mRNA & Genome Editing: Innovations in Therapeutics and Precision Medicine
mRNA platforms and genome editing technologies are redefining how genetic and acquired diseases are treated, with advances in sequence design, delivery technologies, and editing platforms accelerating therapeutic impact. This track brings together practical insights on mRNA engineering, delivery breakthroughs, and gene editing translation. Are you positioned to lead in the age of precision RNA medicine?
What are the key topics for this track?
In Vivo Gene Editing for Therapeutic Impact
Next-generation in vivo gene editing is unlocking durable therapies for genetic and cardiovascular diseases. CRISPR-based platforms and RNA-only systems now enable precise modulation of target genes in living organisms, including LDL-C lowering and CAR-T applications.
Why this matters now:
Patients with unmet needs in hyperlipidemia, sickle cell disease, and other genetic conditions require therapies that go beyond conventional biologics or small molecules, with durable effects and minimal off-target risk.
Key Session to Attend:
Transformative LDL Cholesterol Lowering In Vivo CRISPR Gene Editing Approach - Explore how in vivo CRISPR modulation of LDLR achieves up to 90% LDL-C reduction in preclinical models.
AI and Rational Design for RNA Therapeutics
AI and rational design are accelerating mRNA and RNA-targeted therapeutic development. Machine learning, generative models, and structural insights are enabling precise sequence optimization, RNA modification, and functional prediction for both vaccines and therapeutics.
Why this matters now:
The complexity of RNA biology and expanding therapeutic applications demand computational tools that can predict RNA structure, translation efficiency, and stability, reducing costly trial-and-error cycles.
Key Session to Attend:
Applying Machine Learning to Identify Differentiated Target Mechanisms - Learn how AI platforms identify RNA regulatory mechanisms and accelerate oligonucleotide and mRNA therapeutic design.
Delivery and Targeting Innovations
Efficient and targeted delivery is critical to unlock the full potential of genome editing and mRNA therapeutics. LNP optimization, cell-specific ligands, and novel gene writer platforms enable editing of stem cells and extrahepatic tissues safely and effectively.
Why this matters now:
Therapeutic efficacy relies not just on the molecule, but also on its ability to reach the right cells, in the right dose, without triggering off-target effects or immune responses.
Key Session to Attend:
Delivery of Gene Writers to Hematopoietic Stem Cells - See how RNA-based gene writers paired with optimized LNPs achieve multi-lineage editing in humanized mice and NHPs.
Analytical and Manufacturing Excellence
Advanced analytical and manufacturing innovations are ensuring mRNA therapeutics and gene editing platforms are scalable, high-quality, and regulatory-ready. New sequencing, chromatography, and cell-based production methods are establishing industry standards.
Why this matters now:
As mRNA and genome editing therapeutics move from preclinical to clinical and commercial scale, robust characterization and manufacturing control are essential to ensure safety, efficacy, and reproducibility.
Key Session to Attend:
Enabling Next-Generation mRNA Therapeutics Through a Cell-Based mRNA Design and Manufacturing Platform - Discover a fully integrated platform for design, optimization, and scalable production of mRNA medicines.
Who's Speaking on the mRNA & Genome Editing: Technology & Applications track?
FAQs
What does mRNA and genome editing research involve?
It includes RNA design, chemical modification, delivery optimization, and genome editing to achieve precise and durable therapeutic effects. Platforms integrate AI, high-throughput screening, and in vivo validation to ensure candidates are ready for preclinical or clinical studies.
How is this track bridging discovery and clinical translation?
Speakers will cover innovations in gene editing, RNA design, and delivery, alongside process development, analytical characterization, and manufacturing strategies, highlighting real-world translation into clinical and commercial candidates.
What is in vivo genome editing and why is it important?
In vivo genome editing involves modifying genes directly within a patient’s cells, rather than ex vivo. This enables durable, potentially one-time treatments for genetic diseases, cardiovascular conditions, and other disorders that are difficult to address with conventional therapies.
How does mRNA design impact therapeutic outcomes?
mRNA therapeutics rely on precise control of translation, stability, and immunogenicity. Secondary structures, RNA-binding proteins, and chemical modifications all influence efficacy and safety. Optimized mRNA design is critical for vaccines, protein replacement therapies, and gene editing payloads.
What are the main challenges in delivering genome editing and RNA therapeutics?
Delivering RNA or gene editors to the right cells safely is one of the biggest hurdles. Challenges include immune recognition, off-target editing, and achieving high efficiency in extrahepatic tissues.
This track specifically highlights:
LNP optimization for targeted delivery and extrahepatic tissue penetration.
Cell-specific ligands and formulation strategies to enhance uptake and efficacy.
Preclinical demonstrations of multi-lineage editing in humanized mice and non-human primates.