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CANbridge Reports Data From Preclinical Test of Gene Therapy for Spinal Muscular Atrophy

publication date: May 18, 2023

Beijing CANbridge Pharma reported positive data from its second-gen gene therapy for spinal muscular atrophy in preclinical tests. In a mouse model of SMA, a novel second-gen hSMN1-AAV gene therapy vector improved life span and motor function better than the benchmark vector, The therapy was delivered via intracerebroventricular (ICV) injection, consisting of an endogenous SMN1 promoter and codon-optimized human SMN1 transgene. CANbridge, which developed the novel therapy together with UMass Chan Medical School, develops rare disease and rare oncology therapies. More details....

Stock Symbol: (HK: 1228)

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