Novel treatments for rare diseases and gene therapies reported progress. Solid Biosciences received FDA and Health Canada clearance to begin Phase I trials for SGT-501 gene therapy targeting catecholaminergic polymorphic ventricular tachycardia. AAV-OTOF gene therapy demonstrated hearing restoration across children and adults with autosomal recessive deafness, with notable safety and efficacy in a single-arm trial. Additionally, Chugai and Gero entered a research collaboration aiming to develop antibodies against age-related diseases using AI-discovered targets. These initiatives reflect continued focus on orphan indications with high unmet needs.