Research & Publications

Advancing microRNA therapeutics

Peer-reviewed publications and clinical resources from the Pherrix team and collaborators, spanning preclinical discovery through first-in-human clinical translation.

Publication Category

Foundational Preclinical Studies

In vitro and in vivo anti-tumor activity of miR-221/222 inhibitors in multiple myeloma

Maria Teresa Di Martino et al.

Oncotarget (2013)

Demonstrated anti-myeloma activity of miR-221/222 inhibition in preclinical models.

View resource

A 13-mer LNA-i-miR-221 inhibitor restores drug sensitivity in melphalan-refractory multiple myeloma cells

Annamaria Gulla, Maria Teresa Di Martino, Pierfrancesco Tassone et al.

Clinical Cancer Research (2016)

Showed reversal of drug resistance using LNA-i-miR-221.

View resource

Pharmacokinetics and Biodistribution of Locked Nucleic Acid miR-221 Inhibitor

Pierfrancesco Tassone et al.

Blood / ASH Proceedings (2015)

Evaluated biodistribution and safety of LNA-i-miR-221 in animal models.

View resource

Publication Category

Pharmacology & Translational Development

Dose-Finding Study and Pharmacokinetics Profile of the Novel 13-Mer Antisense miR-221 Inhibitor in Sprague-Dawley Rats

Maria Teresa Di Martino et al.

Molecular Therapy Nucleic Acids (2020)

Defined PK profile and dose optimization of LNA-i-miR-221.

View resource

Allometric Scaling Approaches for Predicting Human Pharmacokinetic of a Locked Nucleic Acid Oligonucleotide Targeting Cancer-Associated miR-221

Maria Teresa Di Martino et al.

(2019)

Predicted human pharmacokinetics using interspecies scaling models.

View resource

Development and validation of bioanalytical methods for LNA-i-miR-221 quantification in human plasma and urine by LC-MS/MS

Samantha Franzoni et al.

(2020)

Developed analytical assays supporting clinical translation.

View resource

Publication Category

Clinical Translation

Safety and activity of the first-in-class locked nucleic acid (LNA) miR-221 selective inhibitor in refractory advanced cancer patients: a first-in-human, phase 1, open-label, dose-escalation study

Pierfrancesco Tassone, Maria Teresa Di Martino et al.

ESMO Open (2023)

First-in-human clinical trial of LNA-i-miR-221 in advanced cancer patients.

View resource

NCT04811898 - A Dose Escalation Study of LNA-i-miR-221

ClinicalTrials.gov

Clinical trial listing

ClinicalTrials.gov listing for the first-in-human trial.

View resource

Publication Category

Reviews & Broader miR-221 Biology

miR-221/222 as biomarkers and targets for therapeutic intervention on cancer and other diseases: A systematic review

Maria Teresa Di Martino et al.

Molecular Therapy Nucleic Acids (2022)

Comprehensive review of miR-221 biology, oncology relevance, and therapeutic targeting.

View resource

MicroRNA in cancer therapy: breakthroughs and challenges in early clinical applications

Maria Teresa Di Martino, Pierfrancesco Tassone et al.

(2025)

Review discussing clinical development of miRNA therapeutics including miR-221 inhibition.

View resource