Drugs in Dev.
Genetic Disease
Phase II
China 
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Lead Product(s) : AGA2115
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
AGA2115 Trial in Adults with Osteogenesis Imperfecta COL1A1/COL1A2 Variations
Details : AGA2115 is a drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Osteogenesis Imperfecta.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
July 14, 2025
Lead Product(s) : AGA2115
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Equecabtagene Autoleucel
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Study of Eque-cel CAR-T Therapy in Newly Diagnosed Severe AL Amyloidosis
Details : Equecabtagene Autoleucel is a Cell and Gene Therapy drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Immunoglobulin Light-chain Amyloidosis.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
July 09, 2025
Lead Product(s) : Equecabtagene Autoleucel
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : CM336
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Efficacy & Safety of CM336 Injection in Treatment of Light-Chain Amyloidosis
Details : CM336 is a drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Immunoglobulin Light-chain Amyloidosis.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
June 26, 2025
Lead Product(s) : CM336
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Plonmarlimab
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Plonmarlimab Safety, PK, Tolerability & Efficacy in HLH/MAS Patients
Details : Plonmarlimab is a Antibody drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Lymphohistiocytosis, Hemophagocytic.
Product Name : Undisclosed
Product Type : Antibody, Unconjugated
Upfront Cash : Inapplicable
June 24, 2025
Lead Product(s) : Plonmarlimab
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ART001
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
AccurEdit's ART001 Becomes first Gene Editing Therapy in China with RMAT Status
Details : ART001 is the first gene editing product from China to receive an FDA RMAT designation, highlighting the FDA's recognition of its innovative nature and clinical potential.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
May 28, 2025
Lead Product(s) : ART001
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ART001
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : ART001 is an in vivo gene editing product delivered by LNP for the treatment of Transthyretin amyloidosis (ATTR).
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
May 28, 2025
Lead Product(s) : ART001
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : BW-20805
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Phase 2 in Adult Subjects With Hereditary Angioedema
Details : BW-20805 is a Oligonucleotide drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Angioedemas, Hereditary.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
February 26, 2025
Lead Product(s) : BW-20805
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : JMKX003142
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : JMKX003142 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Polycystic Kidney, Autosomal Dominant.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
January 30, 2025
Lead Product(s) : JMKX003142
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : KN057
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
KN057 Multiple Dose Study in Patients with Hemophilia a or Hemophilia B with or Without Inhibitors
Details : KN057 is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Hemophilia A.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
December 24, 2024
Lead Product(s) : KN057
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Navepegritide
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Once-Weekly TransCon™ CNP Achieved Primary Efficacy Objective in Accomplish China Phase 2 Trial
Details : TransCon CNP (c-type natriuretic peptide prodrug) is a FGFR 3 inhibitor. It is being evaluated for the treatment of children with achondroplasia (ACH) aged 2 to 10 years.
Product Name : TransCon CNP
Product Type : Peptide, Unconjugated
Upfront Cash : Inapplicable
November 16, 2023
Lead Product(s) : Navepegritide
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
