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AGIL-032

Development stage: Preclinical
Lead developer: PTC Therapeutics
Modality: Gene Therapies

Overview

AGIL-032 is a preclinical AAV9 gene therapy initially developed by Agilis Biotherapeutics and subsequently acquired by PTC Therapeutics in January 2022. It is being investigated for the treatment of spinocerebellar ataxia type 2 (SCA2), a rare neurological disorder caused by CAG repeat expansions in the ATXN2 gene. The therapy aims to deliver genetic material to address the underlying genetic defect responsible for the disease.

Targets

ATXN2 (Ataxin-2)

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