Cure Rare Disease receives $5.69 million CIRM grant for ASO gene therapy research for SCA3 ataxia
- Márcio
- Feb 8
- 2 min read
Cure Rare Disease (CRD) is a nonprofit biotechnology company focused on developing gene therapies for rare diseases. CIRM, the California Institute for Regenerative Medicine, is a funding agency established in 2004.
See www.cirm.ca.gov.
In 2021, CRD began research to develop an ASO (antisense oligonucleotide) gene therapy for spinocerebellar ataxia type 3, with funding initially provided to Gregory Klassen, a patient with SCA3. During 2022 and 2023, CRD collaborated with the Leiden University Medical Center and conducted several mouse model studies, which yielded positive results. In 2024, CRD held a "Type B pre-IND" meeting with the FDA (Food and Drug Administration), which provided regulatory guidance for scaling up manufacturing and designing clinical trials.
In February 2025, CIRM's financial support for CRD was announced. With this support, Cure Rare Disease will be able to complete manufacturing expansion, conduct toxicology studies, and submit an investigational new drug (IND) application to the FDA, enabling it to begin its first clinical trials. Principal investigator Dr. Susan Perlman will lead the first clinical trials at UCLA. Dr. Perlman is one of today's leading experts on ataxia, with extensive clinical experience.
CIRM's investment in CRD is good news for SCA3 patients. If the ASO therapy that CDR intends to develop proves safe and effective, it could "silence" the allele of the ATXN3 gene that carries the mutation that causes abnormal CAG expansions in a protein, which then becomes toxic and can cause cellular dysfunction or even neuronal death, leading to SCA3 symptoms.
Another ASO gene therapy study is already in a clinical trial (phase 1/2a) that could benefit patients with HD (Huntington's disease), SCA1 ataxia, and SCA3 ataxia (all PolyQ, caused by CAG repeat expansions in different genes). This is the VO659 trial by Vico Therapeutics.
