Work

Adopting Model‐Informed Precision‐Dosing for Eculizumab in Transplant Associated‐Thrombotic Microangiopathy to Gene Therapies

Year: 2023

Type: article

Abstract: Gene therapy advances offer great hope to previously uncurable disorders but come with a steep learning curve to assure safety and therapeutic success.Thrombotic microangiopathy (TMA) is a life-threatening complement-mediated vasculopathy that can complicate adeno-associated virus gene therapies.Here, we describe a pharmacokinetic/pharmacodynamic modelinformed precision dosing strategy for the complement blocker, eculizumab.This approach was developed for hematopoietic stem cell transplant recipients but can be applied more broadly to gene therapy-associated TMA.

Source: Clinical Pharmacology & Therapeutics

DOI: 10.1002/cpt.2966

Authors: Sonata Jodele, Kana Mizuno, Anthony Sabulski, Alexander A. Vinks

Institutions: Cincinnati Children's Hospital Medical Center, University of Cincinnati

Language: English

Topic: Complement system in diseases

Subfield: Immunology

Field: Immunology and Microbiology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $4,000