This web site is part of the Business Intelligence Division of Informa PLC
This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.
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.
Additional information available to subscribers only: