57th ASH Annual Meeting and Exposition (December 5-8, 2015): 112. Thalassemia and Globin Gene Regulation: Therapeutic Approaches to Thalassemia and Their Mechanisms

Thalassemia and Globin Gene Regulation Oral and Poster Abstracts
Oral

Valencia A (W415A), Level 4 (Orange County Convention Center)

Moderators:

Vip Viprakasit, MD, PhD, Siriraj Hospital, Mahidol University and Karin E Finberg, MD, PhD, Yale School of Medicine

Disclosures:

Viprakasit: Novartis: Consultancy , Honoraria , Research Funding , Speakers Bureau .

4:30 PM

Rap-536 (Murine ACE-536/Luspatercept) Inhibits Smad2/3 Signaling and Promotes Erythroid Differentiation By Restoring GATA-1 Function in Murine b-Thalassemia

Pedro A. Martinez, PhD¹, Rajasekhar NVS Suragani, PhD², Manoj Bhasin, PhD³^*, Robert Li⁴^*, Robert Scott Pearsall, PhD⁴^* and Ravindra Kumar, PhD⁵^*

¹PreClinical Pharmacology, Acceleron Pharma Inc, Cambridge, MA
²Preclinical Pharmacology, Acceleron Pharma, Cambridge, MA
³BIDMC Genomics, Proteomics, Bioinformatics and Systems Biology Center, Boston, MA
⁴Preclinical Pharmacology, Acceleron Pharma Inc, Cambridge, MA
⁵Biology, Acceleron Pharma, Cambridge, MA

4:45 PM

752

Luspatercept (ACE-536) Reduces Disease Burden, Including Anemia, Iron Overload, and Leg Ulcers, in Adults with Beta-Thalassemia: Results from a Phase 2 Study

Antonio Piga, MD¹, Silverio Perrotta, MD²^*, M.Rita Gamberini, MD³^*, Ersi Voskaridou, MD⁴^*, Angela Melpignano⁵^*, Aldo Filosa, MD⁶^*, Antonello Pietrangelo, MD⁷^*, Vincenzo Caruso, MD⁸^*, Gian Luca Forni, MD⁹^*, Xiaosha Zhang¹⁰^*, Ashley Bellevue¹⁰^*, Dawn Wilson¹⁰^*, Abderrahmane Laadem, MD¹¹, Matthew L. Sherman, MD¹⁰ and Kenneth M. Attie, MD¹⁰

¹Turin University, Turin, Italy
²Second University of Naples, Naples, Italy
³University of Ferrara, Ferrara, Italy
⁴Laiko General Hospital, Athens, Greece
⁵Ospedale A. Perrino, Brindisi, Brindisi, Italy
⁶AORN "A. Cardarelli", Naples, Italy
⁷University of Modena, Modena, Italy
⁸Garibaldi Hospital, Catania, Italy
⁹Hospital Galliera, Genova, Italy
¹⁰Acceleron Pharma, Cambridge, MA
¹¹Celgene Corporation, Summit, NJ

5:00 PM

753

Targeting TMPRSS6 Using Antisense Technology for the Treatment of Beta-Thalassemia

Shuling Guo, PhD¹^*, Mariam Aghajan, PhD¹^*, Carla Casu, PhD²^*, Sara Gardenghi, PhD²^*, Sheri Booten¹^*, Stefano Rivella, PhD³ and Brett P. Monia, PhD⁴

¹Isis Pharmaceuticals, Inc., Carlsbad, CA
²Weill Cornell Medical College, New York, NY
³Weill Cornell Medical College, New York
⁴Antisense Drug Discovery, Isis Pharmaceuticals, Inc., Carlsbad, CA

5:15 PM

754

Down-Regulation of TfR1 Increases Erythroid Precursor Enucleation and Hepatocyte Hepcidin Expression in ß-Thalassemic Mice

Huihui Li, MS¹^*, Tenzin Choesang, MS¹^*, Weili Bao, MS¹^*, Lionel Blanc, PhD², Huiyong Chen, PhD¹^*, Maria Feola, MS¹^*, Jie Li, MD³^*, Guiyuan Li, MD⁴^*, Shuming Sun, PhD¹^*, Antonia Follenzi, MD PhD⁵^*, Petra Pham, MS⁶^*, Xiuli An, MD, PhD³, Narla Mohandas, DSc¹, Robert E Fleming, MD⁷, Stefano Rivella, PhD⁸ and Yelena Ginzburg, MD¹

¹New York Blood Center, New York, NY
²The Feinstein Institute for Medical Research, Manhasset, NY
³Laboratory of Membrane Biology, New York Blood Center, New York, NY
⁴Cancer Research Institute, Central South University, Changsha, China
⁵University of Piemonte Orientale, Amedeo Avogadro, Novara, Italy
⁶Platelet Biology Laboratory, New York Blood Center, New York, NY
⁷Pediatrics, Saint Louis University School of Medicine, St. Louis, MO
⁸Weill Cornell Medical College, New York

5:30 PM

755

Role of Pleckstrin 2 in Improved Erythropoiesis of Transferrin-Treated Beta-Thalassemic Mice

Maria Feola, MS^1,2^*, Andrea Zamperone³^*, Weili Bao, MS¹^*, Tenzin Choesang, MS¹^*, Huihui Li, MS^1,4^*, Guiyuan Li, MD⁴^*, Antonia Follenzi, MD PhD²^*, Shilpa M Hattangadi, M.D.⁵, Christopher Mason, PhD⁶^*, Peng Ji, MD, PhD⁷ and Yelena Ginzburg, MD¹

¹New York Blood Center, New York, NY
²University of Piemonte Orientale, Amedeo Avogadro, Novara, Italy
³Albert Einstein College of Medicine, Bronx, NY
⁴Cancer Research Institute, Central South University, Changsha, China
⁵Division, Pediatric Hematology-Oncology, Yale School of Medicine, New Haven, CT
⁶Meyer Cancer Center, Weill Cornell Medical College, New York, NY
⁷Pathology, Northwestern University, Chicago, IL

5:45 PM

756

Loss of Foxo3 Reduces Erythroblast Apoptosis and Enhances RBC Production in Beta-Thalassemic Mice

Raymond Liang, B.S., Genís Campreciós, PhD, Carolina L. Bigarella, PhD and Saghi Ghaffari, MD, PhD

Department of Developmental and Regenerative Biology, Icahn School of Medicine at Mount Sinai, New York, NY

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112.O2.6 112. Thalassemia and Globin Gene Regulation: Therapeutic Approaches to Thalassemia and Their Mechanisms