[{"orgOrder":0,"company":"EdiGene","sponsor":"3H Health Investment","pharmaFlowCategory":"D","amount":"$67.0 million","upfrontCash":"Undisclosed","newsHeadline":"EdiGene Raises Approximately USD 67 Million in Series B Financing to Advance Gene Editing Based Programs into Clinical Stage","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"EdiGene"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"CANbridge Pharmaceuticals Enters into Rare Disease Gene Therapy Research Agreement with UMass Medical School","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CANbridge-UMass Chan Medical School Gene Therapy Research to be Presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CANbridge-UMass Chan Medical School Gene Therapy Research Presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CANbridge-UMass Chan Medical School Gene Therapy Research in Oral Presentation at the European Society of Gene and Cell Therapy (ESGCT) 29th Annual Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"CANbridge Pharmaceuticals"}]
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Study showed that novel second-generation self-complementary AAV9 gene therapy, expressing a codon-optimized human SMN1 gene under control of its endogenous promoter, demonstrated superior safety, potency, and efficacy across several endpoints in an SMA mouse model.
Endogenous human SMN1 promoter-driven gene replacement improves the efficacy and safety of AAV9-mediated gene therapy for spinal muscular atrophy in mice.
Researchers will present findings from an endogenous human SMN1 promoter-driven gene replacement improves the efficacy and safety of aav9-mediated gene therapy for spinal muscular atrophy in mice.
EdiGene has established four gene editing based platforms and is advancing its early stage programs into clinical development for patients with genetic diseases and cancer. Proceeds from the financing will be used to advance the company’s pipeline into clinics.
This collaboration with the Horae Gene Therapy Center at UMass, affords CANbridge the potential to extend the rare disease pipeline by integrating next-generation technology to advance as a leader in the treatment of rare diseases by adeno-associated virus.