Investigational agents for autosomal dominant polycystic kidney disease: preclinical and early phase study insights

Expert Opin Investig Drugs. 2024 Apr 15. doi: 10.1080/13543784.2024.2342327. Online ahead of print.ABSTRACTINTRODUCTION: Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common inherited kidney condition caused by a single gene mutation. It leads patients to kidney failure in more than 50% of cases by the age of 60, and, given the dominant inheritance, this disease is present in the family history in more than 90% of cases.AREAS COVERED: This review aims to analyze the set of preclinical and early phase studies to provide a general view of the current progress on ADPKD therapeutic options. Articles from PubMed and the current status of the trials listed in clinicaltrials.gov were examined for the review.EXPERT OPINION: Many potential therapeutic targets are currently under study for the treatment of ADPKD. A few drugs have reached the clinical phase, while many are currently still in the preclinical phase. Organoids could be a novel approach to the study of drugs in this phase. Other than pharmacological options, very important developing approaches are represented by gene therapy and the use of MiRNA inhibitors.PMID:38618918 | DOI:10.1080/13543784.2024.2342327
Source: Expert Opinion on Investigational Drugs - Category: Drugs & Pharmacology Authors: Source Type: research