8:55 am Chair’s Opening Remarks
Looking into the Future: Diving Deeper into the Potential & Perceived Added Therapeutic & Market Value for Cardiac Precision Medicine
9:00 am Emergence of Cardiac Precision Medicine & Perspectives on the Future
Synopsis
- Entering a new era of cardiac drug development to move past the ‘one size fits all’, historic approaches to treating cardiac diseases
- Breaking down the genetic components of cardiovascular disease and addressing the transition from small molecules to genetic medicines which may offer a functional cure
- Why will this space see a significant growth in investment?
9:30 am Panel Discussion: Deliberating Current Gene Editing Techniques & Methods to Overcome Off- Target Genome Editing to Advance Its Clinical Ability
Synopsis
- Which gene editing technique holds the most potential?
- How can we overcome off-target genome editing?
- Engaging regulatory authorities in discussions to advance its utilisation
10:00 am Morning Break & Networking
Navigating the Efficacy & Toxicity Balance to Better Assess the Therapeutic Window of Opportunity
11:00 am Exploring Strategies to Align Manufacturing of Stem Cell Derived Products to the Safety & Efficacy of the Product Whilst Meeting CMC Requirements
Synopsis
- Illustrating ways to generate high quality induced pluripotent stem cells (iPSCs)
- Understanding the specific differentiation into ventricular cardiomyocytes to minimize safety risks of the cells
- Establishing a scalable manufacturing system to purify cardiomyocytes and reduce the cost of goods
11:30 am Regulatory Perspective on Navigating the Safety Vs Toxicity Hurdle to Achieve Success in Clinical Trials by Agreeing on a Standardized Approach for Dose Escalation & Endpoints
Synopsis
- Addressing trial design concerns to ensure the risks and benefits are well balanced
- Coupling preclinical knowledge to find the right dose that’s both safe and efficacious in the clinical realm
- How to anticipate adverse events to propose a mitigating strategy
12:00 pm Establishing Potential Endpoints in Clinical Trials to Innovate Treatment
Synopsis
- Establishing Potential Endpoints in Clinical Trials to Innovate Treatment
- Evaluating clinically meaningful endpoints and alternatives to invasive procedures to stimulate patient engagement in trials
- Assessing strategies for engaging regulatory authorities in discussions on redefining approvable endpoints
- Identifying easily measured and highly correlated surrogate endpoints to shorten studies and reduce sample size
12:30 pm Lunch Break & Networking
1:30 pm Improving Predictive Metrics of Efficacy & Preclinical Modelling Systems to Mitigate Against Toxicity & Long-Term Effects Using Modified mRNA
Synopsis
- Ischemic heart disease is the leading cause of death in the world
- Modified mRNA is an ideal tool to induce cardiac regeneration, cardiac protection and cardiovascular regeneration post heart attack
- Specific modified mRNA translational system (SMRTs) allows cell and organ specific translation of modified mRNA
Innovating Clinical Trial Design to Advance Effective Treatments & Uncovering New Techniques to Maximize the Use of Biomarkers in Heart Tissue
2:00 pm High Collagen Type VI Formation Is a Strong and Independent Predictor of Poor Prognosis in HFpEF
Synopsis
- HFpEF is a highly heterogenous disease & patient stratification is essential to inform clinical trial
- Meta-analysis data demonstrated strong, independent prognostic value of PRO-C6 as a novel biomarker in HFpEF (NEJM Evidence 2022)
- FDA issued Letter of Support (LoS) to encourage further PRO-C6 work in HFpEF
2:30 pm Afternoon Break & Networking
3:00 pm Constructing a Selection Biomarker Panel in the CardiAMP Autologous Cell Therapy Phase III HFrEF & Phase III Chronic Myocardial Ischemia Programs
Synopsis
- Patient selection biomarker assessment for autologous cell therapy
- Considerations in the development and use of a biomarker panel
- Logistics of implementation
3:30 pm Challenges & Opportunities for Imaging & Biomarker Endpoints that Reveal Cardiac & Vascular Remodeling
Synopsis
- Adapting cardiovascular imaging from their diagnostic application into drug development tools can serve multiple biomarker roles (efficacy, selection, safety)
- Myocardial structure and function endpoints can elucidate therapeutic response if appropriately selected
- Innovation with these techniques and in combination with peripheral biomarkers will have a role in cardiovascular precision medicine
4:00 pm Reimagining Biomarkers to Illuminate the Right Patient Population & Advance Precision Medicine Impact
Synopsis
- Navigating biomarkers in cardiovascular drug development that span all modalities – genetics, peripheral proteins, lipids, inflammatory markers as well as imaging and device-derived markers
- De-risking cardiovascular outcome trials with early proof of concept biomarkers remains a major challenge
- Exploratory omic analyses in cardiovascular outcomes offer hypothesis generation for precision medicine
4:30 pm Chair’s Closing Remarks
End of Conference
