[{"orgOrder":0,"company":"WuXi Biologics","sponsor":"ViGeneron","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"ViGeneron and WuXi Advanced Therapies Enter Strategic Manufacturing Partnership For Next-Generation Ophthalmic Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"WuXi Biologics"},{"orgOrder":0,"company":"EdiGene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"China NMPA Approves EdiGene\u2019s IND Application of CRISPR\/Cas 9 Gene-Editing Therapy, ET-01 to Treat Transfusion Dependent \u00df-Thalassemia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"CHINA","productType":"Cell and Gene 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Treatment of Gaucher Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"CMG-SDIC Capital","pharmaFlowCategory":"D","amount":"$60.0 million","upfrontCash":"Undisclosed","newsHeadline":"Neurophth Raised Over $60 Million USD in Series-C Financing to Advance Gene Therapies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Neurophth Therapeutics"},{"orgOrder":0,"company":"Ractigen Therapeutics","sponsor":"SDIC Venture Capital","pharmaFlowCategory":"D","amount":"$30.0 million","upfrontCash":"Undisclosed","newsHeadline":"Ractigen Therapeutics Closes an Additional $30 Million in Series A+ Funding to Push Development of Its RNAa Platform","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Ractigen Therapeutics"},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Neurophth Receives IND Clearance from FDA for AAV-ND1 Gene Therapy of LHON","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Neurophth Therapeutics"},{"orgOrder":0,"company":"Mabwell","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mabwell Announces the NMPA Approval Of 9MW3011 (FIC) for IND","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Mabwell"},{"orgOrder":0,"company":"Suzhou GenAssist Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Suzhou GenAssist Therapeutic Co.,Ltd Recently Announced that Its First Base Editing Product, GEN6050, Has Submitted a Pre-IND Application to The FDA and Has Been Accepted","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New 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Find Clinical Drug Development Pipelines & Deals | PipelineProspector
GEN6050 is an in vivo base editing drug that targets exon 50 skipping in the Duchenne muscular dystrophy (DMD) gene. It is designed to restore the expression of dystrophin protein by base editing.
9MW3011 is monoclonal antibody which can upregulate the level of hepcidin expressed by hepatocytes through specific binding, inhibit the absorption and release of iron, and lower the serum iron level, thus regulating the iron homeostasis in vivo.
Investigational NFS-02, a novel recombinant adeno-associated viral serotype 2 vector (rAAV2) containing a codon-optimized of NADH-dehydrogenase subunit 1 gene, is a novel ophthalmic injection that is being developed for the treatment of Leber hereditary optic neuropathy.
Ractigen will use this capital to advance several programs from preclinical to clinical phase, as well as scale-up its oligonucleotide manufacturing capabilities including its lead product RAG-06.
The financing will be used to progress its lead clinical program in LHON to ex-China, enrich R&D pipeline and programs through business development and continue to expand the international standard gene therapy manufacturing platform.
CAN103 is an enzyme replacement therapy (ERT) being developed by CANbridge as part of its rare disease partnership with WuXi Biologics (2269.HK) for the long-term treatment of adults and children with Gaucher disease, Types I and III.
CAN103 is an enzyme replacement therapy (ERT) being developed by CANbridge as part of its rare disease partnership with WuXi Biologics (2269.HK) for the long-term treatment of adults and children with Gaucher disease, Types I and III.
ET-01 is an autologous, in vitro gene editing therapy product in the research and development stage. The clinical trial approved this time is a multi-center, open, single-arm clinical study.
WuXi ATU will accelerate the development of VG901 by manufacturing, testing and making available the clinical trial grade treatment while leveraging its trusted, world-class AAV Suspension and Plasmid DNA platforms.