Jeanne Lusher

Increasing Prevalence of Acquired Hemophilia Among New Borns Demands Effective Treatment - ResearchAndMarkets.com

Retrieved on: 
Thursday, July 22, 2021
Baby, Maternity, Health, Cardiology, Consumer, Haemophilia, Jeanne Lusher

The Acquired Hemophilia Treatment Market is anticipated to grow at a compound annual growth rate of 3.3% for the forecast period of 2021 to 2029.

Key Points: 
  • The Acquired Hemophilia Treatment Market is anticipated to grow at a compound annual growth rate of 3.3% for the forecast period of 2021 to 2029.
  • The key compelling factors of this market are increasing prevalence of Acquired Hemophilia in newborn, rising diagnostic rates, increasing use of Prophylactic treatment, rising awareness among people, rising number of auto-immune diseases and beneficial reimbursement policies.
  • Hemophilia A, is the most severe and commonly found type of Acquired Hemophilia.
  • On the basis of regional segmentation, Acquired Hemophilia Treatment Market includes North America, Latin America, Europe, Asia Pacific, Middle East and Africa, and Rest of the World.

uniQure Announces Positive 52-Week Clinical Data from HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B and Provides Regulatory Update

Retrieved on: 
Tuesday, June 22, 2021
Branches of biology, Life sciences, Biology, Applied genetics, Gene delivery, Biotechnology, Bioethics, Gene therapy, Therapy, Adeno-associated virus, Spark Therapeutics, Jeanne Lusher

and AMSTERDAM, June 22, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive 52-week clinical data on all patients from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec , an investigational adeno-associated virus five ( AAV5 )-based gene therapy for the treatment of patients with severe and moderately severe hemophilia B.

Key Points: 
  • and AMSTERDAM, June 22, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive 52-week clinical data on all patients from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec , an investigational adeno-associated virus five ( AAV5 )-based gene therapy for the treatment of patients with severe and moderately severe hemophilia B.
  • These are the first clinical data to be reported from a Phase III gene therapy study in hemophilia B and, with 54 patients, the largest set of hemophilia B patients receiving a single gene therapy investigational product to date.
  • All patients in the HOPE-B pivotal study achieved steady-state FIX activity levels by 26-weeks after administration of etranacogene dezaparvovec.
  • The pivotal Phase III HOPE-B trial is a multinational, open-label, single-arm study to evaluate the safety and efficacy of etranacogene dezaparvovec.

Enzyre announces strategic partnership with Takeda to accelerate development of pioneering diagnostic technology platform for hemophilia patients

Retrieved on: 
Thursday, March 4, 2021
Coagulation system, Blood, Anatomy, Body fluids, Coagulation, Factor VIII, Factor VII, Haemophilia, Factor V, Jeanne Lusher

Takeda confirmed the partnership by investing in Enzyre and joining its supervisory board.

Key Points: 
  • Takeda confirmed the partnership by investing in Enzyre and joining its supervisory board.
  • The Hemophilia Enzycard, the first product using Enzyre's proprietary Enzypad platform technology, can be used to determine the coagulation status of patients with hemophilia, a rare genetic disorder that affects an estimated 400,000 people worldwide.
  • [1] Those with hemophilia lack sufficient blood-clotting proteins, for example factor VIII (FVIII), causing them to bleed for a longer time after an injury compared to people without hemophilia.
  • Enzycard will enable patients to test in a home setting, immediately transferring coagulation status results to the patient's treating physician through a mobile phone app.

Enzyre announces strategic partnership with Takeda to accelerate development of pioneering diagnostic technology platform for hemophilia patients

Retrieved on: 
Thursday, March 4, 2021
Coagulation system, Blood, Anatomy, Body fluids, Coagulation, Factor VIII, Factor VII, Haemophilia, Factor V, Jeanne Lusher

Takeda confirmed the partnership by investing in Enzyre and joining its supervisory board.

Key Points: 
  • Takeda confirmed the partnership by investing in Enzyre and joining its supervisory board.
  • The Hemophilia Enzycard, the first product using Enzyre's proprietary Enzypad platform technology, can be used to determine the coagulation status of patients with hemophilia, a rare genetic disorder that affects an estimated 400,000 people worldwide.
  • [1] Those with hemophilia lack sufficient blood-clotting proteins, for example factor VIII (FVIII), causing them to bleed for a longer time after an injury compared to people without hemophilia.
  • Enzycard will enable patients to test in a home setting, immediately transferring coagulation status results to the patient's treating physician through a mobile phone app.

Spark Therapeutics Announces Preliminary Data from Phase 1/2 Clinical Trial of SPK-8016 in Hemophilia A at EAHAD 2021 Virtual Congress

Retrieved on: 
Friday, February 5, 2021
Branches of biology, Haemophilia, Biology, Life sciences, Hoffmann-La Roche, Spark Therapeutics, Virotherapy, Gene therapy, Jeanne Lusher

These data were presented at the European Association for Haemophilia and Allied Disorders (EAHAD) 2021 Virtual Congress by investigator Spencer Sullivan, M.D., Mississippi Center for Advanced Medicine.

Key Points: 
  • These data were presented at the European Association for Haemophilia and Allied Disorders (EAHAD) 2021 Virtual Congress by investigator Spencer Sullivan, M.D., Mississippi Center for Advanced Medicine.
  • All four participants who have hemophilia A and no history of FVIII inhibitors received a single intravenous administration of SPK-8016 at a dose of 5X1011 vg/kg.
  • InvestigationalSPK-8016 is a novel, internally developed gene therapy for hemophilia A to address the unmet medical need within the hemophilia A inhibitor patient community.
  • Pairing Roches long-standing commitment to developing medicines in hemophilia with Spark Therapeutics proven gene therapy expertise brings together the best team of collaborators researching gene therapies in hemophilia A.

uniQure Announces Clinical Update on Hemophilia B Gene Therapy Program

Retrieved on: 
Monday, December 21, 2020
Life sciences, Branches of biology, Biology, Biotechnology, Bioethics, Gene therapy, Molecular biology, Therapy, Jeanne Lusher

and AMSTERDAM, Dec. 21, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that its hemophilia B gene therapy program, including the pivotal, Phase III HOPE-B study, has been placed on clinical hold by the U.S. Food and Drug Administration (FDA).

Key Points: 
  • and AMSTERDAM, Dec. 21, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that its hemophilia B gene therapy program, including the pivotal, Phase III HOPE-B study, has been placed on clinical hold by the U.S. Food and Drug Administration (FDA).
  • Patient dosing is complete in each of uniQures three hemophilia B gene therapy studies, and there is no plan to enroll or treat additional patients.
  • We do not anticipate any impact to our regulatory submission timeline for the hemophilia B program as a result of this clinical hold.
  • uniQure holds multiple issued patents in the United States and Canada broadly covering methods of treating bleeding disorders, including hemophilia B, using AAV gene therapy with the FIX-Padua variant.

Gene Therapy for Hemophilia B Found Safe and Effective in First Phase III Trial

Retrieved on: 
Tuesday, December 8, 2020
Branches of biology, Blood, Coagulation system, Body fluids, Factor IX, Gene therapy, Coagulation, Haemophilia B, Prothrombin complex concentrate, Haemophilia, Jeanne Lusher, Spark Therapeutics

WASHINGTON, Dec. 8, 2020 /PRNewswire/ --The gene therapy etranacogene dezaparvovec substantially increased production of the blood clotting protein factor IX among 52 patients in the largest and most inclusive hemophilia B gene therapy trial to date.

Key Points: 
  • WASHINGTON, Dec. 8, 2020 /PRNewswire/ --The gene therapy etranacogene dezaparvovec substantially increased production of the blood clotting protein factor IX among 52 patients in the largest and most inclusive hemophilia B gene therapy trial to date.
  • A majority of trial participants (96%) successfully discontinued factor IX replacement therapy after receiving the gene therapy and have been producing their own factor IX for six months.
  • Hemophilia B, which accounts for about one-fifth of hemophilia cases, is caused by an inherited mutation of the gene for factor IX.
  • While several gene therapies for hemophilia have shown promise in early phase trials, the study is the first phase III trial to test the approach in a large and diverse array of patients, said Dr.

Takeda Presents Real-World Evidence at ASH 2020, Demonstrating Its Long-Standing Commitment to Personalizing Treatments for Rare Bleeding Disorders

Retrieved on: 
Monday, December 7, 2020
Biotechnology, Other Health, Health, Pharmaceutical, Oncology, Blood, Body fluids, Anatomy, Transfusion medicine, Von Willebrand disease, Hematology, Bleeding, Jeanne Lusher

Takeda Pharmaceutical Company Limited ( TSE: 4502/NYSE:TAK ) (Takeda), today presented five hematology poster presentations and four abstracts at the 62nd American Society of Hematology (ASH) Annual Meeting & Exposition that underscore its commitment to advancing treatments for rare bleeding disorders.

Key Points: 
  • Takeda Pharmaceutical Company Limited ( TSE: 4502/NYSE:TAK ) (Takeda), today presented five hematology poster presentations and four abstracts at the 62nd American Society of Hematology (ASH) Annual Meeting & Exposition that underscore its commitment to advancing treatments for rare bleeding disorders.
  • Real-world evidence (RWE) from studies across rare bleeding disorders demonstrate the crucial role understanding patterns of care and the patients holistic experience outside of rigorous clinical studies play in advancing patient-centric treatment in bleeding disorders.
  • Several of Takedas studies presented at ASH generate real-world evidence to better understand clinical management of treatment and associated disease outcomes in hemophilia A and VWD.
  • Patient preferences for hemophilia A treatments: A discrete choice experiment, (poster #1623) measured patients preferences for different hemophilia A treatments, including emerging treatments such as gene therapy.

Global Hemophilia Markets, 2020-2026: Focus on Recombinant Coagulation Factor Concentrates, Plasma-derived Coagulation Factor Concentrates and Others - ResearchAndMarkets.com

Retrieved on: 
Monday, November 30, 2020
Retail, Other Retail, Coagulation system, Body fluids, Anatomy, Haemophilia, Blood, Coagulation, Haemophilia B, Haemophilia A, Jeanne Lusher, Fresh frozen plasma

Global Hemophilia Market Size, Share & Trends Analysis Report By Type (Hemophilia A, Hemophilia B, and, Others), By Therapy (Replacement Therapy, Gene Therapy, and Others), By Product Type (Recombinant Coagulation Factor Concentrates, Plasma-derived Coagulation Factor Concentrates and Others) Forecast Period (2020-2026).

Key Points: 
  • Global Hemophilia Market Size, Share & Trends Analysis Report By Type (Hemophilia A, Hemophilia B, and, Others), By Therapy (Replacement Therapy, Gene Therapy, and Others), By Product Type (Recombinant Coagulation Factor Concentrates, Plasma-derived Coagulation Factor Concentrates and Others) Forecast Period (2020-2026).
  • The global hemophilia market is anticipated to grow at a CAGR of 6% during the forecast period.
  • Further, based on the product type, the market is subdivided into recombinant coagulation factor concentrates, plasma-derived coagulation, and other factor concentrates.
  • The global hemophilia market is further segmented based on geography including North America, Europe, Asia-Pacific, and the Rest of the World.

Medexus Reaches 50% Enrollment in IXINITY® Phase 4 Clinical Trial Targeting Label Expansion for Pediatric Hemophilia B Patients

Retrieved on: 
Monday, November 16, 2020
Health sciences, Health, Clinical research, Drug discovery, Pharmaceutical industry, Clinical trial, Design of experiments, Jeanne Lusher

IXINITY is an FDA approved intravenous recombinant factor IX therapeutic for use in patients 12 years of age or older with hemophilia B.

Key Points: 
  • IXINITY is an FDA approved intravenous recombinant factor IX therapeutic for use in patients 12 years of age or older with hemophilia B.
  • In January 2020, Aptevo had already commenced dosing patients in a Phase 4 clinical trial to evaluate the safety and efficacy of IXINITY in previously treated patients under 12 years of age with hemophilia B.
  • Since the pediatric segment is estimated to represent one-third of the hemophilia B population, a label expansion represents a possible expansion of the potential for IXINITY.
  • We remain determined to advance our IXINITY Phase 4 pediatric clinical trial and look forward to providing further updates as we achieve key milestones.