64th ASH Annual Meeting and Exposition: 321. Coagulation and Fibrinolysis: Basic and Translational I

Symposia: Coagulation and Fibrinolysis: Basic and Translational Oral and Poster Abstracts
Oral

Research, Fundamental Science, Bleeding and Clotting, bleeding disorders, Biological therapies, Antibody Therapy, Translational Research, hemophilia, platelet disorders, Genetic Disorders, drug development, Diseases, immune mechanism, Gene Therapy, Therapies, immunology, thrombotic disorders, VWD, Adverse Events, Infusion, Biological Processes, molecular biology, multi-systemic interactions, pathogenesis

293-294 (Ernest N. Morial Convention Center)

Moderators:

W. Keith Hoots, MD, M.D. Anderson Cancer Center & UT Health Science Center and Shekhar Kumar, PhD, Children's Hospital of Philadelphia

Disclosures:

No relevant conflicts of interest to declare.

This session is the first of two sessions that present scientific data gleaned from basic and translational work focused on explicating mechanisms of hemostasis , anti-hemostasis and fibrinolysis.

9:30 AM

397

An Antibody Targeting Human FV Promotes Thrombin Generation and Reduces Bleeding in a Hemophilic Mouse Model

Sean Quinn, PhD¹^*, Lacramioara Ivanciu, PhD^1,2, Robert J. Davidson, BS¹^*, Francis Ayombil, PhD¹ and Rodney M. Camire, Ph.D.^2,3

¹Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA
²Department of Pediatrics, University of Pennsylvania, Philadelphia, PA
³Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA

9:45 AM

398

Inhibiting Activated Protein C Cleavage of Factor VIII for Hemophilia a Gene Therapy

Anna R. Sternberg, PhD^1,2,3^*, Cristina Martos-Rus, MS^2,3^*, Robert J. Davidson, MS^2,3^*, Noelle C. Novak, BS^2,3^*, Amelia R. Wilhelm, BS^2,3^* and Lindsey A. George, MD^1,2,3

¹Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
²Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
³Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA

10:00 AM

399

Antigen Trafficking and Possibly Intrinsic Immunostimulatory Properties Shape High Immunogenicity of Factor VIII

Radoslaw Kaczmarek, PhD¹^*, Lindsey A. George, MD², Rodney M. Camire, Ph.D.² and Roland W Herzog, PhD³

¹Pediatrics, Indiana University School of Medicine, Indianapolis, IN
²The Children's Hospital of Philadelphia, Philadelphia, PA
³Department of Pediatrics, IUPUI, Indianapolis, IN

10:15 AM

400

Sustained Factor VIII Activity Following Single Dose of Non-Viral Integrating Gene Therapy

Brian Truong¹^*, Giang N. Nguyen, BS²^*, Khal Hajj, PhD¹^*, Joseph Lucas, PhD¹^*, Johannes Schwerk, PhD¹^*, James Yen¹^*, Kevin Briseno¹^*, Michelle Burrascano¹^*, Daniel Smith¹^*, Johnnatan Tamayo¹^*, Fernando Lopez-Espinoza¹^*, Michael Bennett, PhD¹^*, Blair Madison, PhD¹^*, Denise E. Sabatino, PhD² and Joshua Rychak¹^*

¹Poseida Therapeutics, San Diego, CA
²The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA

10:30 AM

401

Coagulation Factor XII Is Activated Via Noncanonical Binding to Platelet Integrin αIIbβ3

Sharjeel Chaudhry^1,2, Randall Burton³^*, Matthew Serrata²^*, Louis Feingold²^*, Lindsay Tomczak²^*, Lindsay Collier²^*, Dylan Laprise³^*, Keiichi Enjyoji, PhD^1,2^*, Virendar Kaushik³^*, Dylan Bertovich²^*, Alex Burgin³^* and Pavan K. Bendapudi, MD^1,2,4,5

¹Harvard Medical School, Boston, MA
²Division of Hemostasis and Thrombosis, Beth Israel Deaconess Medical Center, Boston, MA
³Center for the Development of Therapeutics, Broad Institute of MIT and Harvard, Cambridge, MA
⁴Center for the Development of Therapeutics, The Broad Institute of MIT and Harvard, Cambridge, MA
⁵Division of Hematology and Blood Transfusion Service, Massachusetts General Hospital, Cambridge, MA

10:45 AM

402

Selective Modulation of Activated Protein C Activities By an Exosite-Specific Nanobody Library

Derek S Sim, PhD¹^*, Meenal Shukla, PhD²^*, Cornell R Mallari, BS¹^*, Doug Schneider³^*, Maxine Bauzon, MS³^*, Terry W Hermiston, PhD¹^* and Laurent O. Mosnier, PhD⁴

¹Coagulant Therapeutics, Berkeley, CA
²The Scripps research Institute, La Jolla
³Consultant of Coagulant Therapeutics, Berkeley, CA
⁴Department of Molecular Medicine, The Scripps Research Inst., La Jolla, CA

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^*signifies non-member of ASH

321.O1.6 321. Coagulation and Fibrinolysis: Basic and Translational I