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CGB1001

Development stage: Preclinical
Lead developer: ChainGen Bio
Modality: Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antibody Conjugates → Antibody-Based Therapeutics
Administration: Intravenous

Overview

CGB1001 is an antibody oligonucleotide conjugate developed by ChainGen Bio (Shanghai) for the treatment of myotonic dystrophy type 1 (DM1). The drug is designed to target diseases of the nervous system, congenital disorders, and skin and musculoskeletal diseases. Its precise mechanism of action and molecular targets have not been publicly disclosed. As of March 2025, CGB1001 remains in preclinical development with at least one open-label, dose-escalation clinical study initiated to evaluate its safety and tolerability in patients with DM1[1][3][5].

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