Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
Rhes siRNA
- Development stage
- Preclinical
- Lead developer
- Johns Hopkins University
- 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
- Intracerebral
01
Overview
Rhes siRNA is a small interfering RNA (siRNA) designed to silence the expression of the Rhes protein (Ras homolog enriched in striatum), which is encoded by the *RASD2* gene. Rhes is a striatal-specific small GTPase that has been identified as a key mediator of neurodegeneration in Huntington's disease (HD). It selectively binds to mutant huntingtin (mHtt) and promotes its sumoylation, which enhances mHtt-induced cytotoxicity. Furthermore, Rhes regulates autophagy by interacting with Beclin-1, and its depletion has been shown to decrease autophagy in certain cellular contexts. By reducing Rhes levels, Rhes siRNA aims to mitigate the selective destruction of the striatum and motor dysfunction associated with HD, potentially by restoring autophagic balance and reducing mHtt toxicity.
- Other names
- RASD2 siRNARASD-2 siRNARASD 2 siRNAsiRNA-Rhes
02
Targets
RAS family (RAS small GTPase family)
Beyond the preview
Go deeper on Rhes siRNA.
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 Rhes siRNA.