Drug intelligence / Profile preview

MDK oligonucleotide

Development stage
Preclinical
Lead developer
Roquefort Therapeutics
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies
Administration
Unknown (preclinical Stage; Likely Parenteral Such As Intravenous Or Subcutaneous Based On Typical Aso Delivery)[1]
01

Overview

MDK oligonucleotide is a proprietary antisense oligonucleotide drug candidate developed to target and silence the expression of Midkine (MDK), a heparin-binding growth factor that is highly expressed in many solid tumors and associated with metastasis, poor treatment response, and poor prognosis. The drug works by interfering with the processing of Midkine mRNA, leading to reduced production of active Midkine protein in diseased tissues. Preclinical studies have shown that these antisense reagents can induce a splice-switching event resulting in truncated, non-functional Midkine protein and >90% reduction in functional MDK at the mRNA level. This mechanism has demonstrated efficacy in reducing cancer cell proliferation and tumor size in preclinical models. The program aims to develop first-in-class RNA medicines for "hard-to-treat" cancers expressing high levels of Midkine[1].

Other names
MDK RNA Oligoanti-Midkine antisense oligonucleotide
02

Targets

MDK (Midkine)

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