Workshop Day
Tuesday, December 3
Technical Stream
Strategy Stream
Workshop A <style="teht;">dsfdfsfsfasfs9.00 - 12.00
Workshop B sdfjslkdflsakjdflskfjsalkkjds 9.00 - 12.00
Progressing Lipid Nanoparticle-Based Delivery of Genome
Editing Therapies
• Highlighting the main formulations of lipid nanoparticles
(LNPs) used for CRISPR delivery in mRNA, plasmid, and RNP forms
• What progress is being made targeting other organs (e.g., the
lungs, immune system, bone marrow, stem cells)?
• Understanding key challenges associated with liver delivery
(e.g., endosomal escape, biodistribution, drug exposure)
• Evaluating techniques to target extrahepatic tissues and detarget the liver
• Overcoming toxicity risks associated with LNPs
• How to bridge the gap between efficacy readouts in mouse
and primate models
Carlo Zambonelli, Independent LNP Expert
Jeffrey Marshall, Analytical Director, mRNA & gRNA Platforms,
Beam Therapeutics
Navigating a Crowded Market: Target Selection & Portfolio
Strategy
• Understanding the challenge posed by multiple companies
pursuing the same disease targets
• Working cross-collaboratively between departments for robust
target identification
• Practical advice for risk mitigation when selecting a new disease to target
• When looking outside of the liver, is there an opportunity to go beyond T cells and HSCs?
• Developing a framework for matching novel technologies to
novel targets
• More broadly, how can the gene editing field progress in its
target selection in line with innovation in the technologies themselves?
John Murphy, Chief Scientific Officer, Arbor Biotechnologies
Ryan Leenay, Associate Vice President, Genome Editing, Eli
Lilly
Workshop C <style="teht;">dsfdfsfsfasfs1.00 - 4.00
Workshop D sdfjslkdflsakjdflskfjsalkkjds 1.00 - 4.00
Developing Robust Nonclinical Safety Packages
• Understanding biased and unbiased methods for the
detection of indels, translocations, and other chromosomal
aberrations
• Addressing technical challenges associated with these methods
• Navigating safety and toxicity risks, including when and how
to address reproductive risks
• Key considerations for safety profiling with different delivery
approaches
• Understanding the impact of platform-based technologies on
nonclinical packages
• Tying this all into a robust preclinical safety package for
regulatory submission
Ryo Takeuchi, Senior Director, Genome Editing, Excision
BioTherapeutics
Laura Serwer, Senior Director, Head of Pharmacology &
Toxicology, CRISPR Therapeutics
Ankit Gupta, Senior Director, Head of Gene Editing,
Affini-T Therapeutics
New FDA Guidance on Platform Technologies: Analysis,
Learnings & Implications for Gene Editing Drug Developers
• Unpacking key learnings for the gene editing field from the
recent draft guidance
• What constitutes a platform technology and how can it
qualify?
• How to put together a data package that meets core FDA
criteria for the designation
• Practical advice for FDA interactions regarding meetings,
submissions, and timelines
• Leveraging a platform designation in subsequent regulatory
submissions: how do we ensure this won’t duplicate work for
subsequent BLA submissions?
Jim Wang, Vice President Regulatory Affairs, Genetic Medicine, Regeneron Pharmaceuticals
Narin Ahmed, Vice President, Regulatory Science, Tessera
Therapeutics