{"entity":{"id":"utrxmycn-m1-14","kind":"drug","name":"UTRxMYCN M1-14","aka":["3'UTRMYCN M1-14"],"tldr":"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.","summary":"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.","status":"preclinical","asOf":"2026-11-04","links":[{"label":"UTR Therapeutics","url":"https://www.utrtherapeutics.com/"},{"label":"FDA orphan designation record","url":"https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=1182626"}],"tags":["pipeline","orphan-designation"],"related":[],"cancers":["sarcoma","rhabdomyosarcoma"],"sections":[],"technologies":[],"targets":["mycn"],"drugs":[],"companies":["utr-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo startup monitors","editedOn":"2026-11-04"},"code":"UTRxMYCN M1-14","modality":"Synthetic mRNA (RNA overwriting)","mechanism":"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.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2026-09-22","type":"designation","region":"US","note":"FDA orphan drug designation for soft tissue sarcoma, including rhabdomyosarcoma, granted to UTR Therapeutics","source":"https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=1182626","indication":"treatment of soft tissue sarcoma, including rhabdomyosarcoma"}]},"route":"/drugs/utrxmycn-m1-14/","neighbours":{"cancer":[{"id":"rhabdomyosarcoma","kind":"cancer","name":"Rhabdomyosarcoma","route":"/cancers/rhabdomyosarcoma/"},{"id":"sarcoma","kind":"cancer","name":"Sarcomas (soft tissue, bone, GIST)","route":"/cancers/sarcoma/"}],"target":[{"id":"mycn","kind":"target","name":"MYCN (N-myc)","route":"/targets/mycn/"}],"company":[{"id":"utr-therapeutics","kind":"company","name":"UTR Therapeutics","route":"/companies/utr-therapeutics/"}]}}