Thursday, September 24 - EST (Eastern Time, UTC-05:00
- 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
- 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
- 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
- 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.
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