AAV.GTX-ADAR2

AAV.GTX-ADAR2 is an adeno-associated virus (AAV) vector-based gene therapy designed to deliver the ADAR2 (adenosine deaminase acting on RNA 2) gene. Developed by ONODERA GT Pharma, it is specifically targeted at the treatment of sporadic amyotrophic lateral sclerosis (sALS). The therapeutic rationale is based on the observation that motor neurons in patients with sALS often exhibit a downregulation of ADAR2, which leads to the failure of RNA editing of the GluA2 subunit of AMPA receptors. This failure results in the formation of calcium-permeable AMPA receptors, causing excitotoxic motor neuron death. By restoring ADAR2 expression via intrathecal or medullary cavity administration, AAV.GTX-ADAR2 aims to normalize GluA2 RNA editing and prevent further neurodegeneration.

Other names

GTX-ADAR2GTX-ADAR-2GTX-ADAR 2ADAR2-expressing AAVADAR-2-expressing AAVADAR 2-expressing AAV