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BPR-30221616

Development stage: Phase 1
Lead developer: Chengdu Brilliant Pharmaceutical
Modality: Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Subcutaneous

Overview

BPR-30221616 is a small interfering RNA (siRNA) therapeutic developed by Chengdu Brilliant Pharmaceutical (also referred to as Brilliant Pharmaceutical Co. Ltd.) for the treatment of transthyretin amyloidosis. The drug is currently in Phase 1 clinical trials, with studies focused on evaluating its safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity in healthy subjects. The primary mechanism involves targeting transthyretin protein levels to address amyloid deposition associated with the disease.

Targets

TTR (Transthyretin)

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