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Abeona Therapeutics, Inc.

Drug Names(s): Recombinant AAV9 expressing CLN3, AAV CLN3

Description: ABO-201 is an adeno-associated virus (AAV) gene therapy program expressing ceroid-lipofuscinosis, neuronal 3 (CLN3) for the treatment of the juvenile form of NCL, called Batten disease (some also use Batten disease to refer to all forms of NCL). Mutation in the CLN3 is a major cause of the juvenile form, but its function is not entirely clear.

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