GTRI executes option agreement with major Japanese drugmaker on ALS

Japanese drug major Astellas Pharma (TYO: 4503) has entered into an option agreement with Gene Therapy Research Institution (GTRI) for the exclusive negotiation of rights to global development and commercialization of the gene therapy program GT0001X for the treatment of sporadic amyotrophic lateral sclerosis (ALS).

ALS is a neurodegenerative disorder that selectively impairs motor neurons, gradually leading to a decrease and loss in strength of muscles in the limbs, throat, tongue, and those required for respiration. There are an estimated 55,000 patients with ALS in Japan, the USA, and the EU5 countries (UK, Germany, France, Spain, and Italy). The prevalence rate in patients ³ 60 years of age is as many as 20 to 30 per 100,000 people. The cause of ALS is unknown and no effective therapy to stop the progression of this disease has been established. In patients with sporadic ALS, which accounts for ³ 90% of the whole ALS population, decreased activity of adenosine deaminase acting on RNA 2 (ADAR2)2, an RNA-editing enzyme, has been reported to be a possible cause of the disease.

GT0001X is a modified adeno-associated virus (mAAV) vector expressing human ADAR2. In conditional ADAR2 knockout mice, degeneration of anterior horn cells and the resulting motor dysfunction were prevented by mAAV-mediated delivery of ADAR2. Currently, GTRI has been developing GT0001X in the pre-clinical stage for the treatment of sporadic ALS. In the future, the clinical efficacy and safety will be investigated.