Drug intelligence / Profile preview

synthetic miR-142

Development stage
Preclinical
Lead developer
City of Hope
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
Intrathecal, Intramuscular
01

Overview

Synthetic miR-142 is an RNA therapy designed to restore the function of the microRNA miR-142, which is a short non-coding RNA molecule that regulates gene expression by downregulating target messenger RNAs (mRNAs), leading to mRNA degradation or translational inhibition. A deficit or downregulation of endogenous miR-142 has been linked to the progression of myeloproliferative neoplasms (MPNs) to secondary acute myeloid leukemia (sAML), as well as other hematological malignancies like acute myeloid leukemia (AML), lymphomas, and acute lymphoblastic leukemia (ALL). By re-establishing normal miR-142 levels, this therapy aims to normalize gene regulatory networks and signaling pathways involved in hematopoiesis and cancer progression. City of Hope is developing a synthetic miR-142 therapy for myeloproliferative neoplasms and their progression to sAML.

Other names
synthetic miR-142synthetic microRNA-142
02

Targets

Interleukin-1 alpha mRNA 3' untranslated regionCD274 (Programmed cell death protein 1 ligand 1)RICTORTransforming growth factor beta messenger RNA 3' untranslated regionTwinfilin actin binding protein 2 mRNA 3' untranslated regionAbsent, small, or homeotic 1-like (ASH1L) mRNA 3' untranslated regionPTEN (Phosphatase and tensin homolog deleted on chromosome ten)ADCY9 (Adenylyl cyclase 9)YES1 (YES proto-oncogene 1 tyrosine kinase)

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