ABO-202

ABO-202 is an investigational, one-time gene therapy designed to treat CLN1 disease (also known as infantile Batten disease), a rapidly progressing and fatal lysosomal storage disorder with no approved therapies. The therapy uses a self-complementary adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the palmitoyl-protein thioesterase 1 (PPT1) gene to cells in the central nervous system and peripheral organs. By restoring PPT1 enzyme activity, ABO-202 addresses the underlying genetic and enzymatic deficiency that leads to abnormal accumulation of lipopigments, neuroinflammation, and neurodegeneration characteristic of CLN1 disease. Preclinical studies have shown that ABO-202 normalizes survival rates and improves neurological function in animal models. The therapy can be administered intravenously or intrathecally, with combination administration showing improved efficacy over either route alone[1][3][5][7].

Other names

infantile neuronal ceroid lipofuscinosis gene therapyinfantile Batten disease gene therapyCLN1 disease gene therapyCLN-1 disease gene therapyCLN 1 disease gene therapy