7:30 am Registration & Coffee

8:10 am Chair’s Opening Remarks

Realizing the Potential of Precision Gene Editing Therapies

8:15 am Looking Beyond 2022 – Future Directions for CRISPR

  • Kiran Musunuru Professor of Medicine Director, Perelman School of Medicine, University of Pennsylvania

Synopsis

  • Outlining the latest advances and developments in CRISPR-based therapies, with a focus on in vivo applications
  • Exploring future directions for current gene editing companies including base, prime and epigenome editing technologies
  • Understand what current obstacles and challenges are holding in vivo gene editing therapeutics back from success

8:45 am Generation of Engineered Pluripotent Stem Cell Lines For Clinical Applications

  • Erik Willems Senior Manager, Cell Biology R&D, Thermo Fisher Scientific

Synopsis

  • Detailing advances with tools, reagents and protocols that facilitate the genome editing workflow in hiPSC
  • Demonstrating how the use of these tools can be readily implemented in generating allogenic hiPSC for clinical application
  • Applying this workflow to enable cGMP manufacturing of engineered hiPSC

Exploring Developments in Base and Prime Editing

9:15 am Developing Prime Editing for Therapeutics

Synopsis

  • Exploring recent advances in Prime Editing technologies
  • Understanding the safety profile of these technologies preclinically
  • Showcasing Prime Medicine’s platforms for prime editing
  • Introduction to Prime Medicine’s therapeutic programs

9:45 am Morning Refreshments & Networking

10:15 am Reserved for Akron Bio

Synopsis

10:45 am Comparing Base Editors with Traditional Cas9 Nucleases for Inducing Fetal Haemoglobin Levels

  • Jonathan Yen Director of Therapeutic Genome Engineering, St. Jude Children’s Research Hospital

Synopsis

  • Exploring how base editing more precisely generates de novo novel binding sites
  • Understanding the variability of indels and lower productivity associated with Cas9 usage
  • Comparing the safety profiles of these Cas9 and base editing strategies

11:15 am Leveraging Base Editors for Precision Treatment of Inherited Retinal Disorders

  • Samuel Du MD-PhD Student, Krzysztof Palczewski Lab, University of California Irvine

Synopsis

  • A focused look at the genetic heterogeneity of IRDs
  • Evaluating the use of cytosine and adenine base editors for gene-independent point mutation corrections
  • Analysing the current safety profile of base editing technologies

11:45 am Lunch & Networking

Preparing for Success in Clinical Development for Gene Editing Therapies

1:00 pm Round Tables: Preparing for Success in Clinical Development

Weston Miller
Vice President - Clinical Development, Graphite Bio

Liron Walsh
Head of Development; In Vivo Programs, Intellia Therapeutics

Michael Maitland
Senior Director, Clinical Development Intellia Therapeutics

 

When gearing up for clinical development it is important to careful design protocols to support all parameters of development. These roundtables will focus specifically on common considerations when preparing for a successful clinical trial.

Safety Moniroting
for Gene Editing
Therapies in the Clinic

Clinical Dose Selection

Clinical Surrogate
Endpoints

Patient Selection:
Finding the Right
Population

2:00 pm Showcasing Precision & Efficacy in the Clinic to Regulatory Authorities

  • Darren Hart Senior Vice President, Development, Graphite Bio

2:30 pm The Importance of the Patient Voice in Clinical Development for Gene Editing Technologies

  • Amy Simon Chief Medical Officer, Beam Therapeutics

Synopsis

  • Understanding the unique clinical development challenges with CRISPR-based therapies
  • Identifying the importance of engaging patients and caregivers in the clinical development process, from the preclinical stage and beyond
  • Infusing patient centricity throughout a drug development organization

3:00 pm Afternoon Refreshments & Networking

Improving the Efficiency & Efficacy of Knock-In Gene Editing

3:30 pm Designing Robust Guide RNAs

Synopsis

  • Software tools which predict on-target and off-target effects to support careful design of guide RNAs
  • Tips for assessing sgRNA efficiency before cell transduction
  • Increasing sgRNA specificity for CRISPR knock-out and knock-in experiments
  • Considerations for optimal base editing

4:00 pm Choosing the Right Donor DNA Format

  • Howard Wu Founder & Chief Scientific Officer, Full Circles Therapeutics

Synopsis

  • The use of double-stranded DNA (dsDNA) donors is associated with poor knock-in efficiency and increased cytotoxicity and potential insertional mutagenesis, which is driven by genomic integration of dsDNA through the HDR repair processes
  • Understanding modified ssODN donors as an approach to enhance efficiency for knock-ins
  • Assessing preliminary data from circular single-stranded DNA (cssDNA) and its potential to mitigate dsDNA related safety concerns

4:30 pm Rapid Selection of Scarless HDR Events in Mammalian Cells

Synopsis

  • Understanding HDR as the preferred method for seamlessly installing new genetic information at nuclease induced DSBs
  • Exploring the challenges associated with different activity levels of HDR and NHEJ for treating diseases requiring gene correction or insertion
  • Evaluating a new proprietary methodology for installing user-defined mutations without leaving residual ‘DNA scars’ often associated with conventional SSR techniques

5:00 pm Chair’s Closing Remarks & End of Summit