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ACVR1 small interfering RNA
- Development stage
- Preclinical
- Lead developer
- UMass Chan Medical School
- 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
- Intramuscular, Subcutaneous
01
Overview
ACVR1 small interfering RNA (siACVR1) is an allele-selective, chemically modified small interfering RNA (siRNA) designed to target and silence the mutant ACVR1 gene (specifically the R206H mutation) while sparing the wild-type ACVR1 allele. Developed by researchers at UMass Chan Medical School, this therapeutic candidate is conjugated to docosanoic acid (DCA) to facilitate efficient, non-toxic delivery and sustained gene silencing in extrahepatic tissues, particularly skeletal muscle. It is being investigated for the treatment of fibrodysplasia ossificans progressiva (FOP) to prevent trauma-induced heterotopic ossification (HO). In preclinical studies, it has been evaluated both as a monotherapy and as part of a combinatorial approach (including a divalent siRNA construct) that simultaneously targets ACVR1 and the pro-inflammatory cytokine interleukin-1 beta (IL-1β).
- Other names
- ACVR1 siRNAACVR-1 siRNAACVR 1 siRNADCA-conjugated siACVR1docosanoic acid-conjugated ACVR1 siRNAdocosanoic acid-conjugated ACVR1 small interfering RNA
02
Targets
ACVR1 (Activin receptor type I)
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