Drug intelligence / Profile preview

RG002C0106

Development stage
Phase 2
Lead developer
Rigerna Therapeutics
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
01

Overview

RG002C0106 is a liver-targeted small interfering RNA (siRNA) drug developed by Rigerna Therapeutics and Suzhou Xuanjing Biotechnology for the treatment of complement-mediated disorders, particularly glomerular diseases such as C3 glomerulopathy and IgA nephropathy. The drug acts as an inhibitor of complement component C3 expression via RNA interference, aiming to reduce pathological complement activation in kidney diseases. It is administered subcutaneously and represents a new molecular entity currently in early clinical development, with Phase 1 trials underway to assess its safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy adults[1][2][3][4][6].

02

Targets

C3

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