TVR110

TVR110 is an investigational gene therapy developed by Armatus Bio for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A), a progressive neuromuscular disorder with no approved therapies. It is an adeno-associated virus serotype 9 (AAV9)-delivered engineered microRNA (miRNA) therapy designed to reduce overexpression of peripheral myelin protein 22 (PMP22), the underlying cause of CMT1A. The therapy uses a vectorized RNA interference strategy to bring PMP22 expression down to healthy levels, aiming to repair myelin and restore nerve function. Preclinical studies have shown promising results in normalizing PMP22 levels, improving nerve histology, and restoring functional behavior in animal models. TVR110 is administered as a one-time intrathecal injection and has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA[1][2][3][4][8][9].