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CSL Behring Announces Closing of Global Commercialization and License Agreement with uniQure for etranacogene dezaparvovec

May 7, 2021

Read the full press release from CSL Behring here. Global biotherapeutics leader CSL Behring 聽announced the closing of its global Commercialization and License agreement with uniQure for etranacogene dezaparvovec, a novel gene therapy for the treatment of hemophilia B. Etranacogene dezaparvovec is currently in Phase 3 clinical trials and has been shown聽 to result in […]

uniQure Announces Closing of Commercialization and License Agreement with CSL Behring

May 6, 2021

Read the original press release here uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 for the global licensing agreement with CSL Behring for etranacogene dezaparvovec, an investigational gene therapy for patients with hemophilia B, expired […]

uniQure Announces FDA Removes Clinical Hold on Hemophilia B Gene Therapy Program

April 26, 2021

Read the full release here. uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on the Company鈥檚 hemophilia B gene therapy program after determining the Company satisfactorily addressed all issues identied by the FDA related […]

uniQure Releases Findings in Hemophilia B Gene Therapy Program

March 29, 2021

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 medical needs, announced the results of a comprehensive investigation into the case of hepatocellular carcinoma (HCC) diagnosed in one patient in the HOPE-B pivotal trial […]

uniQure Announces Clinical Hold on Hemophilia B Gene Therapy Program

December 21, 2020

The following is an excerpt from a press release from uniQure. Read the update in its entirety聽here. Today uniQure announced a clinical hold by the U.S. Food and Drug Administration (FDA) in its聽phase III HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients. Per uniQure鈥檚 statement: 鈥淭he clinical hold was initiated following […]

uniQure Presents Data from HOPE-B Pivotal Gene Therapy Trial in Patients with Hemophilia B at ASH Annual Meeting

December 10, 2020

The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure,聽a gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the late-breaking presentation of initial data from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five […]

Pfizer Doses First Participant In Phase 3 Study for People With Severe Hemophilia A and B with or Without Inhibitors

November 25, 2020

The following is from a press release from Pfizer. Read it in its entirety here. Pfizer Inc. announced that the first participant has been dosed in the Phase 3 BASIS study of marstacimab (PF-06741086), an anti-tissue factor pathway inhibitor (anti-TFPI) being evaluated for the treatment of people with severe hemophilia A or B, with or […]

uniQure Announces Positive Data from the HOPE-B Pivotal Trial Gene Therapy in Patients with Hemophilia B

November 25, 2020

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V.,聽a gene therapy company advancing transformative therapies for patients with severe medical needs, announced positive top-line data from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based […]

CSL Behring Announces Discontinuation of Mononine Coagulation Factor IX

September 15, 2020

CSL Behring issued the following statement to patient advocacy groups: “CSL Behring is focused on delivering breakthrough therapies to improve patients鈥 lives and meet the changing needs of our patients. Since CSL Behring鈥檚 introduction of MONONINE庐 Coagulation Factor IX (Human) in 1992, advancements have been made for the treatment of Hemophilia B.聽 Over time, patients […]

Novo Nordisk Resumes Clinical Trials Investigating Concizumab in Hemophilia A and B With or Without Inhibitors

August 20, 2020

The following is an excerpt from a press release from Novo Nordisk. Read the press release in its entirety here. Novo Nordisk announced the clinical trials in the concizumab phase 3 programme (explorer 6, 7 and 8) are being resumed. The clinical trials are investigating subcutaneous concizumab prophylaxis treatment in hemophilia A and B patients […]

A New Study of Hemophilia Occurrence Finds Many More Cases in the United States

July 9, 2020

The following is an announcement from the Centers for Disease Control and Prevention. Hemophilia is an inherited bleeding disorder in which the blood does not clot properly due to a lack or decrease in a protein called clotting factor. The two most common types of hemophilia are hemophilia A, which is due to a lack […]

Novo Nordisk Notice on COVID-19, Rebinyn庐 and Laboratory Assays

May 19, 2020

Novo Nordisk recently distributed a notice relevant to its product Rebinyn庐,聽raising awareness that one-stage clotting assay results can be significantly affected by the type of activated partial thromboplastin time (aPTT) reagent used, which can result in over or under-estimation of Factor IX activity. The notice which was addressed to HTCs, physicians and other healthcare providers, […]

uniQure Announces Highlights of Recent Progress

May 1, 2020

The following is an excerpt from a press release from uniQure. Read the release in its entirety here. uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, reported recent progress across its business. 鈥淒espite the ongoing global crisis caused by the coronavirus pandemic, we ended the first quarter of […]

HEMA Biologics Announces FDA Approval of SEVENFACT

April 6, 2020

The following is a press release from HEMA Biologics. HEMA BiologicsTM Announces FDA Approval of SEVENFACT庐 [coagulation factor VIIa (recombinant)-jncw] for Treatment and Control of Bleeding Episodes Occurring in Adult and Adolescent Hemophilia A and B Patients with Inhibitors Louisville, KY 鈥 April 6, 2020 HEMA Biologics, LLC, (鈥淗EMA Biologics鈥) today announced that the U.S. […]

uniQure Announces Achievement of Target Patient Dosing in HOPE-B Pivotal Trial

March 26, 2020

The following is a press release from uniQure. uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced it has achieved the targeted dosing of patients in the HOPE-B pivotal trial of etranacogene dezaparvovec (AMT-061), an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the […]

Aptevo Therapeutics Sells IXINITY Hemophilia B Therapy to Medexus Pharmaceuticals

March 6, 2020

The following is an excerpt from a press release from Aptevo Therapeutics. Read the press release in its entirety here. Aptevo Therapeutics Inc. , a biotechnology company focused on developing novel immuno-oncology therapeutics based on its proprietary ADAPTIR™ bispecific technology platform, announced it has completed the sale of its marketed recombinant factor IX therapeutic, IXINITY庐 […]

Infusing Love: Stand Up to Bullying

November 6, 2019

  Bullying has no shame. It shows no prejudice. Bullying affects anyone of any size, age, and ethnicity.聽 It affects all of us. When I watch grown people intentionally hurt each other with words and actions, it always surprises me. One day I asked a young lady why she was making fun of her coworker. […]

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 […]


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