Genetic Medicines to Treat Cardiovascular Disease

Jan 25–28, 2027 | Keystone Resort, Keystone, CO, United States
Scientific Organizers: Rick Dewey, Litsa G. Kranias, and James S. Ware

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Jan 25–28, 2027 | Keystone Resort, Keystone, CO, United States

Scientific Organizers: Rick Dewey, Litsa G. Kranias, and James S. Ware

Supported by:

***Meeting program subject to change.

Available Formats:     = Livestream   = In Person     = On Demand
Monday, January 25, 2027
Registration  
4:00–8:00 PM
Welcome Mixer  
6:00–8:00 PM
Tuesday, January 26, 2027
Breakfast  
7:00–8:00 AM
Welcome Remarks      
8:00–8:10 AM
Keynote Address      
8:10–9:00 AM
Sekar Kathiresan, Verve Therapeutics
The Human Genome as Teacher and Target: Two Decades of Genomic Discovery to Genomic Medicines
New Trailheads: Target Identification for Genetic Medicines in CV Disease      
9:00–11:15 AM
James S. Ware, Imperial College London
Large Scale Genomic Discovery of Causal Mechanisms in Heart Failure
Victoria Parikh, Stanford University School of Medicine
Genotype-Phenotype Correlation in Inherited Cardiovascular Disease
Rick Dewey, Versant Ventures
Target Selection for Genetic Medicines: Biobanks to Human Proof of Concept
Tony Zeng †, Stanford University
Short Talk: Perturb-seq in Endothelial Cells Identifies Key Regulators of Coronary Artery Disease States
Coffee Break  
9:30–9:50 AM
Panel Discussion 1: Development, Regulatory, Safety, and Ethical Considerations (including long-term and off-target effects of genetic medicines)      
11:25–12:25 PM
Rick Dewey, Versant Ventures
Silvia G. Priori, Foundation Salvatore Maugeri
Laura F. Michael, Eli Lilly and Company
Poster Setup  
12:25–1:00 PM
On Own for Lunch  
12:25–2:30 PM
Poster Viewing  
1:00–10:00 PM
Symposia Spotlight 1: Emerging Biological Insights Driving the Next Generation of Genetics Medicines for CV Disease      
2:30–4:30 PM
Daniel Yang, University of Washington
Short Talk: Deep Mutational Scanning of MYH7 in Human Cardiomyocytes Enables High-Throughput Variant Interpretation
Yensin Ang †, Lexeo Therapeutics
Short Talk: From Mechanism to Clinic: A Unified and Differentiated AAV Strategy for Genetic Cardiomyopathies
Areeba Iqbal †, University Of Agriculture, Faisalabad
Short Talk: - iPSC-Derived Cardiac Organoids for Personalized Cardiovascular Therapy Testing
Yu-Chieh Chen, The Jackson Laboratory of Genomic Medicine
Short Talk: A Cellular Memory of Dilated Cardiomyopathy Limits Cardiac Plasticity After Genetic Correction and Is Overcome by Myofilament Calcium Sensitization
Eren A Gunes †, University of California, San Diego
Short Talk: Gene-Corrected HSPCs Restore LAMP2 Expression and Improve Survival in a Danon Disease Mouse Model
Jesse Engreitz, Stanford University
Short Talk: Toward Genetic Medicines for Cardiovascular Diseases: Reprogramming Regulatory DNA with Prime Editing
Tom Lumbers †, University College London
Short Talk: Understanding Heart Failure Risk and Progression Through Large-Scale Genomics
Naoto Muraoka †, Gordian Biotechnology
Short Talk: Pooled In Vivo AAV Screening with Single-Nucleus Transcriptomic Readout Identifies Novel Genetic Medicines for HFpEF
Coffee Available  
4:30–5:00 PM
Mechanisms and Models: From Variation to Validation      
5:00–7:00 PM
Cassady Rupert, Propria LLC
Modeling Human Cardiac Biology with Multi-Dimensional Engineered Human Heart Tissues
Leslie A Leinwand, University of Colorado Boulder
Nature- Inspired Solutions to Common Metabolic Challenges
Litsa G. Kranias, University of Cincinnati
Phospholamban Cardiomyopathy: From Bedside to Bench and Back
Stephen Chang, Stanford University School of Medicine
Short Talk: A Primate Genetic Model Organism for Cardiovascular Disease
Pablo Hofbauer, HeartBeat.bio AG
Short Talk: Harnessing Cardioids for Cardiomyopathy Gene Therapy: A High-Fidelity Disease Modeling and Screening Platform
Social Hour with Dinner  
7:00–8:00 PM
Poster Session 1  
7:30–10:00 PM
Wednesday, January 27, 2027
Breakfast  
7:00–8:00 AM
Gene Silencing in Cardiovascular Disease and Promising Avenues      
8:00–11:00 AM
Jiang Jianming, National University of Singapore
Talk Title to be Announced
Stefanie Dimmeler, Institute of Cardiovascular Regeneration
Deciphering Noncoding RNAs in Cardiovascular Disease for RNA Therapeutics
Euan A. Ashley, Stanford University
Allele-Specific Silencing in Cardiomyopathy
Jin Billy Li, Stanford University
Endogenous RNA Editing in CV Disease: Mechanisms and Therapeutic Applications
Annet Linders †, PLaN Therapeutics
Short Talk: - Development of an Allele-Selective siRNA therapy for PLN-R14del Cardiomyopathy
Elena Smekalova, Scribe therapeutics
Short Talk: Cas-X Based Investigational Gene Editor Targeting APOC3 Demonstrates Potent Editing in Non-Human Primates and Effectively Lowers Triglycerides in a Preclinical Model of Hypertriglyceridemia
Coffee Break  
9:00–9:20 AM
Poster Setup  
11:00–1:00 PM
On Own for Lunch  
11:00–3:30 PM
Poster Viewing  
1:00–10:00 PM
Career Roundtable (Joint)  
3:30–4:30 PM
Coffee Available  
4:30–5:00 PM
Therapeutic Gene Augmentation Strategies in CV Disease      
5:00–7:00 PM
Roger J Hajjar, Mass General Brigham
Engineering the Failing Heart: Innovations in Cardiac Gene Therapy
Annemieke Aartsma-Rus, Leiden University Medical Center
Exon Skipping to Restore Gene Expression in Muscle Disease
Farah Sheikh, University of California, San Diego
From Cell Junctional Failure to Gene Therapy in Arrhythmogenic Cardiomyopathy
Timothy Calamaras †, Alexion, AstraZeneca Rare Disease
Short Talk: Development of an Optimized AAV Gene Therapy for MYBPC3 Associated Hypertrophic Cardiomyopathy
Adam Helms, University of Michigan
Short Talk: Mapping and Rewiring the MYBPC3 Promoter for Rescue of Haploinsufficiency-Driven Hypertrophic Cardiomyopathy
Social Hour with Dinner  
7:00–8:00 PM
Poster Session 2  
7:30–10:00 PM
Thursday, January 28, 2027
Breakfast  
7:00–8:00 AM
Homing for Cures: Cardiomyocyte-Directed Delivery Approaches      
8:00–11:00 AM
Luk H Vandenberghe, Harvard Medical School
Engineering Viral Delivery Systems
Anastasia Khvorova, RNA Therapeutics Institute, UMass Chan Medical School
Novel Approaches for Oligonucleotide Delivery to Heart
James Dahlman, Georgia Tech / Emory Medical School
Lipid Nanoparticles: Past, Present, and Future of RNA Delivery
Husam Younis, Atrium Therapeutics, Inc.
siRNA Delivery to the Heart: Opportunities in Genetic Cardiomyopathy
Georgios Karamanlidis †, Atrium Therapeutics
Short Talk: ATR 1086 Prevents Heart Failure and Rescues Survival in a Mouse Model of PLN-R14del Cardiomyopathy
Yunxi Chen, University of Southern California
Short Talk: Engineering MicroRNA-Enriched Extracellular Vesicles to Inhibit Atherosclerotic Plaque Growth and Vascular Calcification
Coffee Break  
9:00–9:20 AM
Panel Discussion 2: Navigating the Valley of Opportunity: Moving Ideas from Idea to Products for Patients with Academia, Venture Capital, and Pharma      
11:10–12:10 PM
Sekar Kathiresan, Verve Therapeutics
Roger J Hajjar, Mass General Brigham
On Own for Lunch  
12:10–2:30 PM
Symposia Spotlight 2: Emerging Translational Strategies in CV Disease      
2:30–4:30 PM
Mena F Abdelsayed †, Lankenau Institute for Medical Research
Short Talk: Alternans in PLN R14del Cardiomyocytes Differs Between Two Isogenic Pairs of Different Origin
Andreas M. Zeiher †, University Frankfurt
Short Talk: Hepatic microRNA-92a Inhibition Attenuates Liver Injury and Reduces Cardiac Fibrotic Remodeling in Metabolic Dysfunction-Associated Steatohepatitis (MASH)
Jeffrey D Steimle, Cincinnati Children's Medical Hospital Center
Short Talk: PITX2 Missense Coding Variant Expression Reveals Direct Function in Atrial Fibrillation
Ze Cheng †, Tenaya Therapeutics
Short Talk: In Vivo Rescue of Cardiomyopathy Through Cardiac Prime Editing
Michael S Kapiloff, Stanford University
Short Talk: Targeting of Perinuclear β1-Adrenergic Receptors Inhibits Pathological Cardiac Remodeling
Tianyu Wu, Duke University
Short Talk: Engineered BacNav Platform for Precision Cardiac Gene Therapies
Brad A. Amendt, University of Iowa
Short Talk: RNA Technology to Regenerate a Myocardial Infarct Through Re-Activation of a Developmental Gene Network
Gabriella Kabboul, Brigham and Women's Hospital & Harvard Medical School
Short Talk: Circular RNA-Mediated CCN5 Expression Reverses Pathologic Cardiac Remodeling and Improves Cardiac Function After Myocardial Infarction
Coffee Available  
4:30–5:00 PM
Products to Patients: Therapeutic Development and Trial Design      
5:00–6:15 PM
Laura F. Michael, Eli Lilly and Company
Lp(a) - From Genetic Insight to Novel Biology and Breakthrough Therapy
Silvia G. Priori, Foundation Salvatore Maugeri
Therapeutic Development of Genetic Medicines for Arrhythmogenic Cardiovascular Disease
Eileen Blasi, Atrium Therapeutics
Short Talk: ATR 1072 Prevents Cardiac Glycogen Accumulation and Hypertrophy in a Preclinical Model of PRKAG2 Syndrome
Closing Keynote Address      
6:15–7:05 PM
Christine E. Seidman, Harvard Medical School
Modeling Cardiovascular Disease: Genetics, Cellular Systems, Human Models
Meeting Wrap-Up: Outcomes and Future Directions      
7:05–7:15 PM
Social Hour with Dinner  
7:15–8:15 PM
Entertainment  
8:00–11:00 PM
Cash Bar  
8:00–11:00 PM
Friday, January 29, 2027
Departure  
12:00–11:59 PM

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