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ADEL-101 is an RNA-based aptamer-drug conjugate developed by Aptadel Therapeutics for the treatment of Ewing sarcoma, a rare and aggressive pediatric cancer. The drug consists of an RNA aptamer that specifically targets the Ephrin type-A receptor 2 (EphA2) on tumor cells, conjugated to a small interfering RNA (siRNA) designed to silence EWS/FLI1, the main oncogenic fusion driver in Ewing sarcoma. This dual mechanism aims to inhibit both tumor proliferation and metastatic potential. Preclinical studies have demonstrated high specificity for tumor targeting, significant reduction in tumor growth and metastasis in animal models, and a favorable safety profile with no observed toxicity at maximum tolerated doses. The platform technology underlying ADEL-101 may also be applicable to other cancers[1][3][4].
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