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.

• 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

• Addressing SP-Cas9 PAM sequence limitations that restrict targetability to only five percent of the human genome for conventional gene editing applications
• Leveraging diverse nuclease platforms recognizing alternative PAM sequences to expand genome accessibility and enable precise editing across previously inaccessible genomic regions
• Implementing next-generation CRISPR technologies including prime editors and base editors requiring PAM flexibility to achieve nucleotide-specific edits at desired genomic locations

• 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

• Educating regulatory agencies on emerging modalities including prime editing, base editing, and epigenetic editing to establish shared understanding of safety and efficacy profiles
• Implementing proactive regulatory engagement strategies throughout preclinical and clinical development to address agency concerns and align on evidence requirements for novel tools
• Establishing validation frameworks for next-generation editing technologies to streamline regulatory review and build confidence in therapeutic potential

• 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

• Expanding CRISPR editing and delivery capabilities, with broader genomic reach, to unlock treatments for previously undruggable diseases
• Accelerating target discovery and CRISPR therapy optimization using in vitro technologies, robotics, automation, and AI to compress development timelines
• Navigating emerging regulatory pathways, designed specifically for gene therapies, to bring CRISPR treatments to human trials faster