Lonrú at Keystone: Precision Genome Engineering and the Future of CRISPR Clinical Translation

The upcoming Keystone Symposium on Precision Genome Engineering: Translating the Human Genome to the Clinic (March 3-6, 2025, in Killarney, Ireland) presents an exciting opportunity for the field of gene editing to converge on the critical scientific and regulatory advancements necessary for therapeutic translation. As a firm dedicated to empowering technology providers in the Cell and Gene Therapy (CGT) sector, Lonrú Consulting is eager to engage in discussions that address key industry challenges and pathways for accelerating CRISPR-based therapeutic approvals.

What We’re Looking Forward To

The Keystone agenda is packed with high-impact sessions, and several key themes stand out as particularly relevant for tool providers and regulatory strategists:

Regulatory Innovation and Harmonization to Expand Access to Precision Gene Therapies

The evolution of global regulatory frameworks remains one of the largest hurdles in transitioning CRISPR therapies from preclinical research to clinical implementation. We are particularly interested in sessions that discuss strategies to streamline Investigational New Drug (IND) and Clinical Trial Application (CTA) submissions, as we consider how tools providers may help therapeutic developers in this space.

From Target to IND: Preclinical Studies for ex vivo and in vivo Therapies

With the rise of in vivo genome-editing therapies alongside more established ex vivo approaches, ensuring safety and efficacy through robust preclinical models is paramount. Lonrú is keen to explore discussions on off-target risk mitigation, potency assays, and long-term safety assessments.

Genome Engineering in the Clinic: Advances in Therapeutic Applications

As CRISPR-based therapies move into human trials, the interplay between genome engineering technologies and clinical strategy becomes increasingly complex. We anticipate valuable insights into how industry leaders are addressing patient risk management, staggered dosing strategies, and immune response monitoring.

Lonrú’s Contribution: Bridging the Gap Between Preclinical and Clinical Translation

Lonrú’s poster evaluates global regulatory trends and identifies common pitfalls in IND/CTA submissions that lead to clinical holds and delays, we also introduce our decision tree framework, a tool to help developers navigate common pitfalls.

At Keystone, Lonrú will present our poster, “Bridging the Gap: Streamlining Preclinical to Clinical Trial Applications (IND/CTA) for CRISPR Therapies.” This work evaluates global regulatory trends and identifies common pitfalls in IND/CTA submissions that lead to clinical holds and delays.

Our analysis emphasizes four strategic imperatives:

• Early engagement with regulators to preemptively address potential concerns.

• Enhanced off-target validation to minimize safety risks.

• Optimized Chemistry, Manufacturing, and Controls (CMC) readiness to ensure scalable production.

• Parallel global regulatory filings to accelerate approvals across different jurisdictions.

To support these strategies, we are unveiling our Decision-Tree Framework, a structured tool designed to help developers de-risk their preclinical-to-clinical transitions. This framework is segmented into three core areas:

• Preclinical & Safety Considerations (off-target assessment, long-term safety studies, in vivo vs. ex vivo risk management)

• CMC & Manufacturing Readiness (potency assay validation, lot-to-lot consistency, delivery system characterization)

• Regulatory & Trial Design Strategy (harmonization of submission dossiers, risk mitigation planning, patient safety protocols)

Post-Conference Insights & Resources

Following the Keystone meeting, Lonrú will provide a comprehensive conference report, including key takeaways from the most impactful sessions and commentary on emerging industry trends.

Additionally, our Decision-Tree Framework and poster will be made available for download, offering a valuable resource for CRISPR developers seeking to refine their IND/CTA strategy.

Bridging the Gap: Streamlining Preclinical to Clinical Trial Applications (IND/CTA) for CRISPR Therapies

We are thrilled to present our poster at Keystone, showcasing key insights into regulatory readiness, risk mitigation, and translational strategies. Proudly featuring insights from Lonrú Consulting and Johns Hopkins Carey Business School, our work highlights data-driven approaches to accelerating CRISPR-based therapeutic development.

For those attending Keystone, we welcome discussions on how Lonrú can support technology providers in navigating regulatory complexities and accelerating the path to clinic.

If you are interested in receiving any of the resources outlined here, please complete the form below and we’ll be in touch after the conference.