Visirna Biotech's siRNA Drug VSA012 Granted IND Approval for New Indication in Complement-Mediated Kidney Diseases

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On November 27, according to the announcement on the CDE official website, the IND application for Visirna's VSA012 injection was approved for the treatment of complement-related kidney diseases, including but not limited to lupus nephritis, IgA nephropathy, C3 glomerulopathy, immune complex-mediated membranoproliferative glomerulonephritis, and atypical hemolytic uremic syndrome (aHUS), among others.

Previously, VSA012 Injection has been approved in China for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

VSA012 is Visirna's first double-stranded small interfering RNA (siRNA) developed based on its proprietary intellectual property siRNA technology platform. Conjugated with N-acetylgalactosamine (GalNAc), it specifically targets complement factor B (CFB) mRNA in the liver, inhibiting the expression of CFB protein in the bloodstream. Preclinical data shows that VSA012 can achieve long-term inhibition of CFB mRNA expression in the liver, significantly suppressing CFB protein levels and alternative complement pathway activity in the blood, while demonstrating good safety.

In January this year, VSA012 successfully completed the first dosing of healthy subjects at Peking University Third Hospital. This trial is a Phase I clinical trial with a single-center, randomized, double-blind, placebo-controlled design conducted in healthy adult subjects in China to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VSA012. It is also planned to subsequently explore the efficacy and safety of this drug in patients with paroxysmal nocturnal hemoglobinuria (PNH).

Source of the article: Collation of public materials

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