Cure Rare Disease Secures $5.69M for SCA3 Therapy Development

Cure Rare Disease (CRD) has received a $5.69 million grant from CIRM to advance the development of an antisense oligonucleotide therapy for untreatable neurodegenerative disorder SCA3. The funding will support manufacturing scale-up, toxicology studies, and early clinical trials. SCA3, impacting one to five in 100,000 individuals, stems from mutations in the ATXN3 gene, leading to toxic accumulation of trinucleotide repeats.