8:25 am Chair’s Opening Remarks

  • Eric Adler Chief Scientific Officer, Lexeo Therapeutics

Delivery Challenges & Solutions: Enhance Distribution in the Heart & Alleviate Safety Risk

8:30 am Development of Novel Gene Therapies for Treatment of Cardiomyopathy

  • Timothy Hoey Chief Scientific Officer, Tenaya Therapeutics


  • Developing AAV gene therapies for various types of genetic cardiomyopathy
  • Using capsid engineering to discover novel AAVs with improved cardiac to liver transduction ratio
  • Optimising promoters and other cis-regulatory elements

9:00 am Exploring the Best Tools to Safely & Effectively Access the Myocardium & the Role of Immunosuppresion

  • Eric Adler Chief Scientific Officer, Lexeo Therapeutics


  • Addressing the best methods to deliver precision therapeutics to the heart
  • What have we learned from previous studies of cardiac genetic therapeutics?
  • What immunosuppression is required to prevent adverse events?

9:30 am Elucidating Novel AAV/SERCA2a Gene Therapy for the Treatment of Duchenne Cardiomyopathy


  • Cardiomyopathy associated with Duchenne continues to cause morbidity and mortality in affected patients
  • Directed gene therapy to treat cardiomyopathy has not been available yet
  • Delivery of AAV/SERCA2a in patients with cardiomyopathy is a promising strategy for improving the lives of patients in need

10:00 am Structured Networking


This session is a great opportunity to introduce yourself to the attendees that you would like to have more in-depth conversations with. This session is the ideal opportunity to get face-to-face time with many of the brightest minds working in

the cardiovascular field and establish meaningful business relationships.

10:30 am Morning Break & Networking

Harnessing Patient Stratification: Promoting Precision Therapy in Genetic Mutations & Rapidly Progressing Disease

11:00 am Opportunities & Challenges for Genomics to Enhance Biomarker- Guided Treatment Strategies in Prevention & Treatment of Cardiometabolic Disease


  • Determining ways to identify patients who would benefit from genetically oriented approaches such as siRNA, ASO and longer-term genome editing
  • How do we ensure we’re treating patients early enough to improve their course of life?
  • Ideal screening method: cascade, paediatric or population?

Regenerative Medicine: Exploring the Potential of Next Generation Therapeutics for Myocardial Regeneration to Prevent Disease Progression

11:30 am Mesenchymal Precursor Cells for the Continuum of HFrEF From Class II/III Through End-Stage on LVADs


  • Intramyocardial MPC Phase 3 trial results showing reduction in CV death, MI and stroke in Class II/III HFrEF Patients
  • Intramyocardial MLC Phase 2/3 trial results showing reduction in CV death and GI bleeding in Class IV ischemic HF patients requiring left ventricular assist device implantation
  • Use of intravenous MLCs to reduce AAV dosage by 100-fold, reduce adverse events and improve efficacy in DMD

12:00 pm Unlocking the Potential of Gene Editing: Recent Progress of CRISPR/Cas9 Strategies to Transform Cardiovascular Therapeutics & Prevention

  • Kiran Musunuru Professor of Medicine, Perelman School of Medicine at the University of Pennsylvania


• Navigating potential therapeutic indications for gene editing in the cardiovascular
disease space
• Delivery methods: Viral vectors vs lipid nanoparticles vs virus-like particles
• Illuminating strategies to assess efficacy and safety of gene editing

12:30 pm Lunch Break & Networking

1:30 pm Clinical Development Synergies of Autologous and Allogeneic Bone Marrow Derived Cell Therapies for Ischemic Heart Failure


  • CardiAMP Cell Therapy for Ischemic HFrEF Phase III Trial
  • CardiALLO NK1R+ MSC Therapy for Ischemic HFrEF Phase I/II Trial

2:00 pm Leveraging the Learnings from Oncology: Delve into in vivo Generation of CAR-T Cells to Reduce Fibrosis & Restore Cardiac Function

  • Haig Aghajanian Co-founder & Vice President - Research, Capstan Therapeutics


  • The promise in vivo generation CAR-T cells hold as a therapeutic platform to treat cardiovascular disease
  • Understanding the use of modified mRNA targeted LNPs to reduce fibrosis and restore cardiac function
  • Evaluating efficacy using a mouse model of heart failure

Better Recapitulating Human Cardiovascular Disease in Preclinical Models to Improve Preclinical Drug Development & Reduce Immunogenicity

2:30 pm Unblinding Preclinical Studies- Enhance Reproducibility & Overcome the Translational Gap


  • Incorporating more of a clinical-type study design to preclinical research, i.e., well-powered, blinded, dose responsive for PK/PD, and predetermined translational readouts
  • Using translational readouts including improvement in pathophysiological phenotype to improve probability of success
  • Back-translation from clinical to preclinical to guide discovery

3:00 pm Afternoon Break & Networking

3:30 pm Using Human Genetics & Digital Medicine to Enhance Drug Development

  • Steven Lubitz Director, Translational Medicine, Novartis Institutes of Biomedical Research


  • Reviewing how human genetic data is being used to identify and prioritize therapeutic targets
  • Discussing the use of surrogate phenotypes for target discovery
  • Address advances in digital medicine that are enhancing drug development

4:00 pm Panel Discussion: Understanding Genetic Aspects of Cardiomyopathy & Deliberating Current & Future Models to Better Recapitulate Human Cardiovascular Disease

  • Pooja Agarwal Head of Gene Therapy Research, BioMarin Pharmaceutical
  • Timothy Hoey Chief Scientific Officer, Tenaya Therapeutics
  • Steven Lubitz Director, Translational Medicine, Novartis Institutes of Biomedical Research
  • Ibrahim Domian Vice President - Development, BlueRock Therapeutics


  • Advancing preclinical animal models to better understand mechanism of action in the heart
  • Establishing robust preclinical lifestyle models that are more reflective of the heterogeneous nature of human cardiovascular disease
  • How to successfully select the right model to maximise the translational relevance of result

4:30 pm Chair’s Closing Remarks

  • Pooja Agarwal Head of Gene Therapy Research, BioMarin Pharmaceutical

Scientific Poster Session


After the formal presentations have finished, the learning and networking carries on. The Poster Session allows you to connect with your peers in a relaxed atmosphere and continue to forge new and existing relationships.


During this session scientific posters will be presented on the very latest advancements in next generation therapies for cardiovascular diseases with a focus on better understanding and overcoming preclincial challenges to inform the direction of future drug development efforts