Thursday, September 24 - EST (Eastern Time, UTC-05:00
Presents a computational pipeline for miR-128 antisense oligonucleotide design in post–MI heart failure, integrating thermodynamics, target accessibility, transcriptome-wide off-target modeling, and LNA/2′-O/PS chemistry optimization to select candidates for in vitro/in vivo testing of remodeling, function, and safety
- Anders Näär - Professor of Metabolic Biology, UC Berkeley
- GBFsen development journey: from genetic target identification to custom antisense design, preclinical validation, and translational challenges in developing individualized therapies for familial ALS patients with specific mutations
- Clinical evidence and future directions: preliminary trial results, biomarker strategies for monitoring therapeutic response, manufacturing and delivery considerations, and implications for expanding personalized antisense approaches in rare neurological disorders
- Tamar Grossman - CEO, La Jolla Labs
- The n-of-1 paradigm shift in rare disease treatment: exploring the scientific, regulatory, and ethical framework for developing patient-specific RNA therapies, including antisense oligonucleotides and siRNA approaches tailored to unique genetic mutations
Operational and translational challenges: addressing rapid drug development timelines, streamlined manufacturing for single-patient therapies, adaptive
- Konstantina Skourti-Stathaki - Senior Director, ASO Discovery and Research, n-Lorem Foundation
Please contact Jennifer Wickett for more information: Jennifer.Wickett@informa.com
In-cell selection methodology for identifying small molecules that bind to RNA targets within living cells, enabling discovery of compounds that can effectively reach and engage RNA in the complex cellular environment rather than only in vitro conditions
Application of this approach to develop RNA-targeted therapeutics with improved cellular activity and specificity
- Kevin Weeks - Kenan Distinguished Professor of Chemistry, UNC Chapel Hill
Exploring small nucleolar RNAs (snoRNAs) as programmable tools to control protein localization within cells, leveraging the natural RNA-guided mechanisms of snoRNAs to direct proteins to specific subcellular compartments
Extending this snoRNA-based system to regulate protein localization both intracellularly and extracellularly, offering a novel platform for spatial control of protein function with potential applications in targeted therapeutics and cellular engineering
- Tao Pao - Professor of Biochemistry & Molecular Biology, Univerity of Chicago
Exploring development of covalent chemical strategies to selectively target RNA molecules.
Application of covalent modification approaches to overcome selectivity challenges in RNA-targeted drug discovery, enabling more precise targeting of disease-relevant RNAs while minimizing off-target effects
Please contact Jennifer Wickett for more information: Jennifer.Wickett@informa.com
- Cory Sago - CEO, Amplitude Therapeutics
- David Hardwicke - Co-Founder, Aerska
- Bakhytar Ali - CEO, Amirxa Pharma
Please contact Jennifer Wickett for more information: Jennifer.Wickett@informa.com
Please contact Jennifer Wickett for more information: Jennifer.Wickett@informa.com
Please contact Jennifer Wickett for more information: Jennifer.Wickett@informa.com
- AI-driven ASO design and optimization: leveraging machine learning algorithms to predict target accessibility, minimize off-target effects, enhance binding affinity, and optimize chemical modifications for improved efficacy, safety, and pharmacokinetic properties
- Accelerating development and enabling scalability: utilizing AI platforms to compress discovery timelines, prioritize lead candidates, predict clinical outcomes, and streamline manufacturing processes for cost-effective production of diverse ASO therapeutics
- Bao Cai, PhD - Executive Director, Process Development, Sarepta Therapeutics
- PMO synthesis and conjugation strategy: developing scalable manufacturing processes for phosphorodiamidate morpholino oligomer (PMO) production, antibody-oligonucleotide conjugate (AOC) assembly, purification methodologies, and quality control measures to ensure consistent drug substance for Duchenne muscular dystrophy treatment
- Analytical characterization and CMC considerations: comprehensive physicochemical profiling, stability studies, potency assays, impurity assessment, and establishing critical quality attributes to support IND filing and clinical development of the AOC-PMO therapeutic candidate
- Mitch Martini - Associate Director, AOC Process Development & Manufacturing, Avidity Biosciences
Peptide conjugation offers opportunities to deliver oligonucleotides to specific cells or tissues and to enhance their drug-like properties. However, the complexity of conjugation chemistry presents significant challenges in lead optimization and scale-up synthesis. Through case studies, this talk will highlight how WuXi TIDES has developed its expertise and platform to overcome these challenges, enabling an integrated approach to the synthesis and de novo discovery of Peptide-Oligo Conjugates (POC).
- Jun Zhou - Senior Director, Oligonucleotide Medicinal Chemistry, WuXi TIDES
- Addressing barriers such as endosomal escape, immunogenicity, manufacturing scalability, cost-effectiveness, biodistribution optimization, and regulatory considerations that impact the clinical development and commercialization of targeted RNA medicines
- Future directions and therapeutic opportunities: exploring next-generation technologies including organ-specific targeting beyond the liver, CNS delivery strategies, intracellular compartment-specific localization, combination approaches, and expanding the therapeutic window to unlock new disease applications for oligonucleotide and RNA-based therapeutics
- Vadim Dudkin, PhD - Founding Chief Technology Officer, Soufflé Therapeutics
- Ranjan Batra - CSO, Dyne Therapeutics
- Annette Bak, Ph.D. - Head of Advanced Drug Delivery, AstraZeneca
Development of mRNA-based immunotherapy platforms specifically designed to treat adult solid tumors and pediatric sarcomas, utilizing messenger RNA technology to stimulate immune responses against cancer cells in these challenging-to-treat malignancies
- Randall Hyer - Chief Executive Officer, Merlin Biotech
Development of self-amplifying RNA (saRNA) therapeutics with chemical modifications and sequence optimization to achieve sustained, durable protein expression.
- Joshua McGee - CSO, Keylicon Biosciences
Manufacturing success of RNA therapeutics depends on precise control of quality of raw materials, in vitro transcription (IVT) reaction conditions and purification strategies that apply across constructs. The presentation will outline a conceptual framework for mRNA and saRNA production that integrates rapid at-line analytics to monitor IVT kinetics, enabling data-driven endpoint selection and feed strategies that raise yield and batch-to-batch consistency. A Quality by Design approach maps critical IVT factors—NTP/Mg2+ ratio, polymerase selection, temperature and time, template design, and capping strategy—to critical quality attributes (CQAs) and cost, defining a robust design space that optimizes productivity without compromising quality.
Downstream processing requires scalable, high recovery purification tools and techniques that effectively remove double-stranded RNA, RNA fragments, residual reaction components (NTPs, T7), to reduce immunogenicity and increase cellular potency for both mRNA and saRNA. Affinity, multimodal and reverse-phase chromatography each offer technical advantages and practical challenges that have to be understood and managed in manufacturing environments.
Universal requirements (integrity, dsRNA burden, residuals) and format-specific needs (capping for mRNA; long-transcript handling and hydrolytic potential of saRNA) demand decision frameworks for selecting appropriate IVT strategies and purification approaches. The ultimate goal is a manufacturing approach that scales efficiently and accelerates onboarding of new RNA constructs—translating bench-top control into reliable, multi-product production platform.
- Rok Sekirnik - Head Process Development mRNA/pDNA, Sartorius BIA Separations
Exploration of circRNA as an emerging therapeutic modality with advantages over linear RNA, including enhanced stability due to resistance to degradation, prolonged expression, and reduced immunogenicity.
Discussion of circRNA design, production, and delivery challenges alongside clinical translation opportunities
- Peter Weinstein - CEO, Circurna
- Lubor Gaal - CFO, Circio AB
- Anna-Rose Welch - Editorial and Community Director Advancing RNA, Life Science Connect
An open Q&A session offering attendees the opportunity to engage directly with leading investors, gain insights into funding strategies, market trends, and what drives investment decisions in the RNA therapeutics space.
- Soyoung Park - General Partner, 1004 Venture Partners
- Overcoming delivery barriers in neuromuscular disorders: addressing challenges of oligonucleotide penetration into muscle tissue and motor neurons, exploring conjugate strategies (including antibody-oligonucleotide conjugates and peptide-mediated delivery), and leveraging receptor-mediated uptake mechanisms to enhance therapeutic distribution
- Delivery platform technologies and clinical translation: evaluating lipid nanoparticles, exosomes, cell-penetrating peptides, and tissue-specific targeting ligands to improve biodistribution, reduce systemic exposure, and maximize therapeutic efficacy in conditions such as Duchenne muscular dystrophy, spinal muscular atrophy, and myotonic dystrophy
- Ranjan Batra - CSO, Dyne Therapeutics
- Engineering BBB-crossing enzyme therapeutics: protein modification strategies (fusion proteins, antibody conjugates, or receptor-targeting domains) that enable systemic administration while achieving therapeutic enzyme levels in the central nervous system for MPS II patients with neurological involvement
- Translational pathway and clinical evidence: preclinical validation of brain penetration, dose optimization studies, early-phase clinical trial results showing CSF biomarker improvements, impact on neurodevelopmental outcomes, manufacturing and regulatory considerations, and potential for platform application to other lysosomal storage disorders with CNS pathology
- Next-generation targeting strategies: exploring advanced conjugation technologies including GalNAc for hepatocyte targeting, antibody-oligonucleotide conjugates for cell-specific delivery, aptamer-guided systems, and novel ligand-receptor pairs that enable precise tissue and cell-type selectivity beyond traditional approaches
- Overcoming off-target effects and improving therapeutic index: leveraging tissue-specific delivery to minimize systemic exposure, reduce toxicity, enhance intracellular uptake in target cells, and achieve superior pharmacological outcomes across diverse disease applications including metabolic disorders, oncology, and neurological conditions
- Jim Weterings - VP Head of Oligo Discovery, Bonito Sciences
Please contact Jennifer Wickett for more information: Jennifer.Wickett@informa.com
Exploring the application of transformer-based language models to mRNA design, demonstrating how AI can accelerate the optimization of codon sequences for improved vaccine and therapeutic performance.
- Sizhen Li - Computational Science Lead, Sanofi
Development of stabilized nanoparticle formulations designed to enable multiple routes of mRNA delivery beyond traditional intravenous administration, including inhaled, topical, and other localized delivery methods to expand therapeutic applications.
Engineering nanoparticle platforms with enhanced stability and tissue-specific targeting capabilities, allowing mRNA therapeutics to be delivered directly to disease sites through diverse administration routes while maintaining cargo integrity.
Development of tissue-specific lipid nanoparticle (LNP) formulations engineered to selectively deliver mRNA therapeutics to muscle tissue, bone marrow, and immune cells, utilizing targeted delivery strategies to enhance therapeutic efficacy and reduce systemic exposure
- 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, Sunny Suite, Unchained Labs
An open Q&A session providing attendees the chance to interact with pharma leaders, explore industry perspectives, and discuss strategies for advancing RNA therapeutics from discovery to commercialization.
- Elena Diez Cecilia - Senior Director External Innovation DPDS, Johnson & Johnson
- Shaan Gandhi - VP and Head of Partnerships, PAVE, Pfizer
| Table 1: Using AI for Oligonucleotide Target Selection, Design, and Synthesis | Table 6: Extrahepatic targeted delivery of mRNAs Cargos |
| Table 2: Preclinical Challenges in RNA Research: Bridging Translational Gaps with Human-Relevant and Non-Animal Models & Immunogenicity | Table 7: Overlooked Opportunities in mRNA/mRNA 2.0 |
| Table 3: Extrahepatic targeted delivery of short RNAs Cargo | Table 8: Optimizing mRNA Design for Enhanced Potency |
| Table 4: Advancements in large-scale oligonucleotide manufacturing: Meeting the demand | Table 9: Scaling RNA Therapeutics: Overcoming Manufacturing Challenges for Clinical and Commercial Success |
| Table 5: Emerging trends in Oligonucleotides | Table 10: Commercialization |
- Yacoub Habib - CEO, Ophidion, Inc.
- Seema Zaidi - Global Business Development – Oligonucleotides, Bachem
- Jeff Milton - CEO, La Jolla Labs
- Praveen Kumar Pogula - Director, Drug Substance,, QurAlis