Lexeo Therapeutics, a clinical-stage gene therapy company, has received FDA clearance for its Investigational New Drug (IND) application for LX2020, an adeno-associated virus (AAV)-based gene therapy candidate targeting Arrhythmogenic Cardiomyopathy (ACM) caused by variants in the PKP2 gene. This genetic cardiac disease affects approximately 60,000 individuals in the United States.

LX2020’s preclinical studies in a relevant murine model of PKP2-ACM demonstrated promising outcomes, with restored desmosomal integrity, reduced arrhythmias, and extended survival. The therapy delivers a functional PKP2 gene intravenously to the cardiac muscle, elevating PKP2 protein levels in the critical cardiac desmosome—a structure vital for cell adhesion.

The upcoming Phase 1/2 trial, HEROIC-PKP2, will be a first-in-human, 52-week open-label, dose-escalating, multicenter study evaluating LX2020’s safety and tolerability in adult PKP2-ACM patients. Additionally, efficacy measures will assess myocardial protein expression, cardiac structure and function biomarkers, and arrhythmia burden. LX2020 will be administered as a one-time intravenous infusion in two ascending-dose cohorts, with potential expansion, followed by long-term safety and efficacy monitoring for four years.

PKP2-ACM is characterized by myocardial cell loss, replaced by fibrotic tissue and fatty deposits, primarily caused by PKP2 gene variants, leading to heart dysfunction, rhythm abnormalities, and sudden death. Current treatments only manage symptoms and lack targeted disease intervention.

The clearance of LX2020’s IND represents a significant step toward offering a potential one-time treatment for PKP2-ACM patients, who currently lack effective therapeutic options.

LEXEO Therapeutics aims to develop effective treatments for genetically defined cardiovascular diseases and a subgroup of genetically defined Alzheimer’s disease through a diverse pipeline of AAV-based gene therapies.

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PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha™ 293 cell AAV high-yield platform and the π-Omega™ plasmid high-yield platform. PackGene’s mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.

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