GT0001X

GT0001X is an adeno-associated virus (AAV) vector-based gene therapy developed by ONODERA GT Pharma for the treatment of sporadic amyotrophic lateral sclerosis (ALS). The therapy utilizes an AAV9 vector to deliver a functional copy of the ADAR2 (adenosine deaminase acting on RNA 2) gene to motor neurons. In sporadic ALS, the downregulation of ADAR2 leads to the failure of RNA editing at the Q/R site of the GluA2 subunit of AMPA receptors. This results in the formation of calcium-permeable AMPA receptors, which cause excitotoxic motor neuron death. By restoring ADAR2 levels, GT0001X aims to normalize GluA2 RNA editing and prevent neuronal degeneration. The program is currently in the regulatory planning stage for conditional and time-limited marketing approval in Japan.

Other names

AAV-ADAR2AAV-ADAR-2AAV-ADAR 2AAV9-ADAR2AAV-9-ADAR2AAV 9-ADAR2