ATS 2020 Advance Program

• highlight areas of uncertainty regarding the use of precise therapeutics, including eligibility, cost, and other ethical considerations. In this session, we will discuss disease-altering and potentially life-saving “precision medicine” therapeutics for pediatric respiratory diseases, including genotype-based treatments and gene therapies. We will focus on cystic fibrosis, neuromuscular diseases, sickle cell disease, and asthma as examples of what the field has accomplished and the road ahead. In addition, we will discuss important ethical and cost-effectiveness considerations that researchers, clinicians, and patients will have to face as we move forward. Chairing: E. Forno, MD, MPH, ATSF, Pittsburgh, PA G.S. Sawicki, MD, MPH, Boston, MA 9:15 Introduction E. Forno, MD, MPH, ATSF, Pittsburgh, PA 9:20 Precision Medicine in Action: The Promise of Genetic Based Therapies in Cystic Fibrosis G.S. Sawicki, MD, MPH, Boston, MA 9:38 Questions 9:43 Progress to Date and Ongoing Studies for Gene Therapy in Sickle Cell Disease M.R. DeBaun, MD, MPH, Nashville, TN 10:01 Questions 10:06 Precision Therapeutics for Spinal Muscular Atrophy and Other Neuromuscular Diseases M. Buu, MD, Palo Alto, CA 10:24 Questions 10:29 Precision Medicine in Childhood Asthma: The Long Road Ahead A.H. Maitland-van der Zee, PharmD, PhD, Amsterdam, Netherlands 10:47 Questions 10:52 Cost-Effectiveness and Ethics Considerations of Precision Medicine for Pediatric Lung Diseases A.C. Wu, MD, MPH, Boston, MA 11:10 Questions CLINICAL • TRANSLATIONAL SCIENTIFIC SYMPOSIUM A9 SEX, LUNGS, AND ENVIRONMENTAL EXPOSURES Assemblies on Environmental, Occupational and Population Health; Behavioral and Health Services Research; Pulmonary Infections and Tuberculosis 9:15 a.m. - 11:15 a.m. Target Audience Providers of lung health, basic and translational researchers who are interested in translational and impacts of environmental exposures, those with clinical or research responsibilities. Objectives At the conclusion of this session, the participant will be able to: • learn new findings about sex specific responses to environmental exposures; • apply and integrate sex as a biological variable appropriately in environmental exposure assessment research; • improve ability to identify environmental lung disease by better understanding sex-specific effects and susceptibilities. Lung disease susceptibility is often sex specific. However, only recently, have clinical and translational research studies incorporated a thorough analysis of sex differences in response to environmental exposures and the potential link of these differences to disease susceptibility. This session will explore the interaction of sex with environmental exposures to increase susceptibility to lung disease and infection. Studies in rodents, non-human primates, and humans will be described, providing an overview of this emerging field. This session will also provide recommendations on next steps for the study of respiratory sex differences in response to environmental exposures and best practices in study design and analysis to facilitate the incorporation of sex as a biological variable. ATS 2020 • Philadelphia, PA SUNDAY • MAY 17 41