CONFERENCE DAY TWO
JUNE 12, 2025
8:30 am Chair’s Opening Remarks
Striving for First- & Best-in-Class with Pioneering Novel Targets & Mechanisms of Action
8:40 am RNAi Medicines for Obesity: Towards Best-in-Class Durability & Tissue Selectivity
Synopsis
- Demonstrating why RNAi medicines are well-positioned to impact cardiometabolic and obesity outcomes and prevention
- SanegeneBio has developed Ligand and Enhancer Assisted Delivery (LEAD™) technology enabling safe, selective, and durable siRNA delivery to adipose, muscle, and liverLEAD™ powered siRNAs achieve preclinical efficacy comparable to semaglutide but with preservation of lean muscle mass, and prevent weight rebound after GLP1 withdrawal
9:10 am JK07: Selective ErbB4 Agonist & Potential First Disease-Modifying Biologic for Heart Failure Across the Ejection Fraction Spectrum
Synopsis
- Discussing JK07, an NRG1/ErbB3 antibody fusion, and the first selective ErbB4 agonist in clinical development for heart failure, with unique potential to increase contractility and reduce fibrosis in the myocardium
- Evaluating phase 1b data in heart failure with reduced ejection fraction (HFrEF) subjects where the potential to increase LVEF durably with a single, well-tolerated dose was demonstrated
- Updating on ongoing 282-subject Phase 2 study in HFrEF and heart failure with preserved ejection fraction (HFpEF), to assess repeat dosing of two dose levels and three dose regimens on myocardial function, quality of life, functional capacity, and cardiovascular outcomes
9:40 am Unlocking the Regulatory Genome to Advance Novel Precision Medicines for Heart Failure
Synopsis
- Discovering high-value LncRNA targets to reprogram diseased cell states in disease
- Understanding underlying LncRNA target biology to accelerate preclinical and clinical translation in the heart and beyond
- HTX-001 as a novel anti-fibrotic therapy for Non-obstructive Hypertrophic Cardiomyopathy
10:10 am Morning Break & Networking
10:30-11:00 am One-on-One Partnering Scheduling Block Four
11:10 am Panel Discussion: Beyond GLP-1: Developing Mono- & Combination Therapeutics to Bolster Targeted Weight loss
Synopsis
- Beyond the incretin space, what alternative receptors, signaling pathways, and metabolic processes can deliver superior efficacy?
- What role do modalities beyond small molecules play in developing future weight-loss treatments?
- Are there unique approaches to mitigate the risk of rebound weight gain after treatment discontinuation?
- How to address co-existing comorbidities such as diabetes, cardiovascular, and renal disease?
- How are you positioning new therapies to compete with established incretin-based solutions in the market?
11:40 am Human Genetics Driven Drug Discovery for Cardio-Renal & Metabolic Disease
Synopsis
- Exemplifying the value of human genetics for selection of therapeutic targets leading to increased clinical success rates
- Explaining Variant Bio’s differentiated approach and opportunities for partnerships
- Sharing examples of novel approaches (including kidney disease-focused) based on genetic support
12:10 pm Innovating Approaches to Manage Metabolic Risk Factors with Novel Diabetes Therapies Delivering Adipose Tissue-Specific Weight Loss & Improve Tolerability
Synopsis
- Achieving metabolic benefits from adipose tissue selective weight loss
- Meaningfully avoiding yo-yo weight loss regimes accelerate the progression to sarcopenia
- Answering the question: is there any such thing as a durable weight loss?
12:40 pm Lunch Break & Networking
1:00-2:00 pm One-on-One Partnering Scheduling Block Five
Innovations in Delivery & Administration to Extend Dosage Intervals for Sustained Therapy & Improved Patient Compliance
2:10 pm Enhancing the Bioavailability of RNA-based Therapies with Nanoparticle Innovations for Precise Protein Modulation in Kidney Diseases
Synopsis
- Overcoming challenges of genetic medicine delivery to the kidney
- Updating on the Judo development candidate components
- Explaining initial platform validation and therapeutic applications
2:40 pm Panel Discussion: Bypassing Injectables: Exploring Oral Innovations to Increase Patient Compliance
Synopsis
- How to maintain comparable efficacy and achieve oral bioavailability?
- What are manufacturing and supply chain nuances- how can these be addressed to ensure adequate and cost-efficient market supply?