TargomiR (EGFREDVmiR-16) / EnGeneIC, Asbestos Diseases Research Institute - LARVOL DELTA

Nanotechnology-enabled miRNA delivery systems next-generation molecular strategies in cancer therapy. (PubMed, Biochem Biophys Res Commun) - "We conclude with a concise roadmap that prioritizes platform selection for near-term translation and integration with tumor-specific miRNA signatures to advance personalized therapy. Our translation-first synthesis links barriers to design solutions (pKa-tuned LNPs; exosome-mimetic/hybrid vesicles) and highlights AI/ML-guided formulation; lessons from MRX34/TargomiRs inform safety, scalability, and CMC-together yielding a practical 24-36-month roadmap toward clinical readiness, with functionalized LNPs best positioned for near-term translation."

Journal • Review • Oncology

MicroRNAs: Novel clinical biomarkers for cancer radiotherapy (Review). (PubMed, Mol Med Rep) - "Despite the early challenges of miRNA‑based therapies, advancements in miRNA delivery systems, including TargomiR‑ and liposome‑based approaches, offer promising avenues for clinical applications. The present review highlights the role of miRNAs as biomarkers and modulators in cancer radiotherapy and discusses ongoing research on miRNA delivery mechanisms to improve therapeutic outcomes. Future studies are needed to address the challenges of miRNA pleiotropy and safety in clinical applications, to advance miRNA‑based interventions in precision oncology, and to enhance the efficacy of radiotherapy across various cancer types."

Biomarker • Journal • Review • Oncology • MIR144 • MIR200C

Targeting Altered microRNA Expression in Mesothelioma (IASLC-WCLC 2019) - "...With miR-16 also impacting response to pemetrexed and contributing to PD-L1 regulation in vitro, restoration of miR-16 levels in combination with chemo or immunotherapy are potential future applications of this approach...The successful phase I trial of TargomiRs demonstrated that miRNA targeting is feasible in MPM and while the majority of miRNA studies in MPM have focused on miRNA mimics, recent studies suggest that antisense inhibitors have similar potential. Notwithstanding the ongoing difficulties in delivering nucleic acid-based drugs, the recent FDA approval of the first siRNA therapy – together with ongoing clinical trials of a number of miRNA mimic drugs – means that gene silencing drugs have moved from concept to reality. Continued preclinical studies and early phase clinical trials are needed to determine the true potential of miRNA targeting in MPM treatment."

IO Biomarker • PD(L)-1 Biomarker

Mir-23B-3P Promotes Carcinogenicity Through Petn/Pi3K/Akt Pathway in Vitro and in Vivo in Pancreatic Carcinoma (DDW 2019) - "...Meantime, PI3K and p-Akt were found higher in the subcutaneous tumor of mice with miR-23b-3p argomir (over expression) injection, but these changes can be reversed by miR-23b-3p antargomir (down expression) injection...Figure2. IL-6 can induce the miR-23b-3p expression and the up-regulated miR-23b-3p can promote PI3K, p-Akt and MMP2 but reduce PTEN expression."

Preclinical