First-in-Class Vectorized RNAi for Facioscapulohumeral Muscular Dystrophy (FSHD)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. There is a significant unmet need among 40,000 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). The second generation myotropic capsid enables E13 vg/kg systemic dosing, and the proprietary miRNAs leverage innate cellular machinery while continuously processing 100s. SPRINT™ (Sequence Prioritization for RNA Interference) is a proprietary engine designed for rapid evaluation with an emphasis on translational fidelity. This algorithm identifies promising constructs for a gene of interest and the automated process ranks key measures of viability. The GOI silences absent exogenous protein expression, while its small size addresses the AAV packaging constraints. As the AAV effectively delivers genetic payloads, the robust datasets validate the benefit/risk profile. The next-generation capsids provide an opportunity for preferential dosing and biodistribution. Because it’s founded on nature’s highly active gene regulation biology, this gene therapy is durable and reduces immunogenicity. |
![]() Tech ID2024-024 College(None) Licensing ManagerInventorsCategories(None) |