Unlocking the Future of Medicine

November 5, 2025 by Derek Jacobs

Insights from the Genome Editing Therapeutics Summit

In many ways, smaller is better. It allows for focus while providing an engine for growth. During the Genome Editing Therapeutics Summit in Boston, that focus was provided by a group of innovators, researchers, and industry leaders to explore the cutting edge of genetic medicine. While there were fewer than 100 attendees, the event provided in-depth and high-impact networking and deep technical exchanges. Most importantly, several trends that are shaping the future of genome editing help show where future opportunities for growth lie.

AI Takes the Driver’s Seat
Artificial intelligence is no longer just a buzzword—it’s becoming a practical tool in genome editing. Predictive design for CRISPR guides and chassis engineering are enabling faster iteration and more precise targeting. This shift is accelerating development timelines and reducing trial-and-error in early-stage research.

Delivery Remains the Bottleneck
Despite significant advances in genome editing tools, delivery remains a persistent challenge. Innovations for in vivo delivery methods, novel lipid nanoparticles (LNPs), and emerging platforms like TEMPO, CasPhi, and mini-TET are critical to ensuring that therapeutic payloads reach their intended targets safely and effectively.

A growing focus on miniaturization—developing physically smaller tools—has improved the ability to package and deliver therapies, especially within the size constraints of viral vectors and LNPs. These compact systems not only enhance delivery efficiency but also unlock new possibilities for targeting previously inaccessible tissues.

The New Gatekeeper
Clinical advancement is increasingly defined by manufacturability. Chemistry, Manufacturing, and Controls (CMC) readiness and scale-up challenges are now the primary barriers to moving therapies from bench to bedside. Companies must design with scalability in mind from day one.

Regulatory Expectations Are Rising
Regulators are setting a higher bar for safety and efficacy. Genome-wide off-target assays, validated clinical biomarkers, and robust safety endpoints are now baseline requirements for Investigational New Drug (IND) applications. The message is clear: scientific rigor and regulatory alignment must go hand-in-hand.

Collaboration Is Key
No one advances alone. Partnerships between pharma, biotech, and academia are now essential as shared resources and expertise are driving innovation faster than siloed efforts ever could. As genome editing moves from concept to clinic, the insights from this summit underscore a pivotal moment: the convergence of AI, delivery science, manufacturing, and collaboration is unlocking the next generation of genetic therapies.

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