At the 2019 American Academy of Neurology Annual Meeting, which took place at the beginning of May, leading gene therapy company uniQure presented preclinical data on its gene therapy candidate, AMT-150, for the treatment of Spinocerebellar Ataxia Type 3 (SCA3).

SCA3 is caused by mutations in the ataxin-3 gene. Genes are a code made up of the letters A, T, G and C. In people with SCA3, a specific region of the ataxin-3 code contains the letters ‘CAG’ repeated many times. This results in the production of an abnormal and toxic form of the ataxin-3 protein. This leads to brain degeneration that results in movement disorders, rigidity, muscular atrophy and paralysis. There are currently no disease-modifying treatments for patients with SCA3, or medications to slow its progression.

uniQure have developed a gene therapy technology, named AMT-150. Data from preclinical studies carried out in mice showed that a single AMT-150 injection significantly decreased the amount of mutant ataxin-3 in the main parts of the brain affected by the disorder; the cerebellum (up to 53%) and brainstem (up to 65%). These results were confirmed in human cells showing a dose-dependent lowering of mutant ataxin-3.

These results demonstrate that this gene therapy may be a potential future treatment for SCA3. uniQure is currently performing further animal studies to demonstrate the safety and efficacy of AMT-150.

View the original press release here.

Posted on 23/05/2019