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Hematopoietics

FDA Grants Breakthrough Therapy Designation to Bristol-Myers Squibb’s Orencia to Help Prevent Acute Graft-Versus-Host Disease

PRINCETON, N.J.–(BUSINESS WIRE)–Bristol-Myers Squibb Company (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for ORENCIA® (abatacept) for the prevention of moderate to severe acute graft-versus-host disease (GvHD) in hematopoietic stem cell transplants from unrelated donors. There are no approved therapies for the prevention of …

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Omeros Reports Positive Data Across Primary and Secondary Endpoints in Pivotal Trial of HSCT-Associated Thrombotic Microangiopathy Patients Treated with Narsoplimab

SEATTLE–(BUSINESS WIRE)–Omeros Corporation (Nasdaq: OMER) today announced positive data from its pivotal clinical trial of the company’s novel investigational complement inhibitor narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), a frequently lethal complication of HSCT. These preliminary data were recently provided to FDA as part of …

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CRISPR Therapeutics and Vertex Announce Positive Early Results for its Investigational CTX001 for Severe Hemoglobinopathies

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Nov. 19, 2019 (GLOBE NEWSWIRE) — CRISPR Therapeutics (NASDAQ: CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase 1/2 clinical trials. One patient with transfusion-dependent …

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Vertex and Molecular Templates Collaborate to Discover and Develop Novel Targeted Conditioning Regimens to Enhance Hematopoietic Stem Cell Transplants

BOSTON and AUSTIN, Texas, Nov. 18, 2019 (GLOBE NEWSWIRE) — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Molecular Templates, Inc. (Nasdaq: MTEM; “Molecular Templates” or “MTEM”) today announced that the two companies have entered into a strategic research collaboration to discover and develop novel targeted conditioning regimens that may enhance the …

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Takeda’s Maribavir Beats Valcyte in a Phase 2 Study as a Potential Treatment of Cytomegalovirus Infection

OSAKA, Japan–(BUSINESS WIRE)–Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) today announced that the New England Journal of Medicine has published results of a Phase 2, randomised, 12-week, open-label study of TAK-620 (maribavir), an investigational, orally bioavailable antiviral compound being evaluated in patients with cytomegalovirus (CMV) infection after undergoing hematopoietic cell transplant or solid …

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Gamida Cell and the CIBMTR Announce Collaboration to Advance Research for Life-Saving Cellular Therapy

BOSTON & MILWAUKEE–(BUSINESS WIRE)–Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, and the CIBMTR® (Center for International Blood and Marrow Transplant Research®) today announced the entry into a research agreement to collect and analyze health outcomes data in patients with hematologic malignancies who receive an allogeneic hematopoietic …

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FDA Grants Fast Track Designation to Akari’s Nomacopan for Pediatric Hematopoietic Stem Cell Transplant-Related Thrombotic Microangiopathy

NEW YORK and LONDON, Aug. 14, 2019 (GLOBE NEWSWIRE) — Akari Therapeutics, Plc (Nasdaq: AKTX), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where the complement and/or leukotriene systems are implicated, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast …

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CRISPR Therapeutics and Vertex Announce Progress in Clinical Development Programs for the Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001

ZUG, Switzerland and CAMBRIDGE and BOSTON, Mass., Feb. 25, 2019 (GLOBE NEWSWIRE) — CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today reviewed recent progress in the clinical development programs for CTX001, an investigational, autologous, CRISPR/Cas9 gene-edited hematopoietic stem cell therapy being evaluated for patients suffering from severe hemoglobinopathies. The companies …

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FDA Grants Orphan Drug Designation to Omeros’ OMS721 for Treatment of Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy

SEATTLE–(BUSINESS WIRE)–Omeros Corporation (NASDAQ:OMER) today announced that OMS721 has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). OMS721 is Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin …

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FDA Lifts Clinical Hold on Vertex/CRISPR Therapeutics’ Investigational Gene Therapy CTX001 for the Treatment of Sickle Cell Disease

ZUG, Switzerland and BOSTON and CAMBRIDGE, Mass., Oct. 10, 2018 (GLOBE NEWSWIRE) — CRISPR Therapeutics (NASDAQ:CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold and accepted the Investigational New Drug application (IND) for CTX001 for the treatment of …

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