CAN203

CAN203 is a second-generation, adeno-associated virus (AAV)-based gene therapy being developed for the treatment of spinal muscular atrophy (SMA). It delivers a codon-optimized human SMN1 transgene under the control of an endogenous hSMN1 promoter to restore the production of the survival motor neuron (SMN) protein. Developed by CANbridge Pharmaceuticals in collaboration with UMass Chan Medical School, the therapy is designed for intracerebroventricular (ICV) administration, which targets the central nervous system directly. Preclinical studies indicate that this approach may allow for lower systemic doses, potentially reducing liver toxicity and improving the safety profile compared to existing benchmark therapies.