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Sangamo and Pfizer Announce Phase 1/2 Interim Data for Investigational Hemophilia A Gene Therapy

BRISBANE, Calif. & NEW YORK–(BUSINESS WIRE)–Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company, and Pfizer, Inc. (NYSE: PFE) today announced interim data from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy for severe hemophilia A. Data indicate that SB-525 was generally well-tolerated and demonstrated a dose-dependent increase …

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FDA Approves Novo Nordisk’s Esperoct for Treatment of Adults and Children with Hemophilia A

Bagsværd, Denmark, 19 February 2019 – Novo Nordisk today announced that the US Food and Drug Administration (FDA) has approved the Biologics License Application for ESPEROCT® for the treatment of adults and children with hemophilia A. ESPEROCT® is the brand name for turoctocog alfa pegol, N8-GP. ESPEROCT® is indicated for use in adults and …

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UniQure Announces Updated Clinical Data from Phase IIb Study of AMT-061 in Patients with Hemophilia B

LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Feb. 08, 2019 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced updated clinical data in patients treated in the Company’s ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene therapy containing …

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Takeda Announces Results from IIIb/IV Trial – a Randomized PK-Guided Prophylaxis Study Evaluating Higher Factor VIII Levels in Hemophilia A

CAMBRIDGE, Mass. & OSAKA, Japan–(BUSINESS WIRE)–Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”), the global biotechnology leader in rare diseases, has today announced results from its phase IIIb/IV clinical trial for ADYNOVATE at the 12th Annual Congress of the European Association of Haemophilia and Allied Disorders (EAHAD), taking place 6-9 February, in Prague, Czech …

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Genentech’s Hemlibra Provided Sustained Bleed Control in the Largest Pivotal Study to Date of Children With Hemophilia A With Factor VIII Inhibitors

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today data from the primary analysis of the Phase III HAVEN 2 study evaluating Hemlibra® (emicizumab-kxwh) prophylaxis in children younger than 12 years of age with hemophilia A with factor VIII inhibitors, including longer …

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uniQure Announces Initial Topline Data from Dose-Confirmation Study of AMT-061 in Patients with Hemophilia B

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Nov. 15, 2018 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced initial clinical data in patients treated in the Company’s Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy containing …

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FDA Approves Genentech’s Hemlibra for Hemophilia A Without Factor VIII Inhibitors

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved Hemlibra® (emicizumab-kxwh) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with …

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FDA Approves Bayer’s Jivi for the Routine Prophylactic Treatment of Hemophilia A

WHIPPANY, N.J., Aug. 30, 2018 /PRNewswire/ — Bayer announced today that the U.S. Food and Drug Administration (FDA) has approved Jivi® (BAY94-9027, antihemophilic factor [recombinant] PEGylated-aucl) for the routine prophylactic treatment of hemophilia A in previously treated adults and adolescents 12 years of age or older. The initial recommended prophylactic …

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Positive Phase 3 Results for Genentech’s Hemlibra for Hemophilia A without Factor VIII Inhibitors Published in NEJM

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that pivotal data from the Phase III HAVEN 3 study, which evaluated HEMLIBRA® (emicizumab-kxwh) prophylaxis administered every week or every two weeks in adults and adolescents aged 12 years or older with hemophilia …

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Sangamo Announces Positive Preliminary Data from the Phase 1/2 Study Evaluating SB-525 Gene Therapy for Hemophilia A

RICHMOND, Calif., Aug. 8, 2018 /PRNewswire/ — Sangamo Therapeutics, Inc. (Nasdaq: SGMO) today announced positive preliminary data from the Phase 1/2 clinical trial evaluating SB-525, a cDNA gene therapy candidate for Hemophilia A (the “Alta study”). SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer …

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