Farah Sheikh, PhD, on Continuing Gene Therapy Research Into Arrhythmias, Cardiac Dysfunction


The professor of medicine at University of California San Diego discussed the advent of gene therapy into the field of cardiac medicine.

“Although we have therapies or interventions that can potentially help arrhythmias, they don't necessarily help with the heart failure end of things. So, we have to move into that venue. And that's why I'm excited about this therapy... So hopefully, this therapeutic has this potential to not only impact the arrhythmias but also that transition to dysfunction.”

Farah Sheikh, PhD, professor of medicine, University of California and her lab are working to address gaps in the field of cardiac medicine. One such gap, as she told CGTLive in a recent interview, was the lack of accurateanimal models of diseases such as arrhythmogenic right ventricular cardiomyopathy (ARVC) to better representthe disease phenotype in humans and improve preclinical research with gene therapy for the indication. Sheikh’s lab recently addressed that problem with a new mouse model and also evaluated the novel LX2020 gene therapy in these models.

Sheikh presented preclinical data on LX2020 as well as the new mouse model at the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting, held May 16-20, in Los Angeles, California.1 CGTLive spoke with Sheikh to learn more about the model and how genetic research has progressed in the field of cardiac medicine. She shared her excitement for the advent of gene therapies in the field, as they have the potential to treat underlying cardiac dysfunctions and not only symptomatic arrhythmias. LX2020 recently received investigational new drug clearance from the FDA for a first-in-human trial.2

Click here to read more coverage of the ASGCT 2023 meeting.

1. Sheikh F, Zhang J, Nair A, et al. LX2020-An AAV based gene therapy improves the arrhythmogenic right ventricular cardiomyopathy phenotype in a severe mouse model harboring human PKP2 mutation. Presented at: ASGCT 2023 Annual Meeting; May 16-30; Los Angeles, California. Abstract #205
2. LEXEO Therapeutics Announces FDA Clearance of IND for LX2020, an AAV-based Gene Therapy Candidate for PKP2 Arrhythmogenic Cardiomyopathy. News release. LEXEO Therapeutics. August 1, 2023. https://finance.yahoo.com/news/lexeo-therapeutics-announces-fda-clearance-113000058.html
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