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Day 1- Wednesday September 10th - EST (Eastern Time, UTC-05:00
- Michael Hodges - CEO, 1strand San Diego County RNA cluster
Explore cutting-edge advancements in oligonucleotide synthesis, manufacturing, and delivery technologies
Discuss strategies for overcoming key challenges in scaling oligo-based therapeutics from research to commercial production
Examine innovative approaches to improve the efficacy, safety, and accessibility of siRNAs and antisense oligonucleotides
- Paul Nioi - SVP, Research, Alnylam
- Tao Pei, PhD - Senior Vice President, Chemistry, Arrowhead Pharmaceuticals
- Alison Moore - Chief Technical Officer, Codexis
- James Powell - VP RNAi CMC, Boulder Site Head, Novo Nordisk
- Michael Hodges - CEO, 1strand San Diego County RNA cluster
What steps must be taken to implement time-saving technologies for automation and AI-driven processes to improve global access?
How is the industry moving to improve cold-chain solutions?
What actions must be taken to ensure greener analytical techniques?
Do new considerations new to be made for production of mRNA 2.0 technologies?
- Patrick Thiaville - VP, 4basebio
- Thaminda Ramanayake - CBO, CureVac
- Clay Alspach - Executive Director, Alliance for mRNA Medicines
- Michelle Hall - Associate VP - Genetic Medicine Research, Eli Lilly & Company
- The chemistry of oligonucleotides has played a crucial role in giving drug-like properties to oligonucleotides.
- To optimize oligonucleotide-based therapeutics, we explored oligonucleotide shapes that bring the 3'- and 5'-ends into transient cyclic structures.
- Oligonucleotide spatial structures exhibit reduced protein binding because of their shape and diminished interactions with pattern recognition receptors resulting from the inaccessibility of the 5’-end.
- These designs find extensive applications across diverse mechanisms of action for RNA therapeutics, including RNaseH-mediated knockdown, modulation of splicing, siRNA, and gRNA.
- Sudhir Agrawal - President and Founder, Arnay Sciences
Presenting a comprehensive platform supporting nucleic acid therapeutic discovery and development at all stages.
A one-stop service offers monomer and oligonucleotide synthesis for a broad range of nucleic acid therapeutics, including special modifications, chiral oligos, and oligo-conjugations
Sharing expertise in formulation, state-of-the-art in vitro bioassays, toxicity testing and in vivo PoC can further accelerate the discovery and development processes
- Dave Madge, PhD - Vice President, Research Services Division, WuXi AppTec
• Illuminate the critical need for standardization in the rapidly evolving field of oligonucleotide therapeutics, exploring how establishing uniform guidelines for design, manufacturing, and quality control can accelerate drug development, enhance regulatory compliance, and improve the overall safety and efficacy of these innovative treatments.
• Delve into the collaborative efforts of industry leaders, regulatory bodies, and academic institutions to develop consensus-driven standards for oligonucleotide therapeutics, highlighting key areas of focus such as analytical methods, impurity profiling, stability testing, and in vivo delivery assessment, while discussing the potential impact of these standards on streamlining clinical trials and facilitating faster market approval processes.
- Mark Lowenthal - Research Chemist, National Institute of Standards and Technology
Developing a novel reactor system that co-immobilizes T7 polymerase and DNA templates, enabling efficient, high-purity mRNA synthesis with reduced dsRNA impurities
Enabling scalable and cost-effective, streamlined mRNA therapeutics manufacturing
- Craig Martin - Professor, University of Massachusetts Amherst
- Adoption of RNA as a vaccine and therapeutic modality highlights the need for robust, cost-effective production and improved targeted delivery.
- Look into how a rapid and efficient manufacturing process that combined with in-process analytics enable achieving IVT yields up to 25 g of RNA per L and attaining 80-100 % of RNA recovery after purification.
- Discover how innovating mRNA-LNP formulation supported by in-process analytics can lead to the generation of mRNA LNPs that have expanded extrahepatic biodistribution, improved drug delivery and cellular targeting.
- Tomas Kostelec - Manager of Segment Technology for Advanced Therapies, Sartorius
- Single-use biochips eliminate patient-to-patient risk of cross-contamination
- Personalized manufacturing and release process with NMU-Symphony™
- From RNA-design to patient-injectable vials in 3 weeks
- Babak Sanii - Director of Biochip R&D, Nutcracker Therapeutics
Discuss recent discoveries highlighting the diverse roles of RNAs, with a focus on microRNAs in gene regulation and cellular processes beyond canonical translational repression.
Discuss the involvement of microRNAs in complex biological pathways and their implications for human health and disease.
Discuss the potential of microRNA-based diagnostics across various
- Attila Seyhan - Director of Translational Oncology Operations at Cancer Center, Brown University
Jaan Biotherapeutics Inc. is a private company that is developing novel therapies to activate the cardiac regeneration process in diseased hearts using proprietary technology.
The technology manipulates microRNAs to activate an endogenous cardiac muscle regeneration process which has been shut down in the adult human heart during evolution.Our current focus is Ischemic Heart Disease (IHD), but the therapy can be applied to many cardiac diseases where cardiac muscle regeneration is required.
- Bhawanjit Brar - CEO, Jaan Biotherapeutics
- Mikko Turunen, PhD - Founder and CSO, RNatives Inc.
- Graham Taylor - Founder & President, Helix Biotech
The n-Lorem Foundation is pioneering a new paradigm in the development of individualized medicines for patients with ultra-rare genetic diseases. As a unique non-profit biotech, n-Lorem provides free, lifetime treatment to patients who would otherwise be invisible to traditional drug development pathways- those with mutations found in one or a few individuals worldwide.
In this talk, I will share how we are building a scalable and sustainable model that challenges the conventions of therapeutic development. Since our inception, we have received over 330 applications, launched more than 160 patient-directed drug discovery programs, submitted 25+ INDs, and treating 27 patients today. Our approach is deeply rooted in our over 35-year of experience in RNA-targeted therapies, especially antisense oligonucleotides (ASOs), offering precision tools that match the urgency and specificity of each patient’s condition.
I will provide an overview of our mission and model, outline our end-to-end discovery and development process, and discuss the scientific considerations that shape our work. Most importantly, I will highlight how our close working relationship with the FDA, our unwavering focus on individual patients, and our deep scientific and clinical expertise are allowing us to deliver highly personalized RNA-targeted treatments where no other options exist.
- Konstantina Skourti-Stathaki - Senior Director, ASO Discovery and Research, n-Lorem Foundation
Divalent siRNA (di-siRNA), a novel small interfering RNA technology, has potency, CNS distribution, and durability appropriate for neurological disease
ATL-201, a di-siRNA targeting the KCNT1 sodium-activated potassium ion channel, gives selective knockdown of KCNT1 transcript and protein and reaches most neurons in the cortex following a dose into cerebrospinal fluid
ATL-201 gives dose-dependent reduction in seizures lasting over four months in mice with Kcnt1-driven epilepsy and shows promise for treatment of gain-of-function KCNT1 genetic epilepsy
- Garth Kinberger - SVP of Chemistry, Atalanta Therapeutics
- Jun Wang - Senior Machine Learning Scientist, Sanofi
- Overview of Endless RNA (eRNA)
- Key differences between mRNA and eRNA
- Development of a quantitative Anion Exchange HPLC method for eRNA purity
- Characterization of eRNA using orthogonal analytical methods
- Dinesh Manvar - Associate Director, Analytical Sciences, Sail Bio
EVE16 (Engineered Valency-Enhanced CD16A) is an immunoactivating receptor whose stability, surface expression, and function are dependent on the signaling adaptor proteins CD3ζ and FCER1G. These adaptors are selectively expressed in T cells, NK cells, and monocytes, restricting EVE16 expression to these immune cell types.
Targeted LNP delivery of EVE16-encoding mRNA enables in vivo expression specifically in immune cells, allowing for high-precision immune cell engineering.
EVE16 simultaneously interacts with a target receptor and the Fc domain of monoclonal antibodies, enabling synergistic activity with therapeutic mAbs.
- Nikola Ivica - Head of Discovery, InnDura Therapeutics
Exploring the use of ligand-siRNA conjugates to selectively silence SLC proteins in kidney cells.
Application of this novel approach to improve patient outcomes of systemic disease
- Alfica Sehgal, PhD - Chief Scientific Officer, Judo Bio
Pioneering the use of CRISPR technologies to modulate long non-coding RNAs driving cancer progression
- Rory Johnson - Associate Professor, University College Dublin
- Michael Khan - CEO, ARGONAUTE RNA LIMITED
RNAi is well-positioned to positively impact the lives of patients living with obesity and related metabolic disorders, due to its strong clinical track record of safety, efficacy and durability. As a modality, mRNA silencing enables pharmacological intervention in diverse disease-associated mechanisms including thermogenesis, lipogenesis, energy expenditure and inflammation. SanegeneBio is developing differentiated tissue-selective delivery technologies using our LEAD (Ligand and Enhancer Assisted Delivery) platform. In this presentation, we will share Phase 1 clinical data demonstrating potential for best-in-class siRNA payloads, our novel ligand-based approaches targeting adipocytes and skeletal muscle, and preclinical proof-of-concept for several obesity targets. Together, these data support the promise of RNAi-based medicines as a transformative modality in the treatment of obesity—capable of addressing limitations of existing therapies and establishing a new standard of care.
SUMMARY BULLETS:
- Novel ligand-mediated approaches for selectively targeting adipocytes, muscle and other metabolic compartments are needed to realize the potential of RNAi for obesity therapy
- LEAD technology represents a new ligand-and-enhancer conjugation approach, enabling improved specificity and potency for obesity targets spanning multiple biological mechanisms
- In this presentation, we will share clinical and preclinical data demonstrating potential for best-in-class RNAi payloads, differentiated pharmaceutical properties and therapeutic proof-of-concept
- Marc Abrams - CTO & Head of US Operations, SanegeneBio
For more information, please contact Jennifer Wicket at jennifer.wicket@informa.com
Explore the innovative Transport Vehicle Technology that leverages the transferrin receptor pathway for enhanced delivery of therapeutic enzymes and oligonucleotides across the blood-brain barrier, potentially revolutionizing treatments for neurological disorders.
Examine the mechanistic insights, preclinical evidence, and clinical potential of this targeted delivery approach, highlighting its ability to overcome traditional obstacles in CNS drug delivery and improve the efficacy of enzyme replacement therapies and oligonucleotide-based treatments for brain diseases.
- Explore innovative RNAi-lipid conjugates targeting immune cell PD-L1 for refractory cancer treatment, focusing on pre-clinical discovery and efficacy of a novel RNAi compound.
- Delve into the compound's mechanism of action and its potential to overcome immunotherapy resistance, highlighting promising pre-clinical results.
- Unveil the strategic clinical trial design, outlining the approach for translating this cutting-edge therapy from laboratory to patient care in challenging cancer cases.
- Shanthi Ganesh - Director, Novo Nordisk
For more information, please contact Jennifer Wicket at jennifer.wicket@informa.com
Explaining the role of lipoprotein(a) (Lp(a)) in cardiovascular risk, the importance of measuring Lp(a) levels and evaluating the potential benefits of Lp(a) lowering
Discussing the role of RNA-targeting therapies in addressing the cardiovascular risk associated with elevated Lp(a) levels
- YanLing He - Executive Director in Translational Medicine, Novartis
- Richard Shen - Co-Founder, EndoTope Bio
• Discover comprehensive solutions for the design, characterization, and optimization of RNA therapeutics, enhancing the development process from concept to clinical application.
• Learn about a state-of-the-art platform that offers in-depth characterization of therapeutic potency, safety, and purity, crucial factors for successful drug development in the RNA space.
• Understand the challenges posed by double-stranded RNA (dsRNA) by-products, which can decrease therapeutic stability and translation efficiency. Explore an advanced technology that identifies specific sequences and structures promoting dsRNA formation, enabling improved therapeutic design and efficacy.
- Wayne Doyle - Head of Platform, Eclipse Bioinnovations
Exploring a first-in-class USP6-based mRNA immunotherapy to upregulate immunostimulatory pathways associated with an anti-tumour immune response
- Randall Hyer - Chief Executive Officer, Merlin Biotech
- Biotech case study scaling mRNA manufacturing
- Industry case study on scaling from a systems perspective
- Panel and Group Discussion covering the following:
- What scaling/capacity thresholds prompt major changes in process and infrastructure?
- Do specialization needs change at certain scale thresholds?
- What are the top three considerations when evaluating current-scale readiness?
- Philipp Schreppel - Senior Director Business Development, Ethris
- Lynda Menard - Advisor, RibroPro
Discover a revolutionary Trojan horse technology that enables intravenous delivery of gene-silencing treatments to the brain. Gain insights into how this approach achieves uniform brain distribution at significantly lower doses compared to intrathecal methods, potentially transforming the treatment of neurological disorders.
Explore an innovative nanoparticle formation process that combines negatively-charged therapeutic cargoes with a proprietary peptide. Learn how these neutral nanoparticles interact with the blood-brain barrier to effectively deliver treatments to brain cells, opening new possibilities for targeted drug delivery.
Uncover cutting-edge optimization techniques for nanoparticle production, including the use of commercial equipment and the impact of particle size on brain uptake. Gain valuable knowledge about the relationship between dosage, brain uptake, and protein knock-down efficacy, potentially revolutionizing treatments for conditions like Huntington's disease
- Yacoub Habib - CEO, Ophidion, Inc.
For more information, please contact Jennifer Wicket at jennifer.wicket@informa.com
• Explore the shift from passive to active RNA delivery systems in precision medicine, examining nuances between delivery methods for siRNA, mRNA, and ASOs.
• Discover cutting-edge tissue targeting strategies, including lipid nanoparticles and conjugate technologies, enhancing specificity for different RNA modalities.
• Compare innovative delivery platforms like exosomes and engineered viral vectors, optimized for various RNA cargoes and therapeutic goals.
• Uncover the potential of active RNA delivery in transforming treatments for genetic disorders and cancer, discussing emerging hybrid systems for optimal targeting.
- Annette Bak, Ph.D. - Head of Advanced Drug Delivery, AstraZeneca