{"entity":{"id":"utr-therapeutics","kind":"company","name":"UTR Therapeutics","aka":["UTR Therapeutics Inc.","UTRx Inc."],"tldr":"UTR Therapeutics is a New York company making RNA medicines that make a cancer-driving gene's message break down before it can be made into protein. Its sarcoma medicine, UTRxMYCN M1-14, aims at MYCN, a cancer gene that ordinary drugs cannot grip.","summary":"UTR Therapeutics Inc., headquartered at 169 Madison Avenue in New York City, describes itself as an IND-stage company with an RNA overwriting platform delivered by an iron oxide nanocage, designed to downregulate a chosen transcript by engineering its 3' untranslated region so that the message is destroyed by nonsense-mediated decay. It reports preclinical work in c-MYC-driven and HER2-expressing tumours and says it has assets against c-MYC, MYCN, TEAD1, YAP1 and HER2; in May 2025 it announced an IND submission for a c-MYC programme. On 22 September 2026 the FDA granted orphan drug designation to UTRxMYCN M1-14 for soft tissue sarcoma, including rhabdomyosarcoma, and the company announced a Rare Pediatric Disease Designation for rhabdomyosarcoma at the same time.","asOf":"2026-11-04","links":[{"label":"Official website","url":"https://www.utrtherapeutics.com/"},{"label":"FDA orphan designation, soft tissue sarcoma, 22 September 2026","url":"https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=1182626"}],"tags":[],"related":[],"cancers":["rhabdomyosarcoma","sarcoma"],"sections":["targeted-therapy"],"technologies":["antisense-sirna"],"targets":["mycn"],"drugs":["utrxmycn-m1-14"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Read on the company website (a single-page application whose text was read from its published script) on 4 November 2026. The efficacy claims for UTRxMYCN M1-14 are preclinical and the company's own. No technology record fits 3' UTR overwriting exactly; antisense-sirna is the nearest RNA-targeting entry."],"hq":"New York, NY","country":"US","companyType":"biotech","website":"https://www.utrtherapeutics.com/","stage":"startup","investors":[],"funding":[]},"route":"/companies/utr-therapeutics/","neighbours":{"cancer":[{"id":"rhabdomyosarcoma","kind":"cancer","name":"Rhabdomyosarcoma","route":"/cancers/rhabdomyosarcoma/"},{"id":"sarcoma","kind":"cancer","name":"Sarcomas (soft tissue, bone, GIST)","route":"/cancers/sarcoma/"}],"section":[{"id":"targeted-therapy","kind":"section","name":"Targeted Therapy","route":"/fronts/targeted-therapy/"}],"technology":[{"id":"antisense-sirna","kind":"technology","name":"Oligonucleotide therapeutics","route":"/technologies/antisense-sirna/"}],"target":[{"id":"mycn","kind":"target","name":"MYCN (N-myc)","route":"/targets/mycn/"}],"drug":[{"id":"utrxmycn-m1-14","kind":"drug","name":"UTRxMYCN M1-14","route":"/drugs/utrxmycn-m1-14/"}]}}