7:00 am Registration & Coffee

7:50 am Chair’s Opening Remarks

Strategies to Refine CRISPR Tools

8:00 am Honing Novel Gene Editing Tools for Improved Activity, Specificity and Deliverability

  • Brett Staahl Co-Founder & VP Platform, Scribe Therapeutics

8:30 am SEND: A New Tool for Delivering Cas9 and Other Therapeutically Important mRNAs


  • SEND (Selective Endogenous Encapsidation for Cellular Delivery), offers a solution to the major technological delivery challenge with CRISPR
  • An introduction to SEND and perspectives on how SEND system research can be advanced using genetically engineered mouse models
  • resenting proof of principle experiments demonstrating SEND in vivo

9:00 am Tailormade CRISPR-Based Solutions for Therapeutic Applications

  • Rafi Emmanuel Senior Vice President - Research & Development, Emendo Biotherapeutics


  • Emendo’s nuclease discovery and protein engineering platforms facilitate the development of the optimal editing strategy for each indication
  • Nucleases with unique PAM sequences allow for targeting exclusive genomic sites
  • Discovery of nucleases of various characteristics enables flexibility in choosing delivery vehicles

9:30 am Enhancing Gene Editing Workflow: Leveraging novel techniques for effective outcomes


  • Manufacturing final formulations and individual genome editing components to high-quality standards with extensive characterization
  • Enabling robust data generation to satisfy new FDA requirements for ribonucleoprotein (RNP) manufacturing and associated novel characterization assays
  • Enabling the development and translation of gene-modified cell therapies to meet regulatory expectations and are commercially viable at scale

10:00 am Morning Refreshments & Speed Networking


Our speed networking is the ideal opportunity to get face-to-face time with colleagues and peers who are passionate about bringing the next generation of CRISPR gene editing tools to those most in need. Introduce yourself to the attendees that you would like to have more in-depth conversations with, explore common challenges, and establish meaningful relationships to pursue for the rest of the conference and beyond.

Rethinking R&D for Novel CRISPR Therapies

11:00 am In Vitro Disease Modelling as a Tool for Identifying Neurological Drug Targets


  • Exploring in vitro neurological disease modelling for identifying drug targets
  • A new approach: marrying CRISPR-based functional genomics with human hiPSC-derived disease models
  • Exploring success stories from modelling epilepsy and autism in hiPSC-derived neurons using CRISPR/Cas9 genome editing

11:30 am VIRTUAL: Innovating CRISPR Gene Editing Safety Assessments


  • Most gene editing applications carry the risk of unfavourable outcomes
  • The level of unwanted gene editing consequences is still highly unknown
  • The safety of gene therapies depends on the accuracy of editing detection

12:00 pm VIRTUAL: Accelerating Gene Editing Workflows Towards the Clinic


  • Approaching the FDA with confidence about On / Off target analytics and safety determinations
  • Aligning nuclease license and guide production from research to clinic from the beginning
  • Automation brings editing and screening depth sufficient for creating multi-edited MCB

12:10 pm Lunch & Networking

Exploring Next Generation Approaches to Preclinical Development

1:10 pm Optimizing Animal Models for Genetic Diseases and Gene Editing Therapeutics

  • Guillaume Pavlovic Head of Genetic Engineering and Model Validation Department, Institut Clinique De La Souris


  • Improving the sophistication of animal models using a combined approach of embryonic stem cells and CRISPR technology
  • Defining the unique requirements for animal models in the preclinical setting compared with basic academic research
  • Beyond proof-of-concept studies, exploring animal models as a tool to investigate unwanted safety issues of a therapy

1:40 pm One Genome or Thousands of Genomes? The importance of evaluating population variants


  • What does the FDA gene-editing draft guidance ask for?
  • Why are population variants important for off-target risk assessments?
  • Methods to efficiently generate and match risk assessments with the intent to treat population

1:50 pm Utilising Hybrid Guide Methods to Refine Genome Editing Precision

  • Paul Donohoue Senior Scientist – Platform Discovery Group Lead, Caribou Biosciences


  • Understanding how CRISPR hybrid DNA-RNA (chRDNA) guides function to increase gene editing specificity and reduce levels of off-target editing compared to traditional all-RNA systems
  • Demonstration of the activity of chRDNA guides across both the Cas9 and Cas12a CRISPR systems
  • Optimization of nuclear delivery to increase editing efficiency in T cell and iPSCs

2:20 pm Leveraging A 4 Billion Year Evolution Experiment: A Function-First Approach to Developing Nature-Inspired Genome Editors


  • Exploring expanded novel sequence diversity using BaseGraph, one of the most diverse proprietary metagenomic datasets
  • Searching protein space by function and performance, instead of sequence or structural homology
  • Generalising our approach for any protein type – nucleases, base editors, transposases, and entirely novel systems to develop a next-generation genome editing toolbox

2:30 pm Afternoon Refreshments & Poster Session

3:00 pm A Next-Generation Treatment for Familial Hypercholesterolaemia

  • Ira Gotliv Vice President – Preclinical Research, Emendo Biotherapeutics


  • Exploring a novel CRISPR-based gene editing approach to upregulate LDLR expression
  • Outlining the potential efficacy, safety and dosing benefits of avoiding PCSK9 knockouts
  • Explaining the benefits of proprietary novel nucleases for this treatment option

3:30 pm Developing a Toolkit of Tailored Novel Nucleases

  • John Murphy Chief Scientific Officer, Arbor Biotechnologies


  • Understanding the cutting patterns of new CRISPR nucleases
  • Using AI and ML in their development
  • Methods for finetuning these nucleases to mitigate against off-target effects

4:00 pm Restoring the Efficacy of Chemotherapy: Utilizing CRISPR as a Gene- Disabling Treatment for Squamous Cell Carcinoma of the Lung

  • Eric B. Kmiec Executive Director & Chief Scientific Officer, ChristianaCare Gene Editing Institute


  • Understanding the science behind disabling the NRF2 gene as a squamous cell carcinoma treatment adjunct
  • Evaluating the efficacy and safety data, and how these are being demonstrated to the regulators to transition to clinical development
  • Exploring the differences and potential benefits in developing a gene editing product in a community-based health system

5:00 pm Chair’s Closing Remarks