Drug intelligence / Profile preview

LeuFectB

Development stage
Preclinical
Lead developer
University of Alberta
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, Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

LeuFectB is a lipid-modified low molecular weight polyethyleneimine (PEI) lipopolymer designed as a delivery vehicle for short interfering RNA (siRNA) therapeutics. Developed by researchers at the University of Alberta, it is specifically optimized for the transfection of primary leukemia cells, including acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML) cells. The lipopolymer facilitates the intracellular delivery of siRNA targeting oncogenic drivers such as STAT5A, FLT3, and BCR-ABL. By downregulating these targets, LeuFectB-mediated siRNA therapy aims to reduce cell proliferation, viability, and the clonogenicity of leukemia stem cells while maintaining a favorable safety profile in non-malignant peripheral blood mononuclear cells (PBMCs).

02

Targets

ABL1 (ABL proto-oncogene 1, non-receptor tyrosine kinase)FLT3 (Fms related receptor tyrosine kinase 3)STAT5A (STAT5)

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