EXPLO...RARE 2026: From Molecular Mechanisms to Precision Therapeutics in Rare Diseases

This Research Topic originates from EXPLO...RARE 2026 – Bridges between Research and Therapy, an international conference held in Bari, Italy, that brought together clinicians, pharmacologists, molecular biologists, biochemists, and computational scientists working toward innovative therapeutic strategies for rare diseases. The EXPLO...RARE conference series was conceived to foster interactions between basic and translational research on rare diseases. Building upon this mission, the present Research Topic aims to highlight and extend selected scientific contributions presented during the meeting while also welcoming original submissions from the broader international community addressing related themes.Rare diseases collectively affect millions of individuals worldwide and are frequently characterized by complex and poorly understood molecular mechanisms, limiting the development of effective therapeutic interventions. Advances in molecular pharmacology and precision medicine are now providing unprecedented opportunities to identify actionable targets and accelerate the translation of basic discoveries into clinical applications.This collection focuses on emerging pharmacological strategies for the identification, validation, and targeting of biomolecular determinants of rare diseases. Contributions encompass the discovery of druggable enzymes, transporters, receptors, and regulatory proteins, as well as the characterization of disease-associated pathways including mitochondrial dysfunction, redox imbalance, metabolic rewiring, and altered signaling networks.Particular emphasis is placed on innovative therapeutic approaches such as drug repurposing, structure-based drug design, artificial intelligence-assisted computational screening, and integrated experimental-computational pipelines for target prioritization and ligand discovery. Studies addressing pharmacokinetics, pharmacodynamics, drug delivery systems, biomarker discovery, and translational validation are equally encouraged.By creating a bridge between the scientific discussions initiated at EXPLO...RARE 2026 and future advances from the wider research community, this Research Topic aims to foster interdisciplinary collaborations and accelerate the development of precision pharmacological strategies for rare diseases. Topic editors: Ciro Leonardo Pierri, Albina Tummolo, Donatella De Giovanni Source: https://www.frontiersin.org/research-topics/82498/explorare-2026-from-molecular-mechanisms-to-precision-therapeutics-in-rare-diseases