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bluebird bio and Novo Nordisk Partner to Develop in vivo Genome Editing Candidates for Hemophilia and Other Severe Genetic Diseases

CAMBRIDGE, Mass. and BAGSVÆRD, Denmark–(BUSINESS WIRE)–bluebird bio, Inc. (Nasdaq: BLUE) and Novo Nordisk A/S (NVO) today announced that they have entered into a research collaboration to jointly develop next-generation in vivo genome editing treatments for genetic diseases, including hemophilia. During the three-year research collaboration, bluebird and Novo Nordisk will focus on identifying a development …

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FDA Approves Octapharma’s Wilate for the Treatment of Adults and Adolescents with Hemophilia A

HOBOKEN, N.J.–(BUSINESS WIRE)–Octapharma USA today announced the U.S. Food and Drug Administration (FDA) has approved WILATE® for treatment of adults and adolescents with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes and on demand treatment and control of bleeding episodes. WILATE® is a human plasma-derived, sterile, purified, double virus inactivated von Willebrand …

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Sigilon’s SIG-001 Receives Orphan Drug Designation from the FDA for Treatment of Hemophilia A

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sigilon Therapeutics today announced it has received Orphan Drug Designation for SIG-001, an investigational therapy for hemophilia A that leverages Sigilon’s Shielded Living Therapeutics™ platform to implant cells engineered to produce stable blood plasma levels of factor VIII, a crucial clotting protein. “We are very pleased to have received …

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Head-to-Head Study Demonstrated Improved PK Characteristics for Jivi Compared to Eloctate in Patients with Severe Hemophilia A

WHIPPANY, N.J., Aug. 13, 2019 /PRNewswire/ — A head-to-head study directly comparing the pharmacokinetic (PK) profiles of two extended half-life (EHL) recombinant factor VIII (FVIII) therapies demonstrated improved PK characteristics for Jivi® (antihemophilic factor [recombinant] PEGylated-aucl) compared to Eloctate® (recombinant FVIII fusion protein) in adult patients (n=17) with severe hemophilia …

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Pfizer and Sangamo’s Investigational Hemophilia A Gene Therapy Shows Sustained Increased Factor VIII Levels in Phase 1/2 Results

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

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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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