Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
ARO-HTT
- Development stage
- Preclinical
- Lead developer
- Arrowhead 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
- Administration
- Subcutaneous
01
Overview
ARO-HTT is an investigational small interfering RNA (siRNA) therapeutic being developed for the treatment of Huntington's disease. It is designed to inhibit the expression of the huntingtin (HTT) gene, thereby reducing production of both normal and mutant huntingtin protein. The drug leverages Arrowhead Pharmaceuticals' TRiM platform, which enables targeted delivery of siRNAs to specific tissues, including deep brain structures affected in Huntington’s disease. ARO-HTT is administered via subcutaneous injection and has demonstrated preclinical ability to cross the blood-brain barrier, a significant challenge for CNS-targeted therapies. The goal is to slow or halt progression of Huntington’s by decreasing toxic huntingtin protein accumulation in neurons[1][2][4].
02
Targets
HTT mRNA (HTT / Huntingtin gene)
Beyond the preview
Go deeper on ARO-HTT.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on ARO-HTT.