Anc80L65-ARSA

Anc80L65-ARSA is an investigational **AAV gene therapy** developed by Affinia Therapeutics for **metachromatic leukodystrophy**. It uses Affinia's synthetic ancestral **Anc80L65** adeno-associated virus capsid to deliver a functional **arylsulfatase A** transgene to the central nervous system after **intrathecal** administration by lumbar puncture. The therapeutic rationale is to restore ARSA enzyme expression in patients with ARSA deficiency, thereby reducing sulfatide accumulation and potentially slowing or preventing progressive neurodegeneration in the central and peripheral nervous systems. It is intended as a **one-time** gene replacement approach for this rare, progressive, and fatal lysosomal storage disorder.

Other names

ARSA gene therapyAnc80L65 capsid ARSA gene therapyAnc-80L65 capsid ARSA gene therapyAnc 80L65 capsid ARSA gene therapy