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

  • Benjamin Oakes Co-Founder & Chief Executive Officer, Scribe Therapeutics

8:30 am 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

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 Reserved for Aldevron


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

11:30 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

12:00 pm Redefining the Future of Oncology: Utilising CRISPR Screens as a Tool for Drug Discovery


  • Upscaling: Leveraging CRISPR loss-of-function screens for an unbiased approach for querying the whole genome and identifying novel drug targets
  • Understanding the use of CRISPR screens for identifying BRCA-PARP synthetic lethality
  • Exploring the role of whole genome screens for the future of cancer treatments

12:30 pm Reserved for Synthego


12:40 pm Lunch & Networking

Exploring Next Generation Approaches to Preclinical Development

1:30 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

2:00 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

2:30 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

3:00 pm Afternoon Refreshments & Poster Session

4:00 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

4:30 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