TS-004703 — For AAVs that harbor gene knockdown machinery, this IP is a novel potency assay development for release testing that creates a HEK293T stable cell line to integrate two genetic components.

TS-004596 — There's a signficiant unmet need among thousands of Americans who struggle with genetic neuromuscular disease; this one-time AAV-delivered proprietary engineered miRNA targets core disease biology, specifically in Facioscapulohumeral Muscular Dystrophy (FSHD). This durable gene therapy reduces immunogenicity by taking advantage of nature’s highly active gene regulation biology.

TS-002175 — The third most common type of muscular dystrophy, Facioscapulohumeral Muscular Dystrophy (FSHD), affects over 870,000 individuals worldwide by causing debilitating pain, muscle weakness, fatigue along with many other symptoms in their face, shoulders, upper arms and lower legs. Researchers at Nationwide Children’s Hospital created a treatment using Salvianolic Acid (SAA) as a drug therapy for neuromuscular disorders including FSHD.