Towards a gene therapy to treat spinocerebellar ataxia

We have pre-clinical proof-of-concept for a novel and non-obvious use of a type of gene therapy called spliceosome-mediated RNA trans-splicing (SMaRT) to treat a set of rare neurological diseases called the spinocerebellar ataxias. This project seeks funds for IP protection as well as funds to undertake some additional new analysis that is required to make our IP an attractive licensing opportunity.

We have developed a new type of gene therapy called RNA trans-splicing to treat a set of rare neurological diseases called the spinocerebellar ataxias. This project seeks to undertake some complex analysis that is required to complete the proof-of-concept phase.