Beyond the Liver: Unlocking the Full Potential of Genetic Medicine
Editing Has Advanced Faster Than Delivery
Genome editing technologies have evolved at a remarkable pace.
Over the last decade, the field has progressed from first-generation CRISPR-Cas9 systems to sophisticated platforms including base editing, prime editing, epigenome editing, multiplex editing, and even gene writing. The industry's focus is increasingly shifting toward translating these technologies into meaningful therapies for patients.
Yet despite this rapid innovation, one challenge continues to slow progress across the field: delivery.
Scientists can now make increasingly precise and complex genetic modifications. The harder question is how to safely and efficiently get those editing tools to the right cells, in the right tissues, at the right dose.
For many developers, delivery has become the single greatest barrier standing between promising science and clinical reality.
Why Now?
The urgency around delivery has never been greater.
The FDA's proposed Plausible Mechanism Pathway is creating new opportunities for genome editing therapies to reach patients faster, while growing clinical maturity across the sector is increasing pressure to demonstrate real-world therapeutic impact. At the same time, major investments from organizations such as Lilly and Regeneron continue to reinforce confidence in the future of genome engineering.
However, as developers seek to move beyond rare liver diseases and target larger patient populations, a key limitation has become clear.
Most successful in vivo editing programs today are still largely confined to the liver.
To unlock the next generation of therapies, the industry must effectively target tissues such as muscle, lung, brain and central nervous system, heart, kidney, immune cells, hematopoietic stem cells.
This challenge is commonly referred to as the extrahepatic delivery problem, and many believe it will determine which companies lead the next phase of therapeutic genome engineering.
Why Liver Success Isn't Enough
The liver has emerged as the natural starting point for many in vivo editing programs because existing delivery technologies, especially lipid nanoparticles (LNPs), tend to accumulate there efficiently.
This has enabled important clinical advances and validated the broader potential of in vivo editing. However, countless diseases cannot be addressed through liver-targeted delivery alone.
Developers now face a new challenge: retaining the efficiency and safety that made liver-directed editing successful while extending delivery to entirely different biological environments.
This requires innovation not only in editing tools but also in:
LNP Design
AAV Engineering
Tissue-Targeting Ligands
RNA Delivery Systems
Novel Non-Viral Vectors
Cell-Specific Payload Delivery
The companies that solve these challenges could unlock a dramatically larger therapeutic market.
Workshop B: Tackling the Delivery Bottleneck Head-On
This challenge is so important that it forms the basis of Workshop B at the 7th Genome Editing Therapeutics Summit.
Designed specifically for developers seeking to improve the safety, specificity, and scalability of in vivo genome editing, the workshop will focus on practical strategies for overcoming delivery hurdles and expanding editing beyond traditional liver-focused applications.
Attendees will explore:
- Real-world approaches for reducing unwanted liver tropism
- Strategies for improving the safety profile of LNP and AAV delivery systems
- Advances in next-generation delivery technologies
- Opportunities to target extrahepatic tissues more effectively
- Methods for accelerating the clinical translation of in vivo editing platforms
Rather than focusing purely on editing chemistry, Workshop B addresses one of the field's most pressing translational challenges: ensuring breakthrough genome engineering technologies can reach the patients who need them most.
It reflects the wider theme of the summit itself, which is focused not simply on novel technologies, but on the practical application of those technologies in therapeutic development.
Workshop B: Advancing Extrahepatic Delivery to Improve Safety & Targeting for Genome Editing Therapeutics
Exploring strategies to minimize LNP and AAV liver tropism, reduce toxicity risks, and enable targeted extrahepatic delivery for in vivo genome editing. This workshop is particularly critical for teams aiming to expand beyond liver-focused indications and broaden the reach of gene editing therapeutics across diverse disease areas.
Looking Ahead
The first chapter of genome editing was defined by proving that DNA could be edited safely and effectively.
The next chapter may be defined by proving those edits can be delivered anywhere in the body.
As editing technologies continue to mature, the industry's competitive advantage is moving beyond the edit itself and toward the delivery systems that make therapeutic success possible. Whether through advanced LNPs, engineered viral vectors, or entirely new platforms, solving the extrahepatic challenge could unlock the next wave of transformative medicines.
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