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Abeona Therapeutics, Inc.
Drug Names(s): ABO202, ABO 202, AAV-CLN1
Description: ABO-202 is an AAV-based CLN1 program gene therapy for treatment of infantile Batten disease (INCL). The gene therapy uses the AIM vector system, a second generation platform of AAV capsids capable of widespread central nervous system gene transfer. The company states that studies indicate that AIM vectors can efficiently and broadly target CNS tissue, and may provide a treatment for patients that have inhibitory antibodies to natural AAV serotypes. Hence it may provide second-generation treatment approaches for patients that have received a previous AAV injection.
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