Drugs in Dev. Genetic Disease
IND Enabling
China 
Lead Product(s) : GEN6050X
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
GenAssist’s GEN6050X IND Cleared by FDA for Duchenne Muscular Dystrophy
Details : GEN6050X, a globally first-in-class base editing drug, which is currently being evaluated for the treatment of duchenne muscular dystrophy.
Product Name : GEN6050X
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
March 06, 2025
Lead Product(s) : GEN6050X
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : GEN6050
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : 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.
Product Name : GEN6050
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
June 05, 2023
Lead Product(s) : GEN6050
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : RAG-06
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : SDIC CS Capital
Deal Size : $30.0 million
Deal Type : Series A Financing
Details : 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.
Product Name : RAG-06
Product Type : Oligonucleotide
Upfront Cash : Undisclosed
January 27, 2022
Lead Product(s) : RAG-06
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : SDIC CS Capital
Deal Size : $30.0 million
Deal Type : Series A Financing
Lead Product(s) : glucocerebrosidase
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : 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.
Product Name : CAN103
Product Type : Enzyme
Upfront Cash : Inapplicable
October 20, 2021
Lead Product(s) : glucocerebrosidase
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : glucocerebrosidase
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : WuXi Biologics
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : 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.
Product Name : CAN103
Product Type : Enzyme
Upfront Cash : Inapplicable
February 08, 2021
Lead Product(s) : glucocerebrosidase
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : WuXi Biologics
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : VG901
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : ViGeneron
Deal Size : Undisclosed
Deal Type : Partnership
Details : 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.
Product Name : VG901
Product Type : Cell and Gene therapy
Upfront Cash : Undisclosed
January 12, 2020
Lead Product(s) : VG901
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : ViGeneron
Deal Size : Undisclosed
Deal Type : Partnership
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