Clinical trials
Follow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
RUNX1::RUNX1T1 siRNA LNP
- Development stage
- Preclinical
- Lead developer
- Princess Máxima Center for Pediatric Oncology
- 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
- Intravenous
01
Overview
RUNX1::RUNX1T1 siRNA LNP is a research-stage RNA interference (RNAi) therapeutic designed to treat acute myeloid leukemia (AML) harboring the t(8;21) chromosomal translocation. The therapeutic consists of chemically modified small interfering RNAs (siRNAs) that specifically target the fusion breakpoint of the RUNX1::RUNX1T1 mRNA, encapsulated within lipid nanoparticles (LNPs). To ensure selective delivery to leukemic hematopoietic cells, the LNPs are functionalized with a Leu-Asp-Val (LDV) tripeptide ligand that targets the Very Late Antigen-4 (VLA-4) integrin receptor (α4β1) commonly expressed on AML blasts. By silencing the oncogenic fusion transcript, the drug eliminates leukemic stem cell self-renewal and promotes myeloid differentiation. This compound is currently in the preclinical stage, having been developed and evaluated in patient-derived xenograft (PDX) models by academic researchers at the Princess Máxima Center for Pediatric Oncology and collaborating institutions.
- Other names
- LDV-functionalized RUNX1::RUNX1T1 siRNA LNPRUNX1::RUNX1T1 siRNA lipid nanoparticle
02
Targets
α4β1 (Integrin α4β1)RUNX1-RUNX1T1 (Runt-related transcription factor 1; RUNX1 partner transcriptional co-repressor 1 fusion mRNA)RUNX1-RUNX1T1 (Acute myeloid leukemia 1–eight-twenty-one fusion protein)
Beyond the preview
Go deeper on RUNX1::RUNX1T1 siRNA LNP.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Explore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Trace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Explore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Compare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Connect 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 RUNX1::RUNX1T1 siRNA LNP.