Capsida Biotherapeutics to Present New Preclinical Data for Potential Best-in-Class Friedreich's Ataxia Next-Generation Gene Therapy.

Capsida Biotherapeutics has introduced new preclinical data for CAP-004, a potential next-generation gene therapy for Friedreich's ataxia. This therapy targets the central nervous system, cardiac, and sensory manifestations of the disease while minimizing effects on off-target tissues. CAP-004 aims to address the key pathophysiological features of Friedreich's ataxia by increasing frataxin protein expression in the CNS, cardiac tissues, and sensory regions. The data will be presented at the International Congress for Ataxia Research in London. [Extracted from the article]