Nuevocor, a pre-clinical stage cardiac gene therapy company, announced that it will present new preclinical data on its adeno-associated virus (AAV) gene therapy for LMNA dilated cardiomyopathy at the American Heart Association (AHA) Scientific Sessions meeting on 5 – 7 November, 2022. This follows its maiden presentation at the American Society of Gene and Cell Therapy (ASGCT) meeting earlier this year.
Mutations in the LMNA gene, encoding lamin A/C, are thought to be the 2nd most frequent cause of familial dilated cardiomyopathy, affecting approximately 60,000 people in the US and EU alone. Being an autosomal dominant disease characterized by gain-of-function of the mutant lamin A/C protein, conventional gene replacement therapies would be ineffective for LMNA DCM. Nuevocor’s unique strategy for treating LMNA DCM instead targets a disease modifier, using AAV to overexpress a transgene that disrupts the Linker of Nucleoskeleton and Cytoskeleton complex to suppress the pathology of LMNA mutations.
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Details of the presentation are as follows:
Room: S102A
Session: HF.AOS.470 – Novel Therapeutics in Heart Failure
Title: AAV Gene Therapy to Treat Underlying Cardiac Biomechanical Defects in Lmna Dilated Cardiomyopathy Improves Lifespan and Preserves Ejection Fraction in Lmna DCM Mouse Model
Presenter: Hendrijke Werner, Principal Scientist, Nuevocor
For more information on the conference, please visit the AHA Scientific Sessions 2022 website.
Nuevocor is a biotechnology company developing targeted gene therapies to restore heart function through a deep understanding of fundamental mechanisms underlying genetic cardiac diseases. Nuevocor is founded and advised by leaders in mechanobiology, cardiac disease and gene therapy. Its lead candidate treats the underlying biomechanical cause of LMNA dilated cardiomyopathy, while its PrOSIA mechanobiology platform enables target discovery and validation for additional genetic cardiomyopathies.