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Hemophilia

Shire Enters Bispecific Hemophilia Research and Development Deal with Novimmune

Lexington, MA – July 18, 2017 – Shire plc (LSE: SHP, NASDAQ: SHPG), the biotech leader in Rare Diseases; and Novimmune S.A., a privately-held Swiss biopharmaceutical company focused on the discovery and development of innovative, antibody-based medicines today announced they have entered into an agreement granting Shire exclusive worldwide rights to …

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Alnylam and Sanofi Genzyme Initiate Phase 3 Program of Investigational RNAi Therapeutic Fitusiran

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, and Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today the initiation of the ATLAS Phase 3 clinical program for fitusiran. The global, multicenter program is designed to evaluate the safety and efficacy of fitusiran in three separate …

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Shire Submits IND for its Gene Therapy for the Treatment of Hemophilia A

Lexington, Mass. – July 6, 2017 – Shire plc (LSE: SHP, NASDAQ: SHPG), the leading biotechnology company focused on serving people with rare diseases, today announced the submission of an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for SHP654, also designated as BAX 888, an investigational factor VIII …

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Genentech’s Emicizumab Demonstrates Positive Results in Late-Stage Studies in Hemophilia A with Inhibitors

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today positive data from the primary analysis of the Phase III HAVEN 1 study in adults and adolescents and interim analysis of the Phase III HAVEN 2 study in children evaluating once-weekly subcutaneous …

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FDA Accepts Bioverativ’s IND Application for its Hemophilia A Drug BIVV001

WALTHAM, Mass.–(BUSINESS WIRE)–Bioverativ (NASDAQ: BIVV), a global biotechnology company focused on the discovery, development and commercialization of innovative therapies for hemophilia and other rare blood disorders, today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Investigational New Drug (IND) application for BIVV001 (also known as …

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FDA Approves New Novo Nordisk Hemophilia B Treatment

PLAINSBORO, N.J., May 31, 2017 /PRNewswire/ — Novo Nordisk today announced that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application for REBINYN® (Coagulation Factor IX (Recombinant), GlycoPEGylated) for the treatment of adults and children with hemophilia B. Hemophilia B is a chronic and inherited bleeding …

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Shire’s Hemophilia Candidate Using Xenetic Technology Platform Fails in Phase 1/2 Trial

LEXINGTON, Mass.–(BUSINESS WIRE)–Xenetic Biosciences, Inc. (NASDAQ:XBIO) (“Xenetic” or the “Company”), a clinical-stage biopharmaceutical company focused on the discovery, research and development of next-generation biologic drugs and novel orphan oncology therapeutics, announced today that it has received an update on the Phase 1/2 clinical study conducted by its partner Shire, evaluating …

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Sangamo’s Hemophilia A Gene Therapy Candidate Receives Fast Track Status from the FDA

RICHMOND, Calif., May 16, 2017 /PRNewswire/ — Sangamo Therapeutics, Inc. (NASDAQ: SGMO) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SB-525, the Company’s clinical stage cDNA gene therapy candidate for hemophilia A, which is being developed as part of an exclusive, global …

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Pfizer Enters Gene Therapy Deal with Sangamo, Expanding Hemophilia Pipeline

Sangamo Therapeutics, Inc. (Nasdaq: SGMO) and Pfizer Inc. (NYSE: PFE) today announced an exclusive, global collaboration and license agreement for the development and commercialization of gene therapy programs for Hemophilia A, including SB-525, one of Sangamo’s four lead product candidates, which Sangamo expects will enter the clinic this quarter. “Sangamo …

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Three Sangamo Clinical Programs Receive Special Regulatory Designations from the FDA

RICHMOND, Calif., May 4, 2017 /PRNewswire/ — Sangamo Therapeutics, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, announced today that the Company has received notice from the U.S. Food and Drug Administration (FDA) of special regulatory designations for three of the Company’s clinical programs. Rare Pediatric Disease (RPD) designation …

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