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SYMPOSIUM THEME
Hemoglobin S - Making an Evolutionary Perfected Process, Imperfect 

Journal of Sickle Cell Disease and Hemoglobinoopathies
SYMPOSIUM ABSTRACTS

Meeting Location:

Greater Ft. Lauderdale Convention Center
1950 Eisenhower Blvd, Fort Lauderdale, Florida, USA  33316
April 28 - 30, 2017 
Website:  fscdr.org
Email:  symposium@fscdr.org
Is this a CME Medical Meeting?  Yes
 
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Friday, April 28 • 4:00pm - 6:00pm
Investigational Drugs, Therapeutic and Device Symposium

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W. Keith Hoots, M.D., N.I.H

Ted W. Love, M.D. 
GBT440: Addressing the Fundamental Cause of Sickle Cell Disease 

Amhed Daak, M.D., Sancilio 
SC411: Cervonic Acid-Based treatment for children with Sickle Cell Disease

JSCDH-D-17-00043,  Edward Gomperts, M.D., Hillhurst Bio

HBI-002, an oral carbon monoxide therapeutic, is a modulator of vaso-occlusion in transgenic sickle cell mice 

JSCDH-D-17-00007, Ronald Jubin, Ph.D., Prolong Pharmaceuticals

SANGUINATE Returns RBCs To More Normal Morphology In Patients With VOC 

JSCDH-D-17-00005, Josh Lehrer, Global Blood Therapeutics Inc

Long-Term Dosing  in Sickle Cell Disease Subjects with GBT440, A novel HbS polymerization inhibitor

JSCDH-D-17-00008, Ken Bridges, Global Blood Therapeutics Inc
The 9-item Sickle Cell Disease Severity Measure (SCDSM): A novel measure of daily SCD symptom severity developed to assess benefit of GBT440, an experimental HbS polymerization inhibitor

JSCDH-D-17-00016, Robert J. Charnigo, Pfizer
Safety and Tolerability of PF-04447943 Across a Clinical Trial Program Including 267 Patients

JSCDH-D-17-00026,  Osheiza Abdulmalik, M.D., Children's Hospital of Philadelphia 
Anti-sickling Properties of a Novel Structurally-Enhanced Pyridyl Derivatives of Vanillin

Moderators
avatar for Gregory J. Kato, M.D.

Gregory J. Kato, M.D.

Professor Of Medicine, University of Pittsburgh, Department of Hematology-Oncology; Heart, Lung, Blood, and Vascular Medicine Institute
Dr. Kato is the Director of the Adult Sickle Cell Center at the Pittsburgh Heart, Lung, Blood and Vascular Medicine Institute and Professor of Medicine at the University of Pittsburgh.  He attended George Washington University School of Medicine and Health Sciences, completed residency in pediatrics at... Read More →

Speakers
avatar for W. Keith Hoots, M.D.

W. Keith Hoots, M.D.

Director of Blood Disease and Resources at the National Heart, Lung and Blood Institute (NHLBI) NIH National Blood and Heart Institue, National Institute of Health
W. Keith Hoots, M.D., is the director of the Division of Blood Diseases and Resources at the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health (NIH). In this role, Dr. Hoots plans and directs research and research training on the causes... Read More →
avatar for Edward Gomperts, M.D.

Edward Gomperts, M.D.

Executive Chairman, Hillhurst Biopharmaceuticals, Inc
My academic focus has been on non-oncologic hematology throughout my career. I am currently Emeritus Clinical Professor of Pediatrics and Pathology at the University of Southern California and an attending hematologist at Children’s Hospital, Los Angeles. I spent over 15 years i... Read More →
avatar for Josh Lehrer, M.D.

Josh Lehrer, M.D.

Vice President, Clinical Development, Global Blood Therapeutics
Dr. Lehrer is a cardiologist, physician scientist and biotechnology executive. He is currently Vice President, Clinical Development at Global Blood Therapeutics, where he has overseen the clinical development program strategy and execution for GBT440, bringing this molecule through early stage studies into a pivotal Phase 3 program in Sickle Cell Disease. Prior to GBT, Dr. Lehrer led clinical development programs through proof of concept at Genentech in multiple indications and held key roles in business development and overseeing cardiovascular safety. Dr. Lehrer has served as adjunct faculty in the Division of Cardiology at Stanford School of... Read More →
avatar for Ted W. Love, M.D.

Ted W. Love, M.D.

Chief Executive Officer, Global Blood Therapeutics
Dr. Love has more than 20 years of broad leadership and management experience in the pharmaceutical industry. Dr. Love most recently served as executive vice president, research and development and technical operations at Onyx Pharmaceuticals, where he played an instrumental role in initiating and completing several of... Read More →
avatar for Kenneth R. Bridges, M.D.

Kenneth R. Bridges, M.D.

Vice President, Medical Affairs, Global Blood Therapeutics
Dr. Kenneth R. Bridges received the MD degree from Harvard Medical School, and subsequently trained in internal medicine and hematology in Boston, at Massachusetts General and Brigham and Women’s Hospitals, respectively. Following medical subspecialty training, Dr. Bridges worked on the biology of cellular iron metabolism for three years at the National Institutes of Health in Bethesda, Maryland. Dr. Bridges returned to Harvard as a member of the Hematology Division at Brigham and... Read More →
avatar for Robert J. Charnigo, M.S.

Robert J. Charnigo, M.S.

Senior Director, Pfizer
Bob is a Senior Director in Pfizer’s Early Clinical Development Organization specializing in Rare Disease. Bob has served as the clinical lead for the development of PDE9 inhibitor for the prophylactic treatment of sickle cell disease. | | Prior to his involvement on the PDE9i team, Bob served for approximately 10 years on the legacy Wyeth/Pfizer hemophilia team and ultimately served as the clinical lead for Xyntha and ReFacto AF (factor VIII replacement products for hemophilia A) during a period when the focus was on global clinical post-marketing studies aimed both at new indications and regulatory commitments. | | Overall, Bob has over 32 years of clinical research experience (phases 1-4) in large pharma, imaging and medical device companies. His experience covers numerous therapeutic areas including, sickle cell disease, hemophilia, oncology, infectious disease, cardiology, and respiratory diseases. Bob received his... Read More →
avatar for Ahmed Daak, Ph.D., MSc, MBBS

Ahmed Daak, Ph.D., MSc, MBBS

Vice President of Research and Clinical Development, Sancilio & Company, Inc.
Dr Daak has a Bachelor degree in Medicine, Bachelor degree in Surgery (MD), Masters in Medical Biochemistry, in addition he has a Ph.D. on Biomedical Science from London. Before joining SCI Dr. Daak was working at Harvard School of Public Health (HSPH), Harvard University, USA as... Read More →
avatar for Ronald Jubin, Ph.D.

Ronald Jubin, Ph.D.

Vice President of Research and Development, Prolong Pharmaceuticals
Dr. Ronald Jubin is Vice President of Research and Development at Prolong Pharmaceuticals, a clinical stage pharmaceutical company developing PEGylated protein therapeutics for clinical conditions with unmet needs. His responsibilities include leading the organization in both pipeline development and translational research that spans the pre-clinical IND-enabling efforts, CMC through proof of concept clinical studies, and biomarker discovery/method development to support clinical trials of... Read More →
avatar for Osheiza Abdulmalik, Ph.D.

Osheiza Abdulmalik, Ph.D.

Research Associate II, Rivella Laboratory, The Children's Hospital of Philadelphia, Research Institute
Dr. Abdulmalik’s research interests broadly encompass the hemoglobinopathies and red blood cell disorders, with particular focus on sickle cell disease and beta-thalassemia. Though both disorders are consequences of simple genetic mutations, the implications of these mutations are inherently complex, and include significant morbidities, lifelong challenges, reduced quality of life, and early mortality in patients; as well as substantial healthcare burden on families, caregivers, and society in general. Dr... Read More →


Friday April 28, 2017 4:00pm - 6:00pm
Floridian Ballroom A & B 3rd Floor Fort Lauderdale Convention Center