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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
- John Zuris, PhD - Principal, John Zuris Consulting LLC
- Familial dysautonomia (FD) is a fatal sensory and autonomic neuropathy caused by an intronic ELP1 mutation that disrupts pre-mRNA splicing. We developed a CRISPR cytosine base-editing strategy to permanently correct the disease-causing mutation and restore correct ELP1 splicing.
- Systematic optimization of base editors and guide RNAs achieved up to 70% correction in human cells. A dual-AAV intein-split base editor enabled in vivo correction in a humanized FD mouse model, increasing correct ELP1 splicing in the brain and other disease-relevant tissues with minimal detectable off-target editing.
- In FD patient-derived sympathetic neurons, even ~10% genetic correction was sufficient to rescue disease-associated neuronal hyperactivity, demonstrating that partial editing can produce meaningful functional rescue and supporting base editing as a potential one-time, disease-modifying therapy for FD while highlighting its broader therapeutic potential for human splicing disorders.
- Elisbetta Morini - Assistant Professor of Neurology, Massachusetts General Hospital and Harvard Medical School
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, University 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
As mRNA therapeutics expand into new applications, manufacturers must improve productivity while maintaining quality and controlling costs. This presentation highlights how a process solution driven approach to mRNA production that combines real-time IVT monitoring, Design of Experiments (DoE), and innovative purification strategies can increase mRNA manufacturing efficiency and quality. Attendees will learn how mRNA titers of up to 25 g/L were achieved alongside improved cost efficiency, and how a novel aqueous dsRNA removal approach combined with Oligo dT chromatography supports high product purity and recovery.
- Rok Sekirnik - Head Process Development mRNA/pDNA, Sartorius BIA Separations
- Ethan Morgan - PhD Biological Sciences, University of Durham
- Cory Sago - CEO, Amplitude Therapeutics
- 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 - Head of US Business and Development and Licensing, Samyang Biopharm USA
Traditional RNA drug development relies on costly trial-and-error design, often treating biological complexities as isolated problems. Therna Bio is redefining this paradigm through RNA-Logix™, an AI-native platform powered by our proprietary AI RNA Biologist. Built on one of the industry’s largest dataset of whole-system biological measurements generated in-house, our recursive AI models evaluate full RNA structures simultaneously rather than piece by piece. This enables Therna to address both sides of the RNA spectrum: designing synthetic mRNA therapeutics with enhanced stability, cell specificity, and protein expression, as well as optimizing oligonucleotides (ASO/siRNA) to precisely silence, edit, or activate target genes. By accelerating discovery from years to months at a fraction of traditional costs, Therna’s end-to-end engine converts complex RNA biology into scalable, high-value clinical opportunities and strategic partnerships.
- Amir Moarefi - Head of Partnering, Therna Biosciences
Zipcode Bio was built on a simple premise: solving delivery to a new tissue doesn't yield one drug, it yields a portfolio. Founded with Nobel laureate Drew Weissman and a delivery team out of BioNTech, the company pairs AI-driven RNA and LNP design — two Nature Machine Intelligence papers to date — with a cutting-edge nanoparticle platform now reaching five organs. Co-founder and CEO Jason Zhang will show how programs in oncology, in vivo CAR-T, and immune aging emerged directly from delivery findings — and where the platform is open to partnership
- Jason Zhang, PhD - Co-founder and CEO, Zipcode Bio
- 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
- Alan Whitmore, PhD - Chief Scientific Officer, Tangram 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
- David Bumcrot, Ph.D. - Scientific Fellow, CAMP4 Therapeutics
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
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 - Chief Financial Officer and Head of Business Development, Circio AB
- Andrew Varley - RNA & Formulation Core Director, The University of British Columbia
- John Zuris, PhD - Principal, John Zuris Consulting LLC
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.
- Max Colbert, MD, MBA - Senior Associate, Medical Excellence Capital
- Mark Springel - Principal, Vida Ventures
- Phil Lavin - Founder, Melior Capital Management
- Paloma Giangrande - President, BioLogic Insights LLC
- 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
- Moving oligonucleotide conjugates beyond the liver widens the target space but adds translational uncertainty: my presentation walks through changes in the preclinical package.
- The talk then turns to de-risking: how human-relevant and genetically informed translational models can close critical gaps in both target and delivery biology, and how the intended conjugate format should drive test article and species selection in nonclinical safety studies.
- 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
A proprietary polymer-based platform utilizes LAT1-targeting ligands to transport oligonucleotide therapeutics across the intact blood-brain barrier following intravenous administration, with demonstrated penetration in both healthy and disease models.
The lead epilepsy asset combines a novel anti-miR antisense oligonucleotide with the delivery platform, achieving anti-seizure efficacy lasting more than 10 weeks after a single intravenous dose across multiple epilepsy models.
Preclinical studies have demonstrated synergistic anti-seizure effects when the lead asset is combined with three different anti-seizure medications representing distinct mechanisms of action, supporting its potential as a combination therapy.
This approach offers a differentiated strategy for central nervous system RNA therapeutics by enabling systemic intravenous delivery to deep brain regions with durable therapeutic activity from a single administration.
- Branden Ryu, PhD - CEO, BIORCHESTRA Co., Ltd.
- Elisbetta Morini - Assistant Professor of Neurology, Massachusetts General Hospital and Harvard Medical School
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
- Andrew Varley - RNA & Formulation Core Director, The University of British Columbia
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 AF (Add Fluorescence) 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.
- Maj Petersen, PhD - Senior Director of Innovation and Project Execution, Novo Nordisk
- David Harwicke - Co-Founder and Sr Director, Head of Business Development, Aerska
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!