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VAL-070

Development stage: Preclinical
Lead developer: ValiRx
Modality: MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Intravenous

Overview

VAL-070 is a preclinical-stage therapeutic candidate developed by ValiRx, utilizing the proprietary Geneice platform technology. It is a targeted gene-silencing agent designed to inhibit the expression of the B-cell lymphoma 2 (BCL-2) gene. BCL-2 is a critical anti-apoptotic protein that is frequently overexpressed in various malignancies, including lymphomas and solid tumors, where it facilitates cell survival and resistance to chemotherapy. Unlike traditional antisense oligonucleotides that target mRNA, VAL-070 is designed to interact directly with the DNA of the BCL-2 gene, preventing transcription and thereby "switching off" the production of the protein. This approach aims to overcome the limitations of existing BCL-2 inhibitors by reducing protein levels at the source. VAL-070 is primarily being investigated for its potential in treating refractory cancers and as a sensitizing agent for other chemotherapeutic treatments.

Other names: Geneice BCL-2 inhibitor

Targets

BCL-2 (BCL-2 family)

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