Cure Rare Disease (CRD) announced it was awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of an antisense oligonucleotide therapy for spinocerebellar ataxia type 3 (SCA3), a neurodegenerative disorder with no current treatment.
Cure Rare Disease launched its SCA3 program in 2021 to develop an antisense oligonucleotide (ASO) therapy, with initial funding support provided by Gregory Klassen, a patient living with SCA3.
In 2022 and 2023, in partnership with Leiden University Medical Center and the Cure Rare Disease team, several in vivo studies were conducted in a disease mouse model, demonstrating functional improvement. A clinical candidate was identified through non-GLP toxicology studies conducted with Charles River Labs. In 2024, a Type B pre-IND meeting with the Food and Drug Administration (FDA) provided regulatory guidance ahead of manufacturing scale-up and clinical trial design.
With CIRM funding, Cure Rare Disease will complete manufacturing scale-up, conduct IND-enabling toxicology studies and submit an investigational new drug (IND) application to initiate early clinical trials. The trials will be led by Principal Investigator Dr. Susan Perlman at UCLA.
KEY QUOTE:
“This program is a powerful example of what can be achieved when patients, organizations, researchers and governing bodies come together with a shared mission. From Greg Klassen’s initial support to our collaborations with leading scientists and clinicians, industry partners and regulatory guidance from the FDA, the development of a therapy for SCA3 has been a truly unified effort. We are incredibly appreciative of CIRM’s support in helping us take this program into the clinic, proving that rare disease treatments can be brought to patients through our model of collective dedication and innovation.”
– Cure Rare Disease CEO and founder Richard Horgan
