UTR Therapeutics Inc. Announces U.S. FDA Orphan Drug Designation and Rare Pediatric Disease Designation for UTRxMYCN M1-14

September 29, 2026
1:10 pm

U.S. FDA Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) underscore UTRxMYCN M1-14's therapeutic targeting of the "undruggable" MYCN (N-MYC) in rare, aggressive soft tissue sarcomas, addressing a significant unmet clinical need in pediatric and adult sarcomas.

NEW YORK, Sept. 29, 2026 /PRNewswire/ — UTR Therapeutics Inc., a biotech company with clinical trial-ready assets defining a new category of RNA therapeutics to conquer the undruggable proteome, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to 3′UTRMYCN M1-14 (UTRxMYCN M1-14) for the treatment of soft tissue sarcoma, including rhabdomyosarcoma (RMS), and Rare Pediatric Disease Designation (RPDD) for the treatment of rhabdomyosarcoma.

The company’s core technology of ultra-targeted 3′UTR engineering, invented by Chidiebere U. Awah, M.D., Ph.D. (CEO, UTR Therapeutics Inc.), targets the oncogenic mRNA of undruggable genes, overwrites their endogenous oncogenic mRNA messages, and triggers them for degradation by the nonsense-mediated decay pathway, thus eliminating the diseased mRNA while sparing the healthy mRNA.

UTR Therapeutics Inc. Announces U.S. FDA Orphan Drug Designation and Rare Pediatric Disease Designation for UTRxMYCN M1-14

U.S. FDA Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) underscore UTRxMYCN M1-14's therapeutic targeting of the "undruggable" MYCN (N-MYC) in rare, aggressive soft tissue sarcomas, addressing a significant unmet clinical need in pediatric and adult sarcomas.

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