HTRS 2021 Scientific Symposium PRELIMINARY VIRTUAL MEETING AGENDA HTRS Research Colloquium: March 8-9 FWGBD Colloquium: March 9 Multidisciplinary Pre-Conference: March 10-11 Agenda: Day 1 | Day 2 Agenda Agenda: Day 1 | Day 2 HTRS Scientific Symposium: March 10-12 Agenda: Day 1 | Day 2 | Day 3 MONDAY, MARCH 8, 2021 HTRS Research Colloquium: Day 1 – separate registration required Eastern Time Event 12:30 PM – 1:00 PM ET PRE-MEETING NETWORKING “BRING YOUR OWN LUNCH” 1:00 PM ET OPENING REMARKS Wolfgang Bergmeier, PhD, and Margaret V. Ragni, MD, MPH, 2021 Colloquium Co-Chairs 1:05 PM – 1:50 PM ET PLENARY SESSION: “HOW DO I BEGIN, AND WHO IS GOING TO PAY FOR THIS?” RESEARCH FUNDING TRENDS AND OPPORTUNITIES Moderator: Margaret V. Ragni, MD, MPH Speaker: Andrei Kindzelski, MD, National Institutes of Health (NIH) 1:50 PM – 2:20 PM ET NETWORKING BREAK -OR- MENTORING SESSIONS 2:20 PM – 3:20 PM ET PANEL PRESENTATION: “GETTING A JOB – A RECRUITER’S PERSPECTIVE” Moderator: Wolfgang Bergmeier, PhD 2:20 PM BASIC SCIENCE PERSPECTIVE James Morrissey, PhD, Professor, University of Michigan 2:35 PM CLINICAL SCIENCE PERSPECTIVE Janis Abkowitz, PhD, Professor, Hematology Division Head, University of Washington 1 HTRS Research Colloquium: Day 1 – continued Eastern Time Event 2:50 PM TRANSLATIONAL SCIENCE PERSPECTIVE Jorge Di Paola, MD, Professor/Division Chief, Washington University in St. Louis 3:05 PM INDUSTRY PERSPECTIVE Robert Peters, PhD, Sanofi Genzyme 3:20 PM – 4:00 PM ET ROUNDTABLE DISCUSSIONS • Basic Science Tables • Clinical Science Tables • Translational Science Tables • Population Science Tables 4:00 PM ET DAY ONE PROGRAM ENDS Session resumes Tuesday, March 9 at 12:30 PM ET TUESDAY, MARCH 9, 2021 FWGBD Colloquium on Uterine Hemostasis – separate registration required Eastern Time Event 9:30 AM – 9:45 AM ET CONVENE AND WELCOME Andra H. James, MD, MPH 9:45 AM – 9:55 AM ET UTERINE HEMOSTASIS OVERVIEW 9:55 AM – 10:15 AM ET WOMEN & GIRLS WITH HEMOPHILIA Sarah H. O’Brien, MD, MSc 10:15 AM – 10:30 AM ET BREAK 10:30 AM – 11:00 AM ET MANAGEMENT OF DELIVERY IN WOMEN WITH HEMOPHILIA Andra H. James, MD, MPH 11:00 AM – 11:30 AM ET RISK OF CLOTTING ON HORMONAL THERAPY Naomi Tepper, MD, MPH 11:30 AM – 12:05 PM ET IS IT HELLP SYNDROME? IS IT TTP? IS IT ATYPICAL HUS? DOES IT MATTER? Gowthami Arepally, MD 12:05 – 12:30 PM ET DISCUSSION AND WRAP-UP 2 TUESDAY, MARCH 9, 2021 – continued HTRS Research Colloquium: Day 2 – separate registration required Eastern Time Event 12:30 PM – 1:00 PM ET PRE-MEETING NETWORKING “BRING YOUR OWN LUNCH” 1:00 PM – 1:05 PM ET DAY TWO - OPENING REMARKS Ming Y. Lim, MD/MBBCH, MSCr, and Joshua Muia, PhD, 2021 Colloquium Planning Committee Members 1:05 PM – 1:50 PM ET PLENARY SESSION: “CRITICAL STEPS FOR A SUCCESSFUL CAREER IN ACADEMIC MEDICINE” Moderator: Mary Cushman, MD, MSc Speaker: Mark T. Gladwin, MD, Jack D. Myers Distinguished Professor and Chair, Chairman of the Department of Medicine, UPMC and the University of Pittsburgh School of Medicine 1:50 PM – 2:10 PM ET BREAKOUT SESSION 1: “PRACTICAL TOOLS EVERYONE NEEDS TO ADVANCE THEIR CAREERS” BREAKOUT 1 SPEAK UP: GIVING EFFECTIVE PRESENTATIONS Discussion Leader: Alisa S. Wolberg, PhD BREAKOUT 2 MANAGING THE K-TO-R TRANSITION: WHAT TO DO IF YOU DON’T GET A K-AWARD Discussion Leader: Nigel Key, MB, ChB, FRCP BREAKOUT 3 FROM GOOD IDEA TO SUBMITTED GRANT Discussion Leader: Gowthami Arepally, MD BREAKOUT 4 BEGIN AGAIN: JUMP START YOUR RESEARCH CAREER AFTER A BREAK Discussion Leader: Menaka Pai, BSc, MSc, MD, FRCPC 2:10 PM – 2:30 PM ET “GET UP AND STRETCH BREAK’ -OR- MENTORING SESSIONS 2:30 PM – 2:50 PM ET BREAKOUT SESSION 2: “PRACTICAL TOOLS EVERYONE NEEDS TO ADVANCE THEIR CAREERS” BREAKOUT 1 SPEAK UP: GIVING EFFECTIVE PRESENTATIONS Discussion Leader: Alisa S. Wolberg, PhD BREAKOUT 2 MANAGING THE K-TO-R TRANSITION: WHAT TO DO IF YOU DON’T GET A K-AWARD Discussion Leader: Nigel Key, MB, ChB, FRCP BREAKOUT 3 FROM GOOD IDEA TO SUBMITTED GRANT Discussion Leader: Gowthami Arepally, MD BREAKOUT 4 BEGIN AGAIN: JUMP START YOUR RESEARCH CAREER AFTER A BREAK Discussion Leader: Menaka Pai, BSc, MSc, MD, FRCPC 3 HTRS Research Colloquium: Day 2 – continued Eastern Time Event 2:50 PM – 3:35 PM ET PANEL PRESENTATION: “OVERCOMING OBSTACLES: MOVING YOUR CAREER FORWARD” Moderator: Rodney Camire, PhD 2:50 PM BASIC/TRANSLATIONAL SCIENCE PERSPECTIVE Veronica H. Flood, MD 3:05 PM CLINICAL SCIENCE PERSPECTIVE Margaret V. Ragni, MD, MPH 3:20 PM INDUSTRY PERSPECTIVE Marcus Carr, MD 3:35 PM – 4:20 PM ET CLOSING RECEPTION “BRNG YOUR OWN SNACKS 4:20 PM ET DAY TWO PROGRAM ENDS WEDNESDAY, MARCH 10, 2021 Multidisciplinary Pre-Conference: Day 1 – separate registration required Eastern Time Event 10:00 AM – 10:05 AM ET DAY ONE - OPENING REMARKS Loren D’Angelo, RN-BC, BSN, MSN, CPNP-PC, HTRS 2021 Planning Committee 10:05 AM – 11:30 AM ET PRE-CONFERENCE SESSION 1: “NEW THERAPIES” Moderators: Loren D’Angelo, RN-BC, BSN, MSN, CPNP-PC, and Caitlin Moncrieff, NP 10:05 AM DOACS IN THE PEDIATRIC POPULATION Riten Kumar, MD, MSc 10:35 AM MANAGING INHIBITORS IN THE REALM OF NON-FACTOR THERAPIES James Munn, RN-BC, BS, BSN, MS 4 Multidisciplinary Pre-Conference: Day 1 – continued 10:55 AM “GENE THERAPY CLINICAL TRIAL EXPERIENCES IN CARE” Darcy Phelan, NP, MSN 11:20AM Live Q&A 11:30 AM DAY ONE PROGRAMMING ENDS Session resumes Thursday, March 11 at 10:00 AM ET HTRS Scientific Symposium: Day 1 Eastern Time Event 12:00 PM – 1:00 PM ET SATELLITE SYMPOSIA: (2) Concurrent Sessions Sanofi Genzyme: "siRNA Technology in Hemophilia" CSL Behring: “On-Demand Bleed Resolution in Hemophilia B With an Extended Half-Life Factor IX Therapy” 1:00 PM – 1:05 PM ET OPENING REMARKS Jordan Shavit, MD, PhD, HTRS 2021 Planning Committee Chair Ayesha N. Zia, MD, HTRS 2021 Planning Committee Vice Chair 1:05 PM – 2:30 PM ET SYMPOSIUM SESSION 1 DEBATE: “THE FUTURE OF HEMOPHILIA THERAPEUTICS: SUBSTITUTION OR GENE THERAPY (To GT or not to GT, a smackdown)” Moderators: Jordan Shavit, MD, PhD and Yasmina Abajas, MD Speakers: Denise E. Sabatino, PhD, Michael Callaghan, MD, and Shawn Jobe, MD, PhD 1:05 PM – 1:10 PM Case Presentation & Introduction to Audience Response System (“ARS”) Jordan Shavit, MD, PhD and Yasmina Abajas, MD 1:10 PM – 1:20 PM Introduction: “The science behind hemophilia therapeutics” Denise E. Sabatino, PhD 5 HTRS Scientific Symposium: Day 1 – continued SYMPOSIUM SESSION 1 DEBATE: “THE FUTURE OF HEMOPHILIA THERAPEUTICS: SUBSTITUTION OR GENE THERAPY (To GT or not to GT, a smackdown)” 1:20 PM – 1:25 PM Pre-Debate ARS Vote Jordan Shavit, MD, PhD and Yasmina Abajas, MD 1:25 PM – 1:40 PM Argument for Substitution Michael Callaghan, MD 1:40 PM – 1:55 PM Argument for Gene Therapy Shawn Jobe, MD, PhD 1:55 PM – 2:05 PM Rebuttal for Substitution Michael Callaghan, MD 2:05 PM – 2:15 PM Rebuttal for Gene Therapy Shawn Jobe, MD, PhD 2:15 PM – 2:25 PM Questions/Discussion Jordan Shavit, MD, PhD and Yasmina Abajas, MD 2:25 PM – 2:30 PM Post-Debate ARS Vote Jordan Shavit, MD, PhD and Yasmina Abajas, MD 2:30 PM – 3:30 PM ET NETWORKING & EXHIBITOR SPEED BREAK 6 HTRS Scientific Symposium: Day 1 – continued 3:30 PM – 4:30 PM ET ORAL ABSTRACT PRESENTATIONS: (2) Concurrent Sessions vWF/HEMOPHILIA A Novel Mechanisms & Trials Moderators: Christine Kempton, MD, MSc Moderators: A. Valance Washington, PhD and Joshua Muia, PhD and Lauren Amos, MD 3:30 PM “THE IMPORTANCE OF GENETIC TESTING IN DIAGNOSING VON “PLATELETS FACTOR 4 (PF4) BINDING ENHANCES IN VITRO WILLEBRAND'S DISEASE TYPE 2M” NEUTROPHIL EXTRACELLULAR TRAPS (NET) CAPTURE OF SARS- Presenter: Radia Ksayer, MD COV-2: THERAPEUTIC IMPLICATIONS” Presenter: Maki Ishizuka, MD 3:45 PM “VON WILLEBRAND FACTOR (VWF) MULTIPLEX ACTIVITY ASSAY “IN VITRO-GROWN MEGAKARYOCYTES (MKS) TAKE UP THE DIFFERENTIATION OF LOW VWF/TYPE 1 VON WILLEBRAND ECTOPIC PROTEIN UROKINASE (UPA) AND THE RESULTING DISEASE (VWD) AND VARIANT VWD: ANALYSIS FROM THE PLATELETS (PLTS) CAN RELEASE THE UPA AT SITES OF VASCULAR COMPARATIVE EFFECTIVENESS IN THE DIAGNOSIS OF VWD” INJURY” Presenter: Sergei V. Zaitsev, PhD Presenter: Jonathan C. Roberts, MD 4:00 PM Non-CME Presentation: “BROAD PATIENT ELIGIBILITY AND “BLEEDING DIATHESIS IN MICE LACKING JAK2 IN PLATELETS” LONG-TERM TOLERABILITY IN THE FIRST-IN-HUMAN GENE Presenters: Nathan Eaton, BSc THERAPY STUDY OF AAVHU37.HFVIIICO IN SEVERE HEMOPHILIA A” Presenter: Francesca Ferrante, MD 4:15 PM Non-CME Presentation: “REAL-WORLD DATA FROM A US “A RANDOMIZED, PRAGMATIC TRIAL TO ASSESS A PEDIATRIC CLAIMS DATABASE AMONG PATIENTS WITH HEMOPHILIA A REAL-TIME RISK PREDICTION MODEL FOR THROMBOTIC EVENTS” TREATED WITH RECOMBINANT FVIII FC FUSION PROTEIN” Presenters: Shannon C. Walker, MD Presenter: Amanda Wilson, MPH, PhD 4:30 PM - 5:30 PM ET OPEN NETWORKING 5:30 PM - 6:30 PM ET SATELLITE SYMPOSIA: (1) Session Takeda: “ADVATE® [Antihemophilic Factor (Recombinant)] Clinical Presentation” 6:30 PM ET DAY ONE PROGRAMMING ENDS General Session resumes Thursday, March 11 at 12:00 PM ET 7 THURSDAY, MARCH 11, 2021 Multidisciplinary Pre-Conference: Day 2 – separate registration required Eastern Time Event 10:00 AM – 10:05 AM ET DAY TWO - OPENING REMARKS Loren D’Angelo, RN-BC, BSN, MSN, CPNP-PC, HTRS 2021 Planning Committee 10:05 AM – 11:30 AM ET PRE-CONFERENCE SESSION 2: INCLUSIVE CARE Moderators: Mary Lesh, PNP, MSN and Caitlin Moncrieff, NP 10:05 AM TRAUMA INFORMED CARE FOR HEMATOLOGY PROVIDERS Amanda Stahl, LICSW 10:35 AM HEMATOLOGIC CONSIDERATIONS IN THE CARE OF TRANSGENDER PATIENTS Meredith Russel, PNP, MSN 11:15 AM Live Q&A 11:30 AM ET HTRS 2021 Multidisciplinary Pre-conference Ends HTRS Scientific Symposium: Day 2 Eastern Time Event 12:00 PM – 1:00 PM ET SATELLITE SYMPOSIA: (2) CONCURRENT SESSIONS Novo Nordisk: “A rFVIIa with over 30 years of experience with treating hemophilia and other rare bleeding disorders” Bayer: “Patient focused prophylaxis management for patients with hemophilia: a case-based discussion” 1:00 PM – 1:05 PM ET OPENING REMARKS Gowthami Arepally, MD, HTRS 2021 Planning Committee – Abstract Co-Chair Alan Mast, MD, PhD, HTRS 2021 Planning Committee – Abstract Co-Chair 8 HTRS Scientific Symposium: Day 2 – continued 1:05 PM – 2:10 PM ET SYMPOSIUM SCIENTIFIC SESSION 2: STATE-OF-THE-ART LECTURE: “COVID-19: THROMBOSIS AND CLINICAL CONSIDERATIONS” Moderator: Jordan A.