CANbridge Pharmaceuticals has exercised its option to secure the exclusive global rights to develop, manufacture and commercialise a novel gene therapy to treat spinal muscular atrophy (SMA) from UMass Chan Medical School.
Animal data presented in 2022 showed that the gene therapy outperformed the benchmark therapy along multiple key endpoints in a mouse model of SMA. It also exhibited much less liver toxicity when administered intravenously.
The benchmark therapy uses a vector similar to that used in the only gene therapy approved for SMA. CANbridge sponsored the research and is evaluating the gene therapy to determine potential additional advantages over the current standard-of-care.
“The novel hSMN1 AAV gene therapy vector, consisting of the endogenous SMN1 promoter and codon-optimised hSMN1, has a remarkably improved potency and safety profile as compared to the benchmark vector, holding great promise for further clinical development,” said Guangping Gao, the Penelope Booth Rockwell Professor in Biomedical Research, Professor of Microbiology & Physiological Systems, Director of the Horae Gene Therapy Center and Co-director of the Li Weibo Institute for Rare Diseases Research.
In addition, the company announced that it has completed the full technology transfer of gene therapy products being developed for the treatment of Fabry and Pompe diseases from LogicBio Therapeutics.
CANbridge also obtained non-exclusive worldwide rights to the LogicBio proprietary manufacturing process for Fabry and Pompe gene therapies.
