SHARE NOW
LEXEO Therapeutics, LEXEO, has announced that the FDA has given clearance for its IND for LX2020, an AAV-based gene therapy candidate for the cardiac disease PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM).
Clinical-stage gene therapy company, LEXEO, is primarily focused on advancing adeno-associated virus (AAV)-based gene therapy candidates for genetically defined cardiovascular diseases and a genetically defined sub-group of Alzheimer’s disease. The IND application for LX2020, an AAVrh10-based gene therapy candidate designed to intravenously deliver a functional PKP2 gene to cardiac muscle for the treatment of Arrhythmogenic Cardiomyopathy (ACM), was cleared by the FDA.
The standard-of-care treatments of ACM include antiarrhythmics, implantable cardioverter-defibrillators (ICDs), and ablation procedures, none of which address the underlying cause of myocardial dysfunction and ACM. there are no approved treatments to slow, prevent, or reverse disease progression in patients with PKP2-ACM.
“LX2020 seeks to address the underlying cause of this disease by delivering a functional PKP2 gene to halt progression and reverse the disease phenotype. We look forward to initiating the Phase 1/2 clinical trial and rapidly advancing the program through the clinic,” expressed Eric Adler, M.D., Chief Scientific Officer of LEXEO.
ACM is caused by variants in the PKP2 gene (PKP2-ACM). LX2020 is designed to intravenously deliver a functional PKP2 gene to cardiac muscle to increase PKP2 protein levels in the cardiac desmosome, a cell structure critical for cell adhesion. The LX2020 treatment in preclinical studies demonstrated restoration of desmosomal and cell-cell junction integrity. The study demonstrated improvements in cardiac tissue composition and integrity and alleviation of arrhythmias as well as improvements in right and left ventricular function resulting in increased survival.
How are you enjoying this news article? Let us know your thoughts, here >>
Led by Dr. Farah Sheikh at the University of California, San Diego School of Medicine, the program is based on collaborative research.
The planned Phase I/II trial, HEROIC-PKP2, is the first in human, 52-week open-label, dose-escalating, multicenter trial that will determine the safety and tolerability of LX2020 in adult subjects with PKP2-ACM.
LX2020 will be administered as a one-time intravenous infusion to patients in two ascending-dose cohorts of three patients each, with the potential for cohort expansion. Following the completion of the initial trial, the long-term safety and efficacy will be evaluated for an additional four years.
Source: LEXEO Therapeutics Press Release
While we have you…
We are continuously working to better optimize our content for you, our audience. Let us know how we’re doing by ticking the box below, or send us your feedback directly to team@phacilitate.com.
