Conference Day Two - Thursday, December 5

8.00 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

10.05 Advancing the Development of Safe and Effective Cell & Gene Therapies Using INDUCE-seqTM

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

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

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

1.30 Discovery and HG-PRECISE Engineering of
hfCas12Max High-Fidelity Nuclease and Pre-clinical Testing
in Mouse and NHP Models to Support M.U.S.C.L.E. Trial for
DMD

TJ Cradick, Chief Technology Officer, HuidaGene
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

John Murphy, Chief Scientific Officer, Arbor Biotechnologies

2.50 Afternoon Refreshments

Plenary Session: Boundary-Pushing In Vivo Delivery Platforms

3.20 Progressing Extrahepatic Lipid Nanoparticle (LNP) Delivery

  • Marco Weinberg Senior Vice President, Head of Research, ReCode Therapeutics, Inc

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 Senior Vice President, Head of Research, ReCode Therapeutics, Inc
  • Luca Biasco Vice President, Nvelop Therapeutics

4.30 Chair’s Closing Remarks

4.40 End of Conference