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UTRxMYCN M1-14 is an experimental RNA medicine from UTR Therapeutics that makes the message of the cancer gene MYCN break down. It has been tested only in the laboratory, and the FDA has given it orphan drug status for soft tissue sarcoma.
UTRxMYCN M1-14 is UTR Therapeutics' MYCN-targeting candidate, delivered with its iron oxide nanocage system. The company reports that in preclinical studies it degraded MYCN in MYCN-driven rhabdomyosarcoma models, including the fusion-positive form, with dose-dependent tumour and metastasis inhibition. On 22 September 2026 the FDA granted orphan drug designation for the treatment of soft tissue sarcoma, including rhabdomyosarcoma, to a synthetic mRNA designed to destabilise the 3' UTR of the MYCN gene; the company also announced a Rare Pediatric Disease Designation for rhabdomyosarcoma.
A synthetic mRNA designed to destabilise the MYCN transcript through its 3' untranslated region, so that MYCN mRNA is degraded by nonsense-mediated decay and the MYCN protein is not made, as the FDA record and the developer describe it. Connects to MYCN (N-myc).
1.UTRxMYCN M1-14 slips into a pocket on MYCN (N-myc).
treatment of soft tissue sarcoma, including rhabdomyosarcoma
FDA orphan drug designation for soft tissue sarcoma, including rhabdomyosarcoma, granted to UTR Therapeutics source
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Query for this drug: (TITLE:"UTRxMYCN M1-14" OR ABSTRACT:"UTRxMYCN M1-14" OR TITLE:"3'UTRMYCN M1-14" OR ABSTRACT:"3'UTRMYCN M1-14") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about UTRxMYCN M1-14, not a curated reading list.
Shares Sarcomas (soft tissue, bone, GIST) and the tags pipeline, orphan-designation.
Shares the tags pipeline, orphan-designation.
Shares the tags pipeline, orphan-designation.
Shares the tags pipeline, orphan-designation.
Shares the tags pipeline, orphan-designation.
Shares the tags pipeline, orphan-designation.
Shares the tags pipeline, orphan-designation.
Shares the tags pipeline, orphan-designation.
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