Conference Day Two - Thursday, December 5

8.05 Check-In & Coffee

8.55 Chair’s Opening Remarks

Plenary Session: Academic Pioneers

9.05 Therapeutic Gene Editing for Cardiovascular and Metabolic Diseases: From the Leading Cause of Death to N-Of-1 Disorders

9.35 Advancing the Development of Safe and Effective Cell & Gene Therapies Using INDUCE-seq®

10.05 Engineering and Evolution of Hyperactive, CRISPR-Associated Transposases (VIRTUAL)

  • Sam Sternberg Assistant Professor, Department of Biochemistry & Molecular Biophysics, Columbia University

10.35 Morning Refreshments

Innovation & Early Development Track

Translating to the Clinic Track

Progressing Epigenome Editors into the Clinic

Moderator: Luca Biasco, Nvelop Therapeutics

Outlining a Path to the Clinic

Moderator: Narin Ahmed, Tessera Therapeutics

11.20 Programmable Gene Activation, Silencing &
Multiplexing for Precise Gene Regulation

Shane Wright, Associate Scientist II, Chroma Medicine

11.20 Regulatory Strategies to Enable the Clinical Trial
Applications of Genome Editing Products

Claire Levée, Director, Regulatory Affairs, Genetics Medicines,
Regeneron Pharmaceuticals

11.50 Epigenome editing with CRIPSR-GNDM® as a treatment platform for unmet medical conditions

Tetsuya Yamagata, Chief Scientific Officer, Modalis Therapeutics

11.50 Editing HBG1/2 and BCL11A for Beta
Hemoglobinopathies

Sheryl Bowley, Director, HSC Therapies, Editas Medicinee

12.20 CRISPR Epigenetic Modification to Treat Hepatitis B

Britta Jones, Senior Scientist, Tune Therapeutics

12.20 Compact Nucleases: Outlining a Roadmap Towards
Clinical Evaluation

Mark DeWitt, Associate Director, Mammoth Biosciences

12.50 Lunch

Innovation & Early Development Track

Translating to the Clinic Track

Validating the Next Generation of Delivery Vehicles

Moderator: Luca Biasco, Nvelop Therapeutics

Driving Novel Nucleases Towards the Clinic

Moderator: Narin Ahmed, Tessera Therapeutics

1.50 In Vivo RNA Delivery to T Cells & Hematopoietic Stem
Cells in Humanized Mice & Non-Human Primates Using
Targeting Lipid Nanoparticles

Apiwat Wangweerawong, Director, Delivery Chemistry,
Tessera Therapeutics

1.50 AFNT-212: A Non-Viral Gene-Edited TCR-T Cell Therapy

Ankit Gupta, Senior Director, Head of Gene Editing, Affini-T Therapeutics

2.20 In Vivo Application of Exosome-Based Genome Editing
Technology

Dave Carter, Head of Research, Evox Therapeutics

2.20 Disruption of Aberrant Splicing by Gene Editing with
a Type V CRISPR-Cas Enzyme as a Potential Treatment for
ALS

Catherine Symonds, Senior Director, Head of Strategy & New
Product Planning,
Arbor Biotechnologies

2.50 Afternoon Refreshments

Plenary Session: Boundary-Pushing In Vivo Delivery Platforms

3.20 Progressing Extrahepatic Lipid Nanoparticle (LNP) Delivery

3.50 Panel Discussion: Selecting the Best Delivery Vehicles for Extrahepatic Targeting Cargos

  • Ryan Leenay Associate Vice President, Genome Editing, Eli Lilly & Co.
  • Marco Weinberg SVP, ReCode Therapeutics, Inc
  • Luca Biasco Vice President, Translational Sciences, Nvelop Therapeutics

4.30 Chair’s Closing Remarks

  • Vivian Choi Vice President, Head of Liver, Lung, Metabolic, Eye & Ear, Prime Medicine

4.40 End of Conference