Thursday, September 24 - EST (Eastern Time, UTC-05:00
Steric-blocking LNA oligonucleotides offer a powerful therapeutic strategy for modulating RNA function without recruiting RNase H or RISC-mediated RNA cleavage. This presentation will describe an AI-guided framework for designing LNA/DNA mixmers that target therapeutically relevant noncoding and coding RNA biology, with emphasis on extrahepatic applications. The approach integrates target biology, RNA accessibility, LNA patterning, potency prediction, toxicity filtering, and tissue-delivery considerations to nominate candidates with improved probability of functional activity. Case examples will highlight how this framework is being applied to anti-miR programs in cardiovascular and retinal disease, as well as targeting "undruggable" oncogenes in cancers. More broadly, the talk will discuss where AI can practically improve oligonucleotide drug discovery by reducing search space, improving candidate selection, and linking ASO design decisions to disease biology and delivery strategies.
- Anders Näär - Professor of Metabolic Biology, UC Berkeley
Discover how La Jolla Labs achieved first-in-human treatment with GBFsen in under one year, demonstrating a breakthrough approach to rapidly translating patient-specific genetic discoveries into individualized ASO therapies for familial ALS.
Learn how AI-enabled drug discovery platforms can accelerate precision medicine development, gaining insights into the selection of highly selective antisense oligonucleotide candidates that target rare TDP-43 mutations in inherited neurodegenerative diseases.
Explore a transformative development model that bypasses traditional commercial frameworks, understanding how this cost-effective, scientifically rigorous approach can serve as a blueprint for creating single-patient therapies in ultra-rare genetic neurological conditions.
- Tamar Grossman - CEO, La Jolla Labs
- Precision RNA medicines are redefining what is possible for patients with ultra-rare genetic diseases. At the n-Lorem Foundation, we are building a scalable framework for individualized antisense oligonucleotide (ASO) therapeutics, translating decades of RNA-targeted drug development experience into real-world treatments for patients who otherwise have no therapeutic options.
- As the world’s first non-profit organization dedicated to providing free, lifetime personalized ASO therapies, n-Lorem has now evaluated more than 450 patient applications, advanced over 240 patient-specific discovery programs, submitted more than 40 INDs, and is currently treating over 50 patients across neurodevelopmental, neurodegenerative, ocular, and kidney diseases, with emerging clinical benefits observed across multiple programs.
- In this presentation, I will discuss how individualized RNA medicines are evolving from isolated ‘n-of-1’ efforts into a scalable therapeutic paradigm enabled by platform innovation, translational science, regulatory experience, and deep drug discovery expertise. I will highlight key lessons learned across active patient programs, including allele-selective targeting, splicing modulation, transcript knockdown, and emerging upregulation strategies.
- The insights gained from these individualized programs are not only transforming the lives of ultra-rare patients, but are also beginning to shape the future of precision RNA medicine globally.
- Konstantina Skourti-Stathaki - Vice President, ASO Discovery & Research, n-Lorem Foundation
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
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 Pan - 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
- Brittany Morgan - Professor, University of Notre Dame
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
Please contact Jennifer Wickett for more information: Jennifer.Wickett@informa.com
- Cory Sago - CEO, Amplitude Therapeutics
- David Hardwicke - Co-Founder, Aerska
- Bakhtyar Ali - CEO, Amrixa Pharmaceuticals
- David Weinberg - CEO, ParcelBio
- RNA-based therapeutics and other advanced modalities are often limited by rapid hepatic uptake and clearance, reducing systemic bioavailability, restricting delivery beyond the liver, and contributing to liver-associated toxicity. These delivery barriers can constrain dose, efficacy, and therapeutic window.
- Nanobiotix’s Nanoprimer is a biocompatible, engineered liposome platform designed to transiently occupy cells of the mononuclear phagocyte system involved in hepatic clearance. Administered shortly before the therapeutic agent, Nanoprimer is designed to reduce liver elimination during the agent’s critical window of activity, increasing systemic exposure and improving the opportunity for target-tissue delivery.
- Multiple preclinical proof-of-concept studies have been demonstrated across modalities, including nucleic acid-LNPs, liposomes, oncolytic viruses, and recombinant proteins. By decreasing liver clearance, Nanoprimer has shown the potential to enhance efficacy, improve extrahepatic exposure, and reduce hepatotoxicity for therapies whose performance is limited by rapid hepatic uptake.
For years, Samyang Biopharm has successfully commercialized surgical sutures and modified anti-cancer generics based on its biodegradable polymers and polymeric-micelle formulation technology. Selectivity Enabling NanoShell (SENS™), is Samyang’s latest innovative lipid- biodegradable polymer hybrid technology optimized for tissue-selective delivery of nucleic acid therapeutics. SENS™ platform includes formulations targeting lung, muscle, liver, CNS, etc. tissues and specific cell types, including epithelial, endothelial, tumor, hepatocytes and neurons.
NanoReady SENS™ formulation is characterized by high splenic DC cell selectivity and its ready-to-use format. As a pre-made nanoparticle system, it can simply be combined with mRNA molecule before use in patient, which allows to streamline process development, reduce time-to-patient, and enable development of personalized cancer vaccines.
By pursuing active collaborations with global partners, Samyang is expanding SENS™ platform applications to various nucleic acid-based therapeutics, including cancer vaccines, gene therapies, tissue-specific gene regulation, gene editing, protein replacement, and more.
- Natalia Ulyanova - ead of US Business and Development and Licensing, Samyang Biopharm USA
Please contact Jennifer Wickett for more information: Jennifer.Wickett@informa.com
- 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
- 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
As oligonucleotide therapeutics evolve from early-stage candidates into complex clinical modalities, establishing a robust, phase-appropriate analytical control strategy is paramount for both technical execution and critical business milestones. This presentation provides a strategic overview of the analytical challenges defining modern Oligo Chemistry, Manufacturing, and Controls (CMC) projects, highlighting the implementation of platform analytical technologies designed to accelerate early-stage development.
Addressing the structural and process complexities of next generation oligos, this presentation delves into the intricacies of de novo method development. We will explore targeted solutions for distinct analytical hurdles, including the management of reactive linkers, monitoring of residual enzymes following enzymatic ligation, and the specialized characterization required for complex modalities such as oligo-peptide conjugates.
To support rigorous process development and scale-up, the presentation emphasizes the deployment of advanced instrumentation. We will outline how High-Resolution Mass Spectrometry (HRMS) is leveraged for impurity profiling, confirming peak purity, and quantifying impurities to ensure product quality and regulatory readiness.
Finally, bridging the gap between bench-level data and commercial readiness, this talk details best practices for analytical validation and the establishment of compliant release strategies. By integrating these high-resolution tools, our strategy supports the timely, compliant delivery of high-quality therapeutics to market.
- Constanze Schmies - Group Leader QC, Bachem AG
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
Please contact Jennifer Wickett for more information: Jennifer.Wickett@informa.com
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
- Max Colbert, MD, MBA - Senior Associate, Medical Excellence Capital
- Bao Cai, PhD - Executive Director, Process Development, Sarepta Therapeutics
- Paloma Giangrande, Ph.D. - VP Platform Discovery Sciences, Biology, Wave Life Sciences
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
- 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
- Pedro Morais - Preclinical Development Lead DMPK, Bayer
- 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.
- Philipp Schreppel - Senior Director Business Development, Ethris
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
Lipid nanoparticles are at the heart of nanomedicine development, but moving from early formulation screening to a production-ready process can be time consuming, sample-hungry and operationally complex. In this talk, we’ll explore the evolving LNP synthesis needs across the nanomedicine development journey and show how the combination of Sunny Suite and Stunner from Unchained Labs helps teams step up their nanoparticle game. From low-volume, automated screening that conserves precious reagents, to high-throughput characterization that provides encapsulation efficiency at the same time as sizing, join us to see how the right tools can simplify development, accelerate iteration and help turn promising nanoparticle ideas into real therapeutic progress.
- Ben Knappett - Sr. 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
- Maj Petersen, PhD - Senior Director of Innovation and Project Execution, Novo Nordisk
Think you know RNA? Put your knowledge to the test! Join us for RNA Showdown: a fast-paced trivia challenge covering everything from basic biology to cutting-edge therapeutics and maybe a little local Boston history as well. Compete, learn, and win prizes!