Explore the Agenda
7:30 am Check In & Light Breakfast
8:20 am Program Directors Opening Remarks
8:25 am Chairs Opening Remarks
Entering the RNAi-Based Therapeutics Revolution to Treat Symptoms & Reprogram their Cause
8:30 am Reversing Time: Exploring the Utility of RNAi for Anti-Aging Applications
- Evaluating multi-target RNAi cocktails to modulate inflammatory and aging pathways, offering personalized skincare solutions
- Using topical delivery vehicles enhance skin penetration, enabling non-invasive treatment of wrinkles and other aspects of aging
- Demonstrating the Paradigm shift that RNAi-Based Therapies Can Be for Modern Skin Health Applications
Expanding Horizons in RNAi Therapeutics to Develop Medicines for Systemic & Extrahepatic Diseases
9:00 am Beyond Appetite: Extrahepatic RNAi to Transform Obesity & Metabolic Disease – Dual siRNA Reprogramming Fat for Durable, Muscle- Enhancing Weight Loss
- Exploring advances in extrahepatic dual RNAi delivery to enable targeted delivery to adipose tissue for treating the root cause of metabolic disease
- Demonstrating preclinical efficacy through human adipocyte validation to provide robust proof-of-concept and de-risk clinical translation
- Discussing how dual-target RNAi can expand beyond obesity into MASLD/MASH and cardiometabolic health to systemically improve metabolic parameters
- Positioning multi-target RNAi as the next wave beyond appetite suppression to move beyond the limitations of central appetite suppression and offer a more durable, tissue-specific, and holistic solution for sustainable weight loss
9:30 am Engineering a Dual-Targeting siRNA Therapeutic with AI-Designed Ligands to Treat the Underlying Biology of Sarcopenia
- Introducing a novel protein-conjugated siRNA modality for muscle that enables targeted delivery to skeletal muscle to overcome limitations of current antibody-based inhibitors, while avoiding off-target tissues
- Leveraging dual-targeting to silence redundant nodes in the inhibitory signalling pathway of muscle growth, moving beyond single-target inhibition to achieve functional recovery
- Demonstrating in vivo delivery, potent target knockdown, and significant functional improvement in models of muscle wasting
10:00 am Conquering Cardiac Fibrosis: A New RNAi Paradigm for Heart Failure
- Leveraging highly specific RNAi therapeutics, directly silencing key pro-fibrotic genes, to halt the underlying disease progression and move beyond symptom management
- Utilizing a novel, targeted delivery platform, enabling efficient extrahepatic delivery to cardiac tissue, to achieve robust efficacy and open new treatment possibilities for patients
- Addressing the root cause of pathological remodeling, by precisely reducing fibrosis and hypertrophy, to restore cardiac function and significantly improve patient quality of life
10:30 am Speed Networking
Put a face to a name: this session is the perfect opportunity to get face-to-face time with key opinion leaders, leading companies, and innovative researchers in the RNAi-based therapeutics field. Establish meaningful connections to build upon for the rest of the conference and gain individual insight beyond the papers and press releases into pioneering research and technique applications.
11:00 am Morning Break
Silencing the Drivers of Fibrosis: From Systemic Challenges to Precision Therapies
11:30 am Examining the Common Challenges of RNAi Therapies in Immunology and Fibrosis to Improve Targeted Delivery and Increase Biodistribution
- Engineering immune cell and fibroblast-targeted LNPs or antibodies to deliver RNAi payloads, reducing cytokine-driven inflammation in autoimmune disorders
- Co-administering siRNA to produce better a therapeutic response
- Optimizing biodistribution for fibrotic tissues via novel chemistries, reversing organ fibrosis with localized effects
12:00 pm A combinatorial, Unimolecular siRNA Approach for the Treatment of Fibrodysplasia Ossificans Progressiva
- Novel Hydrophobic divalent architecture for delivery to the muscle
- Allele specific targeting with siRNAs
- Prevention of severe disease phenotype in mouse model of FOP
12:30 pm Leveraging an RNAi Solution for a Novel Integrin in Chronic Fibrosis
- Leveraging self-delivering siRNA technology to silence a novel integrin target that drives the pathological switch to chronic fibrosis
- Unlocking regenerative healing by reversing myofibroblast persistence
- Validating a new treatment paradigm to demonstrate potent target knockdown and functional recovery in models of fibrotic disease
1:00 pm Lunch & Networking
Paradigm Shifts: Applying RNAi to Chemo-Resistance and Heart Failure
2:00 pm Highlighting miRNA’s Increased Oncological Efficiency in Treating Chemotherapy-Resistant Cancers
- Exploiting multi-gene silencing by miRNA to disrupt cancer pathways, overcoming chemo-resistance in aggressive tumours
- Deploying tumour-homing vehicle free delivery for precise miRNA uptake, sparing healthy tissues and reducing toxicity
- Showing high safety and activity in Phase I clinical trials with miRNA-15a positioning it as a first-in-class oncology RNAi drug
2:30 pm Testing Novel Drug Design Engines for Cancer for Improved Efficacy and Toxicity Mitigation
- Exploring the challenges of delivering RNAi to cancer
- Evaluating potential strategies to enable the development of RNAi-based drugs
- Presenting examples of emerging RNAi-based therapeutics for cancer
Breaking Barriers in RNAi Delivery to the Brain & CNS: From Innovation to Clinical Translation
3:00 pm Overcoming the Blood-Brain Barrier Challenge on the Trojan Horse
- Utilizing the advanced Trojan Horse formulations to enhance blood-brain barrier penetration, enabling systemic delivery of RNAi therapeutics for neurodegenerative diseases with gain in function
- Engineering receptor-mediated transcytosis to selectively transport siRNA and ASOs into the CNS, reducing off-target effects and improving therapeutic precision
- Demonstrating preclinical efficacy with a primary focus on Huntington’s disease, achieving significant knockdown of huntingtin protein and paving a clear path toward clinical development for severe neurological disorders
3:30 pm Afternoon Break & Poster Session
Connect with peers in a relaxed atmosphere and continue to forge new and existing relationships while exploring the latest in RNAi-based therapies and research advancements.
To submit a poster, please contact info@hansonwade.com
Optimizing RNAi Therapeutics for Advanced CMC & Formulation Strategies Streamline Scale-Up and Manufacturing
4:30 pm Engineering Self-Assembling RNA NanoStructures for Improved Precision Delivery in RNAi Therapeutic Delivery
- Developing programmable RNA origami structures that self-assemble into precise nanocarriers enabling tissue-specific delivery with reduced off-target effects
- Incorporating conditional stability triggers that respond to cellular microenvironment cues for controlled payload release at disease sites
- Demonstrating encapsulation efficiency through optimized nanostructure design significantly improving therapeutic payload delivery
Understanding Investor Insights to Define Success in RNAi Therapeutics for de-risking development strategies
5:00 pm Panel Discussion: Understanding RNAi Therapeutic Insights from Investors to Identify High-Value Assets and Maximizing Investment Returns
The RNAi therapeutics landscape is rapidly evolving, with breakthroughs in delivery, efficacy, and commercialization reshaping investor expectations. Key discussion points will include clinical differentiation, market potential, and exit strategies, offering a reality check on valuation drivers and red flags.
- Prioritizing delivery platform versatility as a key value driver, enabling investors to back RNAi companies with broad therapeutic applicability
- Assessing clinical milestones beyond knockdown efficiency, including durability and patient-centric endpoints, to identify therapies with superior commercial potential
- Balancing platform validation with indication selection for optimal risk/reward, ensuring portfolio companies can attract pharma partnerships or IPO success