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uniQure Announces Updated, Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B

December 15, 2017

  Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here. uniQure N.V. a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced on Dec. 11, 2017, updated results from its ongoing, dose-ranging Phase I/II trial of AMT-060, its investigational gene […]

BioMarin Provides 1.5 years of Clinical Data for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

December 15, 2017

Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here. BioMarin Pharmaceutical Inc. announced an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia A. The data […]

Spark Therapeutics and Pfizer Announce Longer-term Preliminary Data Showing Consistent and Sustained Factor IX Levels in Hemophilia B

December 15, 2017

Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here. Spark Therapeutics and Pfizer Inc. announced on Dec. 11, 2017, with a cumulative follow-up of more than 13 patient years of observation, all 11 participants in the ongoing Phase 1/2 clinical trial of investigational SPK-9001 for […]

Spark Therapeutics Presents Preliminary Data on Investigational SPK-8011 Phase 1/2 Dose-escalation Clinical Trial in Hemophilia A

December 15, 2017

Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here. Spark Therapeutics announced on Dec. 11, 2017, it has dosed seven participants in the Phase 1/2 clinical trial of SPK-8011 in hemophilia A. The first four participants, who have been followed at least 12 weeks post […]

Bioverativ Announces Results of Eloctate Study Showing High Bleed Protection and Target Joint Resolution

December 15, 2017

Note: The following is edited from a press release from Bioverativ. Read the full press release in its entirety here. Bioverativ Inc. and Swedish Orphan Biovitrum AB announced the results of a new, post-hoc, longitudinal analysis of the pivotal Phase 3 A-LONG study and ASPIRE long-term extension study, showing that weekly prophylactic dosing with its […]

Bioverativ Announces Findings From Novel Imaging Study Using Alprolix

December 15, 2017

Note: The following is edited from a press release from Bioverativ. Read the full press release in its entirety here. Bioverativ Inc., a global biopharmaceutical company dedicated to transforming the lives of people with rare blood disorders, today announced findings from a novel imaging study investigating extravascular distribution of factor IX therapies, including its leading extended […]

FDA Lifts Clinical Hold on Fitusiran

December 15, 2017

Note: The following is edited from a press release from Alnylam Pharmaceuticals. Read the full press release in its entirety here. Alnylam Pharmaceuticals Inc., a RNAi therapeutics company, and Sanofi Genzyme, the specialty care global business unit of Sanofi, announced Dec. 15, 2017, the U.S. Food and Drug Administration (FDA) has lifted the hold on clinical studies with fitusiran, including […]

Genentech Presents New Data on Hemlibra at ASH 2017

December 15, 2017

Note: The following is edited from a press release from Genentech. Read the full press release in its entirety here. Genentech, a member of the Roche Group, announced on Dec. 9, 2017, new data from the ongoing HEMLIBRA® (emicizumab-kxwh) clinical development program were presented at the 59th American Society of Hematology (ASH) Annual Meeting. These data […]

UniQure Announces New Data in Phase I/II Clinical Trial

December 12, 2016

______________________________________________________________________________ Note: The following is edited from a press release from UniQure. Read the full press release in it’s entirety here. UniQure announced new and updated results during the American Society of Hematology (ASH) meeting in relation to its ongoing, dose-ranging Phase I/II trial of AMT-060. AMT-060 is UniQure’s proprietary, investigational gene therapy for patients with severe […]

Sangamo Biosciences Announces Plans to File for New Investigational Drug

December 12, 2016

______________________________________________________________________________ Note: The following is edited from a press release from Sangamo Biosciences. Read the full press release in its entirety here. Sangamo Biosciences presented data during the recent American Society of Hematology (ASH) Annual Meeting regarding preclinical and manufacturing data of SB-525, its gene therapy program for hemophilia A. “We have developed an improved […]

Spark Therapeutics Shares Updated Phase I/II Clinical Trial Data

December 12, 2016

______________________________________________________________________________ Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in it’s entirety here.. Spark Therapeutics and Pfizer announced updated results from the Phase 1/2 study of SPK-9001, an investigational recombinant gene transfer product in hemophilia B, at the 58th American Society of Hematology (ASH) Annual Meeting, […]

Alnylam Presents Phase I Clinical Drug Trial Data at ASH 2016

December 12, 2016

  Note: The following is an excerpt from a press release from Alnylam Pharmaceuticals, Inc. regarding news from their recent presentation at the American Society of Hematology (ASH) Annual Meeting. The original article can be read in its entirety here. ______________________________________________________________________________ Alnylam Pharmaceuticals, Inc. presented data from the phase 1 study of fitusiran at the […]


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