Day 1- Wednesday September 10th - EST (Eastern Time, UTC-05:00
- Jade Osei-Tutu - Head of RNA Leaders, Informa Connect
- Michael Hodges - CEO, 1strand San Diego County RNA cluster
Targeting microRNAs vs. siRNA/ASO: MicroRNAs regulate multiple genes simultaneously, offering comprehensive pathway control but with potential off-target effects. In contrast, siRNA/ASO technologies provide highly specific single-target precision with established delivery platforms and clinical validation.
Nobel Prize Recognition: The groundbreaking discovery of microRNAs and their regulatory functions was honored with the Nobel Prize in Physiology or Medicine. This prestigious recognition underscores the significance of microRNA research in advancing both basic science and clinical applications.
Investment Landscape: The microRNA therapeutics sector has experienced significant growth through mergers, acquisitions, and strategic investments from major pharmaceutical companies. Venture capital funding has accelerated the development of novel microRNA-based approaches for previously untreatable conditions across oncology, cardiovascular disease, and rare genetic disorders.
- Michael Hodges - CEO, 1strand San Diego County RNA cluster
- Bhawanjit Brar - CEO, Jaan Biotherapeutics
- Christine Esau, Ph.D. - Group Vice President, Arrowhead Pharmaceuticals
- Jim Weissman - Principal, JBW Consulting, LLC
- Malgorzata Gonciarz - Global Head xRNA Therapeutics, Novartis
- Bo Rode Hansen - Executive Chairman, Heqet Therapeutics
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
- Dmitry Samarsky - Chief Science Officer, ARNAgen Therapeutics
- Kaitlin Dailey - Program Leader, Informa Connect
- Miroslav Gasparek - CEO, Senisble Biotechnologies
Evaluate strategies for companies to progress RNA programs in growing markets, highlighting successful partnership models and the potential impact of next-generation technologies on the future of the RNA space.
Discuss critical steps for accelerating clinical progression of new RNA approaches, considering the shift from large-scale vaccine production to diverse, small-scale applications for cancer and rare diseases.
- Renee Williams - Managing Partner, Williams Biotech Consulting, LLC
- Samir Ounzain - CEO, HAYA Therapeutics
- Tamar Grossman - CEO, La Jolla Labs
- Jim Weterings, PhD - VP, Head of Oligonucleotide Therapeutics, Bonito Biosciences
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?
- Shyam Subramanian - VP, Technical Development and CMC Strategy, Moderna
- Danny Crawford - Senior Director, Early-Stage Process Development, Intellia Therapeutics
- Amy Walker - COO, 4Basebio
- Miroslav Gasparek - CEO, Senisble Biotechnologies
- Sandy Hinckley - Vice President, Head of Discovery, QurAlis
- 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
The transition from discovery to clinical manufacturing of oligonucleotide therapeutics can be hindered by fragmented workflows, supply chain vulnerabilities, and inefficient analytical method transfer. These issues can delay the progression of promising candidates. Through the selected case studies, we will explore how an integrated development framework can address these challenges with featuring harmonized process development, scalable oligonucleotide synthesis, and robust analytical method transfer. This approach would reduce the risk of raw material shortages and streamline analytical method transfer, and enables accelerated, reproducible, and high-quality advancement of oligonucleotide therapeutics toward clinical application.
- Yi Lei - Senior Manager, Discovery Chemistry Project Management, WuXi TIDES Discovery
- Mayurbhai Patel - Senior Director, Chemistry Development & CMC, Judo Bio
- Elvan Cavac - RNA Manufacturing Strategy & Partnerships, Cavac Consulting
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
Next-generation delivery systems with novel cationic lipids enable precise targeting and improved biodistribution for RNA/DNA therapeutics
Advanced manufacturing technologies, including microfluidic platforms, offer scalable and cost-effective LNP production across multiple scales
Innovative chromatographic purification using CIM® OH columns ensures high recovery while removing impurities from RNA-LNP products
The PATfix LNP Switcher analytical platform comprehensively characterizes LNP-based biopharmaceuticals, assessing critical quality attributes
- Ian Villamagna - Sales Specialist DNA RNA, Polyplus, now part of Sartorius
- Nejc Plavin - Project Manager, 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
- Jessica Kenison - Scientific Director, Immunocodex
- Michael Hodges - CEO, 1strand San Diego County RNA cluster
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
- Zdravka Medarova - CSO, TransCode Therapeutics
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
- Jeff Abbey - CEO, RNatives
- Elisbetta Morini - Assistant Professor of Neurology, Massachusetts General Hospital and Harvard Medical School
- Galnac ligand as core material of oligonucleotides play very import role in delivering the ONs to hepatic cell. Its quality shall affect the final ONs API quality. Huaren did much study on the Galnac ligand quality and can support our clients to file their product.
- Huaren has many years’ experiences on Galnac and amidites production. Huaren has good quality control system and GMP production platform
- Chen Yao - QA director, Wuhu Huaren
- Joshua McGee - CSO, Keylicon Biosciences
- Peter Berglund - CSO, HDT Bio
- Konstantina Skourti-Stathaki - Senior Director, ASO Discovery and Research, n-Lorem Foundation
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
- Jon Brice - Senior Vice President Business Development, Asymchem
- Garth Kinberger - SVP of Chemistry, Atalanta Therapeutics
- Ryan Sowell - Director, Strand Therapeutics
- Jun Wang - Senior Machine Learning Scientist, Sanofi
- Design potent, cell-specific therapies with eVERSE, a comprehensive database capturing key dimensions of RNA biology.
- De-risk development early using eMERGE, a sequencing-based platform for actionable RNA quality assessments.
- Track and manage quality over time with eCOMPASS, a web-based portal that connects discovery to validation.
- Peter Chu - CEO, Eclipse Bio
- Syed Ali - Senior Scientist, Moderna
- Elisbetta Morini - Assistant Professor of Neurology, Massachusetts General Hospital and Harvard Medical School
- 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
From screening early-stage nucleic acid payloads up to GMP-scale production, researchers need tools that grow up with their workflow and keep the process consistent.
Sunshine and Stunner AF team up to cover the critical steps in LNP development
See how this duo helps you hit the ground running with your first gene therapy, reduces hands-on time, and delivers more meaningful sample
- Ben Knappett - Product Manager, Unchained Labs
- Yacoub Habib - CEO, Ophidion, Inc.
- Maria del Carme Pons Royo, PhD - Postdoctoral Researcher, Massachusetts Institute of Technology
· Raina Biosciences developed GEMORNA, a deep generative AI platform that designs mRNA sequences with enhanced expression and stability.
· GEMORNA enables zero-shot design of both coding sequences (CDS) and untranslated regions (UTRs) with a high success rate, and is also applicable to circular RNA design.
· Extensive in vitro and in vivo validations demonstrate that GEMORNA-derived sequences achieve superior expression, durability, and potency across diverse therapeutic applications, including mRNA vaccines, gene therapy, and in vivo CAR-T.
- He Zhang - CTO, Raina Biosciences
With a UV flow cell with a 0.3mm pathlength it is possible to follow the consumption of the phosphoramidite during the coupling reaction.
Integration of the area under the conductivity peak generated during the oxidation step can be used to assess the efficiency of the coupling reaction. This can also be applied for thiolation using xanthane hydride.
- Łukasz Frankiewicz - Product Leader, PeptiSystems AB
- RNARISE™ Platform: A novel, non-viral self-amplifying RNA backbone, designed for safe and effective use in therapeutics and vaccines.
- AI-Assisted Design: Integration of advanced computational approaches to optimise efficacy, stability, and performance.
- Differentiation: Unlike viral-based saRNA systems, RNARISE™ is only 1.8 kbs in size, and close to human physiology.
- Ahmet Berkyurek - CEO, Cambridge Medixine Ltd.
Detection of RNA degradation under LNP formulation conditions
Measurement of structure and structure changes in a 5’ UTR due to ligand binding
- Scott Gorman - Application Scientist, RedShiftBio
- Garth Kinberger - SVP of Chemistry, Atalanta Therapeutics
Cardiovascular disease is the leading cause of death worldwide; heart failure carries high mortality, and substantial health-economic burden. Cardiac infarcts can lead to loss of heart function and heart failure. Cardiomyocytes have limited regenerative capacity and current therapies only improve residual function and are not curative.
Small non-coding microRNAs delivered as synthetic mimics in cardiomyocyte specific lipid nanoparticles can reactivate cardiomyocyte proliferation in the adult heart. Preclinical studies in mice and pigs demonstrate increased cardiomyocyte division, reduced infarct size, and improved function after myocardial infarction.
The miRNA–LNP approach provides potent, transient, and repeatable effects with efficient myocardial delivery; Compared with AAV, protein and cell therapies, miRNA–LNPs offer better control of dose and optimal duration.
miRNA-based regenerative therapy represents a promising, scalable strategy for curative treatment against heart failure, warranting further clinical development and safety evaluation.
- Bo Rode Hansen - Executive Chairman, Heqet Therapeutics
Delivery of RNAi to the central nervous system (CNS) has potential to treat a broad range of neurological disorders, but so far has been limited to intrathecal administration. Arrowhead Pharmaceuticals has developed an optimized CNS TRiM™ SC (Targeted RNAi Molecule, subcutaneous) platform which shows effective and long-lasting target inhibition throughout the CNS in preclinical species after systemic delivery. This platform can deliver siRNA to deep brain regions that weren’t feasible with intrathecal delivery, and the convenient administration route will enable application to larger patient populations such as Alzheimer’s and Parkinson’s disease. Preclinical data from several CNS TRiM™ SC programs that are currently moving into clinical development will be presented, including ARO-MAPT-SC, targeting Tau for the treatment of Alzheimer’s disease
- Christine Esau, Ph.D. - Group Vice President, Arrowhead Pharmaceuticals
- Annette Bak, Ph.D. - Head of Advanced Drug Delivery, AstraZeneca
- Yacoub Habib - CEO, Ophidion, Inc.
- Marc Abrams - CTO & Head of US Operations, SanegeneBio
- Andrea Engel - Head, Cell and Delivery Systems, Evonik
Presentation: Solving the Scale-Up Bottleneck: Integrated Systems for mRNA Therapeutics
Lynda Menard, RiboPro
Presentation: Stable and scalable mRNA therapeutics: Transforming Respiratory Medicine and Beyond
Philipp Schreppel, Director of Business Development, Ethris
Discussion:
Part 1: Strategic Scaling: Partnerships, CDMOs & Supply Chain
Focus: external-facing strategy, CDMO dynamics, partnerships, supply chain
Part 2: Tactical Scaling: Platforms, Process Design & Infrastructure
Focus: internal-facing decisions, platform evolution, technology, process shifts
Philipp Schreppel, Director of Business Development, Ethris
Lynda Menard, RiboPro
- Lynda Menard - Advisor, RibroPro
- Philipp Schreppel - Senior Director Business Development, Ethris
- Peter Berglund - CSO, HDT Bio