This segment focuses on the research and development of small molecule therapeutics for neurological disorders caused by nucleotide repeat expansions. The primary focus is on developing treatments for Friedreich Ataxia (FA) and Myotonic Dystrophy Type-1 (DM1). Research and development activities include preclinical studies, clinical trials, and the utilization of the GeneTAC™ platform to identify and develop drug candidates. The company is actively involved in clinical trials for FA, aiming to improve neurological function and slow disease progression. Market positioning is centered on addressing unmet medical needs in rare neurological diseases. Future opportunities include expanding the pipeline to other nucleotide repeat expansion disorders and securing regulatory approvals for commercialization. Partnerships and collaborations are essential for clinical trial execution and access to patient populations.

This segment encompasses the ongoing development and refinement of the GeneTAC™ (Gene Targeted Chimera) platform. This platform is a core technology used to discover and develop small molecule therapeutics that target the underlying genetic causes of nucleotide repeat expansion disorders. Research activities include the design, synthesis, and testing of novel compounds, as well as the optimization of delivery mechanisms. The platform's application extends to various therapeutic areas, including Fragile X syndrome, spinocerebellar ataxias, and Huntington's disease. The patient impact is significant, as the platform aims to provide disease-modifying therapies for currently untreatable conditions. Market positioning is based on the platform's potential to address a broad range of genetic diseases. Future opportunities include expanding the platform's capabilities and securing intellectual property protection. This segment is crucial for the company's long-term growth and success.