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Mechanobiology-Based Gene Therapy for Genetic Cardiomyopathy with Al Gianchetti Nuevocor

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20:07
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Al Gianchetti, CEO of Nuevocor,  discusses genetic cardiomyopathies, an underdiagnosed condition that might explain multigenerational heart failure. Current treatments include beta blockers, pacemakers, and heart transplants but do not alter the course of the disease.  Because of multiple gene mutations, Nuevocor is developing a precision therapy for LMNA-related cardiomyopathy based on mechanobiology that prevents cardiac cell death using a method different from gene replacement and gene editing.

Al explains, "We're focused on genetic cardiomyopathies, and our lead drug, NVC001 in particular, is focusing first on LMNA cardiomyopathy, and LMNA is a specific gene mutation. We're focusing on LMNA-related cardiomyopathy for several reasons. First of all, it's one of the most severe forms of genetic cardiomyopathy, with a very poor prognosis for patients and their families who have the genetic mutation."  

"The second reason is that although it's a rare disease, it affects a fairly large number of people. For example, about 50,000 people in the United States are estimated to have LMNA-related cardiomyopathy. The third reason is that this disease is not amenable to gene replacement strategies where you're replacing the defective gene. The size of the trans gene that's required doesn't make it amenable to current gene therapy viral vectors. Finally, because there are multiple gene mutations, it's not amenable to gene editing."

#Nuevocor #DrugDiscovery #Innovation #Biotech #Cardiology #GeneTherapy #GeneticCardiomyopathy #HeartFailure #LMNACardiomyopathy #PrecisionMedicine #RareDisease #ClinicalTrials

nuevocor.com

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