Precision Medicine, Gene Editing and Gene Therapy: Current Status and Regulatory Challenges of Integrating Genetic Medicine into Clinical Care
Tshaka Cunningham, PhD
Associate Director, Scientific Collaboration
DIA, United States
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 Objective : Review the development of next generation sequencing technology and it’s enabling of precision medicine; Review the development of CRISPR-based gene editing technology and its therapeutic application for genetic diseases such as Sickle Cell Disease; Discuss prominent issues regarding regulation and pricing/reimbursement of gene-based therapeutics for disease and diagnostics for precision medicine.
From Next Generation Sequencing to Gene Editing and Beyond: The Future of Gene-Based Medicine
Robert Winthrop Professor of Genetics
Harvard University, United States
Patient Perspective on Gene Editing and Gene Therapy: Can We Move Faster for a Cure for Sickle Cell Disease
Michael A. Friend
Minority Coalition For Precision Medicine and Health Ministries Network, United States