CANbridge Acquires Global Rights to Rare Disease Therapy from UMass

Beijing CANbridge Pharma exercised an option for global rights to a novel second-gen gene therapy aimed at spinal muscular atrophy (SMA) from UMass Chan medical school. A rare disease/rare oncology company, CANbridge will have rights to develop, manufacture and commercialize the therapy. The company supported the UMass research effort. In late 2022, data was presented from preclinical tests showing the new SMA gene therapy was more effective and caused less liver toxicity than the current therapy. SMA is caused by the lack of a functional motor survival motor neuron 1 (SMN1) gene that results in loss of motor function and death. More details....

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