Day One

• Unpacking strategies to minimize off-target toxicity in CRISPR Cas9 therapies to equip you with approaches to build a stronger safety profile for regulators
• Leveraging sequencing-based methods to assess gRNA purity, raw material quality, and genomic instability to de-risk your CRISPR therapy
• Benchmarking industry consensus on sequence purity standards for gene editing materials to help you align your analytical strategy with regulatory expectations

• Exploring how CRISPR arrayed screens can be designed and deployed to identify and validate novel targets, giving you a scalable framework for accelerating early discovery
• Examining how validated CRISPR screen hits are assessed for pipeline fit across autoimmune disease indications, helping you strengthen your own target selection criteria
• Discussing strategies to minimize off-target effects during target identification screening, equipping you with optimized guide RNA and high-fidelity Cas9 approaches to improve hit confidence

As the genome editing community is reunited, this valuable session will ensure you get the chance to reconnect with peers and make brand new connections. This structured networking opportunity will pair you with fellow attendees for several 3-minute introductions, ensuring you have the opportunity to meet and network with your industry colleagues.

• Addressing SP-Cas9 PAM sequence limitations that restrict targetability to only five percent of the human genome for conventional gene editing applications
• Leveraging AI-based technology to optimize diverse nuclease platforms recognizing alternative PAM sequences, reducing off-target activity and improving delivery compatibility for therapeutic applications
• Implementing AI-driven nuclease engineering to expand platform robustness and efficiency, enabling faster development of next-generation CRISPR tools including prime editors and base editors for therapeutic purposes

• We have engineered the compact CRISPR effector CasX (XE) to achieve >100-fold higher activity than wild-type CasX, establishing a potent platform for therapeutic in vivo application
• The XE platform demonstrates clinical readiness with saturating editing in non-human primate liver models and no detectable off-target activity above background at supersaturating doses in primary human hepatocytes
• We introduce “DeepXE,” an AI model trained on a quantitative dataset of >40,000 gRNAs; this predictive engine accurately identifies high-efficiency therapeutic guides, dramatically reducing the cycle time for candidate selection
• We advanced targetability of XE by building models such as DeepXE into our comprehensive engineering pipelines

• Streamlining manufacturing and analytical collaboration to preserve product purity, potency, and consistency throughout scale-up
• Optimizing CDMO partnerships and tech transfer strategies to reduce development delays and manufacturing inefficiencies
• Exploring flexible guide RNA manufacturing approaches to support scalable production across diverse rare disease populations

Securing regulatory approval for novel genome editing modalities presents unique challenges, with unclear expectations around preclinical pharmacology, toxicity and analytics from agencies. Join this workshop to explore how late-stage and commercial companies are structuring IND packages, bridging preclinical and clinical data, and collaborating directly with regulators. Gain robust strategies to accelerate your program into the clinic.

Workshop highlights:

• Examining how late-stage companies structure IND packages for novel genome editing modalities, helping you build stronger regulatory submissions faster
• Identifying which preclinical outcomes most reliably predict clinical success, enabling you to design more robust studies that satisfy regulatory scrutiny
• Exploring commercial-stage regulatory strategies across new genome editing tools, giving you a roadmap to navigate approval pathways with greater confidence
• Engaging regulators directly in panel discussion to surface industry best practices, empowering you to foster more productive agency relationships for your program

• Leveraging evolved phage integrases for non-viral, site-specific genomic integration to enhance safety and minimize off-target effects
• Engineering a super-efficient non-viral delivery system for large DNA fragments to overcome current limitations and unlock complex gene therapies
• Delivering exceptionally large therapeutic gene payloads (up to 16kb) to address complex multi-gene disorders and reduce manufacturing costs

• Developing enGager system by fusing nuclear-localized Cas9 with ssDNA-binding motifs to achieve up to 6-fold higher integration efficiency
• Demonstrating CAR transgene integration in 33% of primary human T cells using non-viral cssDNA donors to enhance anti-tumor functionality safely
• Validating compact 20-amino acid RecA L2 ssDNA-binding motifs as alternatives to recombination proteins to improve HDR-mediated genome integration