Unravel Biosciences and CSNK2A1 Foundation Expand Collaboration to Find Drugs for Okur-Chung Neurodevelopmental Syndrome

Unravel Biosciences, Inc., a company utilizing artificial intelligence (AI) to advance therapeutics for intricate diseases, and the CSNK2A1 Foundation have announced an extension to their existing drug discovery collaboration. This extension aims to categorize patients with Okur-Chung Neurodevelopmental Syndrome (OCNDS) into subgroups based on their anticipated response to specific drugs identified in prior phases of their research.

The partnership initially employed Unravel's proprietary BioNAV™ platform, which analyzed RNA data from cells derived from patients and mouse models to pinpoint potential drug candidates through in silico methods. The study indicated that responses to these predicted drugs might significantly differ depending on the particular CSNK2A1 gene mutation responsible for OCNDS in each patient. The CSNK2A1 Foundation independently verified multiple in silico drug candidates using a Drosophila fruit fly model of OCNDS, conducted by Dr. Clement Chow at the University of Utah, demonstrating potential clinical benefits.

Unravel will now use its rareSHIFT™ discovery services to implement a new project that collects RNA transcriptomes from OCNDS patients and their healthy family members using a proprietary nasal swab technique. The BioNAV™ platform will then process this data to identify drug candidates most likely to be effective for patients with specific genetic mutations. This project will primarily focus on the K198R mutation in the CSNK2A1 gene, a variant known to be prevalent among over 70 individuals registered with the Foundation.

Dr. Richard Novak, Co-Founder and CEO of Unravel, emphasized the importance of this stratified approach, stating that it could enhance the success rate in clinical trials and reduce the time and costs associated with developing effective treatments for OCNDS patients. Gabrielle Rushing, the CSNK2A1 Foundation’s Science Program Director, highlighted the potential for more personalized and effective therapies through this innovative methodology, which moves beyond a one-size-fits-all model.

OCNDS is a rare genetic disorder first described in 2016 and caused by a mutation in the CSNK2A1 gene located on chromosome 20. This syndrome can manifest through a wide array of symptoms including speech difficulties, global developmental delays, epilepsy, autism spectrum disorder characteristics, behavioral issues, hypotonia, feeding problems, and severe sleep disturbances. Currently, there is no available treatment for OCNDS. The overall prevalence of the syndrome remains unclear, although more than 250 individuals have been registered with the CSNK2A1 Foundation globally.

Unravel Biosciences stands out as a rapid prototyping therapeutics company that integrates AI-driven systems biology with swift in vivo screening and clinical validation, bringing unprecedented efficiency to the discovery and development of therapeutic targets. Their proprietary BioNAV™ platform consolidates target and drug discovery, preclinical screening, and patient stratification to develop treatments for complex diseases. This approach has enabled Unravel to initiate four clinical trials in 2024. Among the notable developments from their platform are RVL002, a novel small molecule targeting mitochondrial metabolism, and RVL027, a molecule addressing a unique mechanism to treat dystonias. The rareSHIFT™ program also offers platform access to foundation and biotech partners to expedite and clinically derisk therapeutic developments.

The CSNK2A1 Foundation is dedicated to finding a cure for OCNDS and ensuring affected individuals lead happy and fulfilling lives. Operated and funded by a dedicated team of volunteers, advocates, and researchers, the Foundation has registered over 250 individuals with OCNDS in its patient registry worldwide.