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Hemophilia

FDA Grants Breakthrough Therapy Designation to uniQure’s Investigational Hemophilia B Drug

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Jan. 30, 2017 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ:QURE), a leader in human gene therapy, today announced that AMT-060,  its proprietary, investigational gene therapy in patients with severe hemophilia B, has received Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA).  This designation …

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Takeda to Acquire ARIAD Pharmaceuticals for $5.2 Billion

CAMBRIDGE, Mass. & OSAKA, Japan–(BUSINESS WIRE)–Takeda Pharmaceutical Company Limited (TSE:4502) (“Takeda”) and ARIAD Pharmaceuticals, Inc. (NASDAQ:ARIA) (“ARIAD”) today announced that they have entered into a definitive agreement under which Takeda will acquire all of the outstanding shares in ARIAD for $24.00 per share in cash, or an enterprise value of …

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Genentech’s Emicizumab for Hemophilia A Meets Primary Endpoint in Phase III Study

December 22, 2016 01:00 AM Eastern Standard Time SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the primary endpoint has been met for the Phase III HAVEN 1 study evaluating emicizumab prophylaxis in people 12 years of age or …

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UT Austin Engineers Develop First-Ever Capsule to Treat Hemophilia

In the near future, hemophiliacs could be able to treat their disease by simply swallowing a capsule. Thanks to a breakthrough led by researchers in the Cockrell School of Engineering at The University of Texas at Austin, treatment for hemophilia can now be administered via a biodegradable system, a capsule, …

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Sanofi Genzyme to Co-Develop and Co-Commercialize Hemophilia Candidate with Alnylam

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company today announced that, pursuant to the companies’ global alliance signed in January 2014, Sanofi Genzyme elected to opt in to co-develop (through Sanofi R&D) and co-commercialize fitusiran, an investigational RNAi therapeutic for the treatment of hemophilia and rare bleeding …

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In Crafting New Treatments for Hemophilia, A ‘Less is More’ Approach

Hematology researchers have found that blocking the role of a common protein may offer unexpected benefits for patients with the inherited bleeding disorder hemophilia A. The finding offers potential for developing both gene therapy and more effective protein replacement treatments for hemophilia A, the most common form of hemophilia. In …

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Biogen to Name its Hemophilia Drug Spinoff Bioverativ

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Biogen (NASDAQ: BIIB) today announced that Bioverativ will be the name of the standalone, publicly-traded global biotechnology company that it expects to launch in early 2017. Bioverativ will be focused on the discovery, research, development and commercialization of treatments for hemophilia and other blood disorders. Following completion of …

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Biogen to Spin Off Hemophilia Business into Separate Company

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Biogen Inc. (NASDAQ: BIIB) today announced that it intends to spin off its hemophilia business as an independent, publicly traded company. The strategic goal of this transaction is to create two focused companies dedicated to driving current and future value in their respective therapeutic areas of expertise. “We …

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Kedrion Biopharma Receives FDA Approval to Package Koāte® Double Viral Inactivation (DVI) Antihemophilic Factor (human) with Mix2VialTM Needle-Free Transfer Device

FORT LEE, N.J.–(BUSINESS WIRE)–Kedrion Biopharma: Kedrion Biopharma will extend its focus on patients by offering Mix2Vial™, a needle-free, double-ended, all plastic filter transfer set for use in reconstituting Koāte® Double Viral Inactivation (DVI) Antihemophilic Factor (human), a lyophilized (i.e., powdered) human factor-derived clotting factor Mix2Vial reduces exposure to accidental needle …

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CSL Behring’s Hemophilia B Drug Idelvion Now Available in the US

CSL Behring announced today that IDELVION® [Coagulation Factor IX (Recombinant), Albumin Fusion Protein], its novel, long-acting recombinant albumin fusion protein for treating hemophilia B, is now available nationwide. IDELVION is the first and only factor IX therapy that delivers high-level protection with up to 14-day dosing in appropriate patients. This extended dosing interval …

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