On February 6th, the US not-for-profit biotechnology company Cure Rare Disease announced that they have been awarded 5.69 million dollars in funding from the Californian Institute of Regenerative Medicine (CIRM) to advance gene therapy for Spinocerebellar Ataxia Type-3 (SCA3).
Cure Rare Disease launched their SCA3 program in 2021 to develop a gene therapy called an antisense oligonucleotide (ASO) for SCA3. SCA3 is caused by a mutation in the ATXN3 gene, which provides the instructions for a protein called Ataxin-3. Ataxin-3 is needed throughout the body to destroy and remove damaged proteins. In SCA3, sections in the ATXN3 gene are repeated too many times, known as CAG repeats. This leads to the Ataxin-3 protein becoming longer than normal, and accumulating at toxic levels in cells. ASOs work by preventing the gene from forming a protein. Cure Rare Disease has tested their ASO in mouse models of SCA3, showing functional improvement.
Cure Rare Disease will submit an investigational new drug (IND) application to the US drug regulators, the FDA, which if granted will allow them to start phase 1 clinical trials of their gene therapy in people with SCA3 in the US.
Read more here.
Find out more about the Cure Rare Disease SCA3 programme here.
