TGT-001

TGT-001 is an investigational CRISPR/Cas9-based gene editing therapy being developed by Toolgen in collaboration with the Charcot-Marie-Tooth Research Foundation (CMTRF) for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A). CMT1A is a rare genetic neurological disorder caused by a duplication of the *PMP22* gene, which leads to the overexpression of peripheral myelin protein 22 and the subsequent demyelination of peripheral nerves. TGT-001 utilizes CRISPR/Cas9 technology to specifically target and reduce the expression of the *PMP22* gene, aiming to restore normal myelin sheath formation and improve nerve function. The therapy is designed to address the underlying genetic cause of the disease rather than just managing symptoms.