CANbridge DMD gene therapy

CANbridge DMD gene therapy is an investigational, preclinical-stage gene therapy being developed by CANbridge Pharmaceuticals (Beihai Kangcheng) for the treatment of Duchenne Muscular Dystrophy (DMD) and other dystrophinopathies. The program utilizes an adeno-associated virus (AAV) vector to deliver a functional version of the dystrophin gene (or a miniaturized micro-dystrophin variant) to muscle cells. By restoring the expression of the dystrophin protein, which is deficient or absent in patients with DMD, the therapy aims to stabilize the muscle cell membrane (sarcolemma), prevent progressive muscle fiber degeneration, and improve motor function. The development program leverages advanced AAV capsid technology, potentially through collaborations with academic institutions like UMass Chan Medical School, to optimize muscle-specific delivery and minimize potential immunogenicity.

Other names

Beihai Kangcheng DMD gene therapyCANbridge Duchenne Muscular Dystrophy gene therapy