treatment of Rett syndrome.
- Also known as
- Recombinant serotype 9 adeno-associated virus (AAV) encoding an intron encoding a miRNA generating sequence, complementary miRNA binding sites and a full-length human methyl cytosine binding protein 2 (MECP2)
- Code names
- NGN-401
What you can answer from here — as of November 20, 2025
Which companies are running trials of NGN-401, in which indications and phases, and which of those programs are still active?
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