NVO-101

NVO-101 is an investigational small interfering RNA (siRNA) therapeutic being developed by Nervosave Therapeutics in collaboration with the Charcot-Marie-Tooth Research Foundation (CMTRF). It is specifically designed to treat Charcot-Marie-Tooth disease type 1A (CMT1A), a rare genetic peripheral neuropathy caused by a duplication of the PMP22 gene. This duplication leads to the overexpression of Peripheral Myelin Protein 22 (PMP22), which disrupts the formation and maintenance of the myelin sheath. NVO-101 utilizes RNA interference (RNAi) to specifically target and degrade PMP22 messenger RNA (mRNA), thereby reducing protein levels toward a normal physiological range to restore nerve function and slow disease progression. The drug is currently in Phase 1 clinical development.