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ALN-HTT02
- Development stage
- Phase 1
- Lead developer
- Alnylam Pharmaceuticals
- 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, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intrathecal
01
Overview
ALN-HTT02 is an investigational small interfering RNA (siRNA) therapeutic developed for the treatment of Huntington’s disease. It is designed to be administered intrathecally and leverages a C16-siRNA delivery platform to enhance uptake in the central nervous system. The drug targets a conserved sequence within exon 1 of the huntingtin (HTT) gene’s messenger RNA, promoting its degradation via RNA interference and thereby reducing production of both wild-type and mutant forms of HTT protein. By lowering all forms of HTT protein—including those with expanded polyglutamine tracts that drive toxicity—ALN-HTT02 aims to slow or halt disease progression in Huntington’s disease. Preclinical studies have shown broad CNS distribution, durable reduction in HTT levels, and favorable safety profiles in non-human primates. The drug is currently being evaluated in Phase 1 clinical trials for safety, tolerability, pharmacokinetics, and pharmacodynamics.
- Other names
- ALN-HTTALN-HTT02ALN-HTT-02ALN-HTT 02
02
Targets
HTT mRNA (HTT / Huntingtin gene)
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