uniQure releases new data from Spinocerebellar Ataxia Type 3 gene therapy study – Ataxia

uniQure releases new data from Spinocerebellar Ataxia Type 3 gene therapy study

uniQure releases new data from Spinocerebellar Ataxia Type 3 gene therapy study

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


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