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VSA 012

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Drug Profile

VSA 012

Alternative Names: VSA-012

Latest Information Update: 12 Feb 2026

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At a glance

  • Originator Visirna Therapeutics
  • Class Small interfering RNA
  • Mechanism of Action Complement factor B inhibitors
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Phase I Paroxysmal nocturnal haemoglobinuria

Most Recent Events

  • 06 Dec 2025 Phase-I clinical trials in Paroxysmal nocturnal haemoglobinuria (Treatment-naive) in China (SC) (NCT06848296)
  • 06 Dec 2025 Interim adverse event, pharmacodynamic and efficacy data from a phase Ib trial in Paroxysmal nocturnal hemoglobinuria presented at the at the 67th American Society of Hematology Annual Meeting and Exposition (ASH 2025)
  • 27 Feb 2025 Visirna Therapeutics plans to initiate a phase Ib trial for Paroxysmal Nocturnal Hemoglobinuria (NCT06848296)

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