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Wednesday, June 27 • 10:30am - 12:00pm
#346: Precision Medicine, Gene Editing, and Gene Therapy: Current Status and Regulatory Challenges of Integrating Genetic Medicine into Clinical Care

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Component Type: Forum
Level: Intermediate
CE: ACPE 1.50 Knowledge UAN: 0286-0000-18-654-L04-P; CME 1.50; IACET 1.50; RN 1.50

This DIAmond forum on precision medicine/gene therapy/gene editing will discuss the scientific, regulatory, and ethical/access issues surrounding these emerging technologies as they progress through the development process and become more integrated into clinical practice. Precision medicine is an emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment, and lifestyle for each person. Gene editing and gene therapy are cutting edge molecular technologies that give scientists the ability to change an organism's DNA by allowing genetic material to be added, removed, or altered at particular locations in the genome, influencing desired gene expression. Collectively, these rapidly emerging disruptive technologies are poised to dramatically change the practice of medicine and transform patient care and disease monitoring and prevention in the future. There will be a diverse group of speakers in this session representing the various stakeholders in these areas represented including scientists, government regulators, pharmaceutical company and HMO executives, as well as patient advocates and experts on value and access.

A particular focus of this forum will be a discussion of the anticipated changes in the global regulatory landscape for gene-based therapeutics and diagnostics as the technology in the space continues to evolve at a rapid pace.

Learning Objectives

Describe the development of next generation sequencing technology and it’s enabling of precision medicine; Discuss the development of CRISPR-based gene editing technology and its therapeutic application for genetic diseases such as Sickle Cell Disease; Identify prominent issues regarding regulation and pricing/reimbursement of gene-based therapeutics for disease and diagnostics for precision medicine.


Tshaka Cunningham, PhD


From Next Generation Sequencing to Gene Editing and Beyond: The Future of Gene-Based Medicine
George Church, PhD

Patient Perspective on Gene Editing and Gene Therapy: Can We Move Faster for a Cure for Sickle Cell Disease
Michael A. Friend

Peter W. Marks

Christopher O'Donnell, MD

Samarth Kulkarni, PhD

avatar for George Church

George Church

Professor / PI, Harvard University
George Church is Professor at Harvard & MIT, co-author of 450 papers, 95 patent publications & the book Regenesis. He developed methods used for the first genome sequence (1994) & genome recoding & million-fold cost reductions since. He co-initiated the BRAIN Initiative (2011) & Genome... Read More →
avatar for Tshaka Cunningham

Tshaka Cunningham

Associate Director, Scientific Collaboration, DIA
avatar for Michael Friend

Michael Friend

Founder, Minority Coalition For Precision Medicine and Health Ministries Network
Michael A. Friend was born and raised in Baltimore, Maryland, he's a Veteran of the Armed Forces and the Founder of the Minority Coalition for Precision Medicine and Health Ministries Network, which is a clearinghouse of various health care initiatives to help mobilize faith based... Read More →

Samarth Kulkarni

Chief Executive Officer, CRISPR Therapeutics
Dr. Samarth Kulkarni has significant expertise in strategy and operations in biotech and a wide range of related cutting-edge therapeutic technologies. He joined CRISPR in the early stages of the company and was responsible for setting the company's corporate strategy, supporting... Read More →
avatar for Peter Marks

Peter Marks

Director, Center for Biologics Evaluation and Research, FDA, United States
Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching... Read More →
avatar for Christopher O'Donnell

Christopher O'Donnell

Chief Scientist, Million Veteran Program, US Department of Veteran Affairs
Christopher J. O’Donnell, MD, MPH, is a cardiologists/epidemiologist, with 2 decades of executive scientific leadership experience directing large clinical/population research programs and international genomics consortia and steering large genomics programs at NHLBI/NIH and the... Read More →

Wednesday June 27, 2018 10:30am - 12:00pm EDT
Room 210AB Boston Convention and Exhibition Center 415 Summer Street, Boston, MA 02210 USA
  13: DIAmond, Forum