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VSA012
- Development stage
- Phase 1
- Lead developer
- Visirna Therapeutics
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Injection
01
Overview
VSA012 is an investigational drug developed by Visirna Therapeutics for the treatment of diseases associated with abnormal activation of the complement system, such as paroxysmal nocturnal hemoglobinuria (PNH). It is a new molecular entity and functions as a complement activation modulator. The drug is currently being evaluated in Phase I clinical trials to assess its safety, tolerability, pharmacokinetics, and pharmacodynamics in both healthy volunteers and patients with PNH. The mechanism of action involves targeting components of the complement system to modulate its activity, aiming to address conditions where dysregulation leads to disease.
- Other names
- VSA 012VSA012VSA-012
02
Targets
Complement (Complement system)
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