SonoThera advanced non-viral ultrasound-mediated gene delivery with a major Series B that raised $125 million, aiming to move its platform into clinical development. The financing targets two lead programs—Duchenne muscular dystrophy and autosomal dominant polycystic kidney disease—seeking to address limitations of viral vectors and lipid nanoparticles, including safety, repeat dosing, and manufacturing complexity. Also in gene therapy, Chinese investigators reported safety and feasibility for a subretinal AAV8 approach targeting RS1 in pediatric X-linked retinoschisis (XLRS). Published in the New England Journal of Medicine, the study supports the potential shift from observation to early molecular intervention for a retinal disease with no approved underlying-gene therapy, and it frames ongoing late-stage efforts such as Atsena Therapeutics’ ATSN-201.