DB18616ExperimentalGene Therapies
AAV9/MFSD8 is a gene therapy developed by Neurogene Inc. It is an adeno-associated virus serotype 9 (AAV9) vector with an engineered transgene encoding the human CLN7/MFSD8 gene for expression of... It was investigated for the treatment of neuronal ceroid lipofuscinosis type 7.
- Also known as
- An adeno-associated virus serotype 9 (AAV9) vector with engineered transgene encoding the human CLN7/MFSD8 gene for expression of active human major facilitator superfamily domain containing 8
Which companies are running trials of AAV9/MFSD8, in which indications and phases, and which of those programs are still active?
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