Project Grant R13HL165804

ELEVENTH COOLEY'S ANEMIA SYMPOSIUM - ELEVENTH COOLEY'S ANEMIA SYMPOSIUM PROJECT SUMMARY THALASSEMIA SYNDROMES AFFECT AT LEAST TWO MILLION PEOPLE WORLDWIDE, AND RESEARCHERS BELIEVE THAT SEVERAL HUNDRED MILLION PEOPLE MAY BE "SILENT CARRIERS" OF A THALASSEMIA TRAIT, MAKING IT ONE OF THE MOST COMMON SINGLE- GENE DISORDERS IN HUMANS. THE ELEVENTH COOLEY'S ANEMIA SYMPOSIUM, WHICH WILL BE HELD THE CURE IN NEW YORK CITY, NY, USA ON OCTOBER 17-20, 2022, REPRESENTS THE LARGEST GLOBAL GATHERING OF THALASSEMIA EXPERTS, AND PROVIDES ONE OF THE FEW INSTANCES EACH DECADE THAT THE INTERNATIONAL RESEARCH COMMUNITY, THE PHARMACEUTICAL INDUSTRY, AND FUNDING AGENCIES DEDICATED TO FIGHTING THIS BLOOD DISORDER COME TOGETHER. THIS 3.5- DAY CONFERENCE WILL CONVENE APPROXIMATELY 250 ATTENDEES, INCLUDING SCIENTIFIC RESEARCHERS WORKING IN THE FIELDS OF MOLECULAR AND CELLULAR BIOLOGY, GENE THERAPY, AND STEM CELL DEVELOPMENT, AS WELL AS CLINICAL SPECIALISTS FROM THE FIELDS OF HEMATOLOGY, STEM CELL TRANSPLANTATION, MATERNAL-FETAL HEALTH, TRANSFUSION MEDICINE, AND PEDIATRICS, AMONG OTHERS. THE MAIN OBJECTIVES OF THIS CONFERENCE ARE TO: (I) PROVIDE A NEUTRAL FORUM FOR DISCUSSION OF EXISTING CLINICAL DATA, EMERGING STRATEGIES, AND FUTURE DIRECTIONS FOR RESEARCH AND TREATMENT OF GENETIC HEMOGLOBINOPATHIES, INCLUDING COOLEY'S ANEMIA; (II) ATTRACT/SHOWCASE EARLY CAREER AND UNDERREPRESENTED INVESTIGATORS (INCLUDING WOMEN, ETHNIC/RACIAL MINORITIES, AND PERSONS WITH DISABILITIES) VIA SHORT TALKS, POSTERS, TRAVEL FELLOWSHIPS, AND TO PROVIDE OPPORTUNITIES TO INTERACT WITH SENIOR INVESTIGATORS; (III) DISSEMINATE THE CONFERENCE PROCEEDINGS TO THE INTERNATIONAL COMMUNITY; AND (IV) FOSTER COLLABORATION BETWEEN ACADEMIA, MEDICINE, INDUSTRY, AND GOVERNMENT TO PROMOTE KNOWLEDGE EXCHANGE AND SUCCESSFUL TRANSLATION OF RESEARCH INTO IMPROVED DIAGNOSTICS AND PATIENT THERAPIES FOR THALASSEMIA AND TREATMENT-RELATED ORGAN DAMAGE. CUTTING- EDGE TOPICS TO BE DISCUSSED DURING THIS SYMPOSIUM INCLUDE GLOBIN GENE REGULATION, IRON METABOLISM, MANAGEMENT OF CLINICAL COMPLICATIONS OF THALASSEMIA INCLUDING A FOCUS ON ALPHA THALASSEMIA, A MAJOR GLOBAL HEALTH PROBLEM, ASSESSMENT AND TREATMENT OF IRON OVERLOAD, RESULTS OF ONGOING CLINICAL TRIALS OF GENE THERAPY, HEMATOPOIETIC STEM CELL TRANSPLANTATION, AND NOVEL DRUGS, AS WELL AS INNOVATIVE APPROACHES TO GENE THERAPY. THE GOALS OF THIS SYMPOSIUM STRONGLY SUPPORT THE MISSION OF THE NATIONAL HEART, LUNG, AND BLOOD INSTITUTE AND NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES IN THAT THE MEETING WILL FOCUS ON MULTIPLE PRIORITY AREAS OF NEED: TO ADVANCE HEMATOLOGY RESEARCH, TREATMENT AND CURE OF DISORDERS OF HEMOGLOBIN, AND TO IMPROVE SAFETY AND EFFICACY IN BLOOD TRANSFUSION MEDICINE - INCLUDING THE DIAGNOSIS, MANAGEMENT, AND PREVENTION OF IRON OVERLOAD AND SUBSEQUENT DAMAGE TO THE LIVER, HEART, AND ENDOCRINE SYSTEM. DISCUSSIONS ORIGINATING FROM THE CONFERENCE, AND THEIR DISSEMINATION VIA PUBLICATION IN ANNALS OF THE NEW YORK ACADEMY OF SCIENCES, WILL HAVE GLOBAL IMPACT BY IMPROVING SCIENTIFIC UNDERSTANDING, ADVANCING BREAKTHROUGH THERAPIES, AND ULTIMATELY REDUCING THE HEALTHCARE BURDEN OF THIS FAMILY OF GENETIC BLOOD DISORDERS.