Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
ER2001
- Development stage
- Phase 2
- Lead developer
- ExoRNA Bioscience
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
ER2001 is an investigational gene therapy designed as a genetic circuit (plasmid) encoding both a neuron-targeting rabies virus glycoprotein (RVG) tag and an HTT siRNA. After intravenous administration, the plasmid reprograms liver cells to transcribe and self-assemble HTT siRNA into RVG-tagged exosomes. These exosomes circulate systemically and deliver the HTT siRNA specifically to neurons in the cortex and striatum, targeting mutant huntingtin protein expression via RNA interference. Preclinical studies have shown that ER2001 lowers mutant huntingtin levels, improves behavioral deficits, and ameliorates neuropathology in models of Huntington's disease. The drug is being developed by ExoRNA Bioscience Nanjing for early manifest Huntington's disease[5][7].
- Other names
- ER 2001ER2001ER-2001
02
Targets
HTT mRNA (HTT / Huntingtin gene)
Beyond the preview
Go deeper on ER2001.
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 ER2001.