Hemophilia

FDA Accepts and Grants Priority Review to Biomarin’s BLA for Valoctocogene Roxaparvovec for Patients with Hemophilia A

SAN RAFAEL, Calif., Feb. 20, 2020 /PRNewswire/ — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) to the FDA for its investigational AAV5 gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A.  This …

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Light Chain Bioscience Achieves Milestone Payment for Successfully Completing Factor VIII Mimetic Bispecific Antibody Discovery for Takeda

Light Chain Bioscience, a company developing bispecific antibodies with a pipeline focused in immuno-oncology, announced that it has achieved a milestone and received a payment under its research and collaboration agreement with Takeda Pharmaceutical Company Limited (Takeda). The milestone was achieved upon the successful completion of the discovery phase and …

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Biomarin Submits Valoctocogene Roxaparvovec BLA to the FDA for Treatment of Hemophilia A

SAN RAFAEL, Calif., Dec. 23, 2019 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the company submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A.  Subject to completion of the …

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Bayer Collaborates with Children’s Hospital of Philadelphia to Research Novel Small Molecule Therapies for Oral Treatment of Hemophilia

WHIPPANY, N.J., Dec. 5, 2019 /PRNewswire/ — Bayer announced today that it has entered into a three-year collaboration agreement with Children’s Hospital of Philadelphia (CHOP) for the discovery and development of small molecules (SMOLs) to develop a first-in-class oral non-replacement therapy (NRT) for the treatment of hemophilia A and B. …

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Takeda and Enzyre to Develop Diagnostic Device Allowing Hemophilia Patients to Determine their Coagulation Status at Home

NIJMEGEN, Netherlands, Dec. 5, 2019 /PRNewswire/ — Enzyre, which is developing breakthrough ambulant diagnostic technology for coagulation testing, today announced that it has entered into a research collaboration agreement with Takeda Pharmaceutical Company Limited (Takeda) to develop a diagnostic device that will enable hemophilia patients to determine their coagulation status …

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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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