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UTR Therapeutics
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.
Also called: UTR Therapeutics Inc.UTRx Inc.
Overview
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.
Headquarters
New York, NY, US
Scorecard
All companies#595of 1706 companies, score 6
- targets +2
- modalities +2
- 24-month regulatory events +2
0 approved, 0 in phase 3, 0 earlier, 1 targets, 0 registry studies. Formula on the scorecards page.
Type
Stage
Startup
Website
Products
1PreclinicalSynthetic mRNA (RNA overwriting)
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.
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.
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cancers
fronts
technologies
targets
drugs
PreclinicalSynthetic mRNA (RNA overwriting)
Sources
Every source this page cites, grouped by who published it. Grouping is read from the web address, not assigned by hand.
US Food and Drug Administration · 1
utrtherapeutics.com · 1
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