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uniQure Announces Follow-Up Data from Study in Patients with Hemophilia B

July 10, 2019

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure, a gene therapy company advancing transformative therapies for patients with severe medical needs, announced updated clinical data on the three patients treated in the company鈥檚 ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene […]

Infusing Love: Tattoos

June 26, 2019

  Our son, Michael, recently surprised us with the news that he was going to get a tattoo! I know, I know, he is 26 years old and can make his own decisions.聽But he still has severe hemophilia B with inhibitors. I got that knot in my stomach that all moms get 鈥 it鈥檚 a […]

uniQure Presents Updated Clinical Data on Gene Therapy for Hemophilia B

May 10, 2019

The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure聽announced updated clinical data in patients treated in the company鈥檚 ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene therapy containing a patent-protected FIX-Padua variant, for the treatment of patients with severe and moderately severe hemophilia B. […]

Sanofi Genzyme Launches Innovative and Educational Audio Adventure Series for People Treated with ALPROLIX庐

March 15, 2019

The following is a press release from Sanofi Genzyme. Find the full press release here. Sanofi Genzyme, the specialty care business unit of Sanofi, announced the launch of a first-of-its-kind audio adventure series designed to provide an engaging and educational infusion experience for ALPROLIX庐 [Coagulation Factor IX (Recombinant), Fc Fusion Protein] patients and their caregivers. […]

uniQure Announces Updated Clinical Data from Phase IIb Study in Patients with Hemophilia B

February 8, 2019

The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure,聽a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced updated clinical data in patients treated in the company鈥檚 ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene therapy containing a […]

uniQure Announces First Patient Treated in HOPE-B Pivotal Trial of AMT-061 in Patients with Hemophilia B

February 6, 2019

The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure, a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced that it treated the first patient in its HOPE-B pivotal trial of AMT-061, an investigational AAV5-based gene therapy incorporating the […]

Sangamo Announces Treatment Of First Patient In Clinical Trial Of In Vivo Genome Editing Therapy For Hemophilia B

December 20, 2018

The following is an excerpt from a press release from Sangamo. Read the press release in its entirety here. Hemophilia B is a rare, genetic bleeding disorder caused by a lack of the factor IX (FIX) protein, which is necessary for normal blood clotting. Current hemophilia B treatments typically require frequent intravenous infusions of clotting […]

uniQure Announces Long-Term Clinical Data from Ongoing Phases and Confirms Dose for Hemophilia B Study

December 5, 2018

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, announced updated results聽from its ongoing Phase I/II trial of AMT-060, and provided an update on AMT-061, the company鈥檚 next-generation gene therapy聽candidate […]

Update on uniQure’s Hemophilia B Gene Therapy Program

November 28, 2018

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure announces the initial topline data from our Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B. All patients achieved and sustained therapeutic […]

uniQure Announces First Patient Treated in Dose-Confirmation Study in Patients with Hemophilia B

November 19, 2018

The following is an excerpt from a press release from uniQure. uniQure announced聽that they have treated the first patient in our Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B (see attached press release). 鈥淎s a one-time administered therapy, AMT-061 has the […]

uniQure Announces First Patient Treated in Dose-Confirmation Study in Patients with Hemophilia B

August 24, 2018

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here.聽 uniQure, a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced it has treated the first patient in its Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy […]

Catalyst Biosciences releases update on trial of Factor IX treatment of severe hemophilia B

June 19, 2018

The following is a summary of a press release from Catalyst Biosciences by Dr. Robert Sidonio, a trusted medical advisor. Read the full press release here. On June 18, Catalyst Biosciences provided an update of the progress from its Phase 陆 trial investigating the use of FIX variant product, called CB 2679d/ISU304, which is a […]

Novo Nordisk Launches Rebinyn in the United States for People with Hemophilia B

February 21, 2018

Note: The following is an excerpt from a press release from Novo Nordisk. Read the full press release here.聽 Novo Nordisk, a global healthcare company, today announced that Rebinyn, Coagulation Factor IX (Recombinant), GlycoPEGylated, is now available in the United States for the treatment of hemophilia B. Rebinyn聽is an extended half-life injectable medicine used to […]

Breakthrough in Hemophilia Gene Therapy

January 31, 2018

The following is an excerpt from an article in Medical News Bulletin. Read the full article here. Researchers at Spark Therapeutics and Pfizer tested the safety and efficacy of a new hemophilia gene therapy vector for the treatment of hemophilia B. The body鈥檚 ability to stop bleeding and repair damaged blood vessels depends heavily upon […]

Catalyst Biosciences Initiates Phase 2 of Potential Hemophilia A and B Therapy

January 5, 2018

Note:聽The following is聽an excerpt聽from an article by Rare Disease Report. Read the full聽article in its entirety聽here. On Jan. 4, Catalyst Biosciences announced the initiation and open enrollment of the Phase 2 part of its Phase 2/3 program of marzeptacog alfa (activated) (MarzAA), a highly potent, subcutaneously administered Factor VIIa therapy in development for the treatment […]

uniQure Announces Hemophilia B Gene Therapy Program To Enter Pivotal Study

October 19, 2017

Note: The followed is excerpted from a press release from uniQure.聽Read uniQure’s full press release HERE.聽 uniQure announced today聽plans to advance their development program for hemophilia B gene therapy into a pivotal study using an enhanced version of their聽product candidate called AMT-061. AMT-061 is identical to AMT-060 in every way apart from a change from […]

Update 2: Bioverativ Files Complaints Against CSL Behring’s Idelvion

July 19, 2017

As noted in our July 10, 2017 story, Bioverativ has filed complaints in the U.S. District Court for the District of Delaware and with the U.S. International Trade Commission alleging that Idelvion, a novel factor IX albumin fusion protein developed by CSL Behring, infringes upon three Bioverativ patents. HFA, in collaboration with the National Hemophilia […]

Spark Therapeutics Presents Updated Interim Hemophilia B Data Supporting Consistent & Sustained Response at ISTH 2017

July 11, 2017

Note: The following is an edited version of a press release from Spark Therapeutics.聽Read the press release from Spark Therapeutics聽in it鈥檚 entirety here. Spark Therapeutics recently announced that 10 participants in its ongoing Phase 1/2 clinical trial of聽SPK-9001 for hemophilia B, as of the June 5, 2017 data cut off, had their AIR reduced approximately […]

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

July 11, 2017

Note: The following is an edited version of a press release from uniQure.聽Read the press release from uniQure聽in it鈥檚 entirety here. uniQure N.V.聽has聽announced updated results聽from its ongoing, dose-ranging Phase I/II trial of AMT-060, its proprietary, investigational gene therapy聽in patients with severe hemophilia B. The data includes up to 18 months of follow-up from the low-dose […]

Bioverativ Files Complaints Against CSL Behring’s Idelvion

July 10, 2017

Note: This story has been sourced from the website, www.thepharmaletter.com聽and The Australian Business Review. On Friday, July 7, 2017, Bioverativ filed complaints in the U.S. District Court for the District of Delaware and with the U.S. International Trade Commission alleging that Idelvion, a novel factor IX albumin fusion protein developed by CSL Behring, infringes upon […]

FDA Approves New Novo Nordisk Treatment for Patients with Hemophilia B

June 1, 2017

  Novo Nordisk recently announced that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application for REBINYN庐 (Coagulation Factor IX (Recombinant), GlycoPEGylated) for the treatment of adults and children with hemophilia B.聽Novo Nordisk expects to launch REBINYN庐 in the U.S. in the first half of 2018. For more info on REBINYN, […]

uniQure Receives EU PRIME Designation For Hemophilia B Product

May 2, 2017

聽uniQure recently received the designation of a “priority medicine” from the European Union for a potential gene therapy treatment for Hemophilia B. From the press release: uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that AMT-060, its proprietary, investigational gene therapy聽in patients with severe […]

Catalyst Biosciences Completes New Hemophilia B Drug Toxicology Study

April 14, 2017

On April 11, Catalyst Biosciences, a clinical-stage biopharmaceutical company focused on developing novel medicines to address hematology indications, reached a key milestone towards starting human trials with聽completion of the CB 2679d/ISU304 toxicology studies. Their press release can be read in full聽here.

Aptevo’s Ixinity Found To Be Safe In Children Under 12 With Hemophilia B

April 14, 2017

Aptevo’s Ixinity, previously approved for use in people over the age of 12, just received approval for use in children under 12, per their ongoing phase 3 trial. Read the full press release here.

Novo Nordisk Announces New Data on Psychosocial Impact of People Living with Hemophilia B

July 26, 2016

Note: The below is an edited version of a press release by Novo Nordisk. The original release can be read in it’s entirety聽here.聽 Novo Nordisk today announced the first results from the Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S) study, which found that many adults and children living with even mild or […]

Spark Therapeutics and Pfizer Announce Receipt of FDA Breakthrough Therapy Designation for SPK-9001 for the Treatment of Hemophilia B

July 21, 2016

Note: The below is an edited version of a press release by Spark Therapeutics.聽The original release can be read in it’s entirety聽here. Spark Therapeutics and Pfizer Inc. announced today that the聽U.S. Food and Drug Administration聽(FDA) has granted breakthrough therapy designation to聽SPK-9001,聽the lead investigational candidate in the companies’聽SPK-FIX聽program, in development for the treatment of hemophilia B.聽SPK-9001,聽a […]

uniQure Announces Data from Phase I/II AMT-060 Clinical Trial

June 28, 2016

Note: The below is an edited version of a press release by UniQure.聽The original release can be read in it’s entirety聽here. On June 11, 2016,聽uniQure N.V. announced that additional data from its Phase I/II clinical trial of AMT-060 in hemophilia B patients were presented at the 21st聽Congress of the European Hematology Association (EHA) in Copenhagen, […]

Spark Therapeutics Announces Updated Data from Hemophilia B Trial

June 14, 2016

Note: The below is an edited version of a press release by Spark Therapeutics.聽The original release can be read in it’s entirety聽here. PHILADELPHIA,聽June 13, 2016 –聽Spark Therapeutics announced today updated results of the first cohort from the ongoing Phase 1/2 clinical trial of聽SPK-9001, the lead investigational candidate in its聽SPK-FIX聽program, which is being studied for the […]

Trial for SubQ Hemophilia Inhibitor Treatment Begins

March 14, 2016

Note: The below is an edited press release from Alnylam Pharmaceuticals, Inc. The original release can be read in its entirety聽here.聽 Alnylam Pharmaceuticals, Inc. announced on Monday, March 14, 2016, that it has initiated dosing of hemophilia patients with inhibitors in Part D of an ongoing Phase 1 clinical trial evaluating a once-monthly subcutaneous dose […]

FDA Approves First 14-Day Hemophilia B Treatment

March 5, 2016

Note: The following is an edited form of a press release from the FDA . The original form of the release can be read聽here. The U.S. Food and Drug Administration approved on Friday, March 4, Idelvion, Coagulation Factor IX (Recombinant), Albumin Fusion Protein, for use in children and adults with Hemophilia B. Idelvion is the […]


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