The professor of medicine at University of California San Diego discussed promising preclinical findings she presented at the American Heart Association’s Scientific Sessions 2023.
“I think the big picture is that gene therapy—which is what this is, it's replacing a gene which is defective—is safe and it can work [in disease states]. I think this is really remarkable.”
Arrhythmogenic cardiomyopathy (ACM) is a rare genetic disease, although it is more commonly found in certain populations, such as athletes. The condition can lead to a loss of pump function, heart failure, and sudden death. With no currently available treatments specifically targeted at ACM, significant unmet need exists for these patients. ACM is linked to desmosomal genes such as plakophilin-2 (PKP2), and as such may be a candidate for future treatment with gene therapy. Lexeo Therapeutics is currently developing one such approach: LX2020. This adeno-associated virus vector-based gene therapy, which is intended to provide a functional copy of PKP2, was recently cleared for a first-in-human trial by the FDA. At the American Heart Association’s (AHA) Scientific Sessions 2023, held November 10-13 in Philadelphia, Pennsylvania, Farah Sheikh, PhD, a professor of medicine at University of California San Diego, presented findings from mouse model and nonhuman primate (NHP) research that has been conducted with LX2020.
In an interview with CGTLive™ at the conference, Sheikh gave an overview of the unmet needs in ACM and discussed the key results from research on the gene therapy that she presented. She highlighted that in a mouse model of late-stage ACM, mice who received treatment with the gene therapy had substantially longer survival than mice who did not receive the gene therapy. The treated mice also showed improvement in their heart rhythm and function and showed disappearance of a substantial amount of scarring in the heart. Sheikh also pointed out that LX2020 was demonstrated to be safe and well-tolerated in NHPs, with no signs of pathology, at a dose-level expected to be efficacious in future human studies.