UniQure gene therapy shows rise in blood-clotting protein levels

Drug developer UniQure NV said on Friday its gene therapy to treat hemophilia B increased the levels of a protein that helps in blood clotting after 12 weeks in a small study.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news

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BioMarin Pharmaceutical Inc said on Tuesday early trial data for its gene therapy for hemophilia A suggested the one-time infusion's effect on some patients' bleeding disorders would last eight years.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
A potential one-shot-and-you're-done hemophilia A treatment from BioMarin Pharmaceutical Inc. will target regulatory approval in the United States and Europe after showing dramatic decreases in bleeding during clinical trials. But in a competitive market to find the next big and expensive treatment to help — or even cure — hemophilia A patients, questions linger around how long the treatment sticks with patients. BioMarin (NASDAQ: BMRN) apparently didn't allay those concerns Tuesday: The San…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
Timing of Regulatory Submissions to Be Determined in 3Q 2019 Conference Call and Webcast to be Held Tuesday, May 28, 2019 at 8:00 AM Eastern SAN RAFAEL, Calif., May 28, 2019 -- (Healthcare Sales &Marketing Network) -- BioMarin Pharmaceutical Inc. (... Biopharmaceuticals BioMarin Pharmaceutical, valoctocogene roxaparvovec, hemophilia A, gene therapy
Source: HSMN NewsFeed - Category: Pharmaceuticals Source Type: news
Liver-directed gene therapy for the coagulation disorder hemophilia showed safe and effective results in clinical trials using adeno-associated viral vectors to replace a functional coagulation factor, although some unmet needs remain. Lentiviral vectors (LVs) may address some of these hurdles because of their potential for stable expression and the low prevalence of preexisting viral immunity in humans. However, systemic LV administration to hemophilic dogs was associated to mild acute toxicity and low efficacy at the administered doses. Here, exploiting intravital microscopy and LV surface engineering, we report a major ...
Source: Science Translational Medicine - Category: Biomedical Science Authors: Tags: Research Articles Source Type: research
Semin Thromb Hemost DOI: 10.1055/s-0039-1688445Hemophilia is a monogenic disease with robust clinicolaboratory correlations of severity. These attributes coupled with the availability of experimental animal models have made it an attractive model for gene therapy. The road from animal models to human clinical studies has heralded significant successes, but major issues concerning a previous immunity against adeno-associated virus and transgene optimization remain to be fully resolved. Despite significant advances in gene therapy application, many questions remain pertaining to its use in specific populations such as those ...
Source: Seminars in Thrombosis and Hemostasis - Category: Hematology Authors: Tags: Review Article Source Type: research
Reena Goswami1, Gayatri Subramanian2, Liliya Silayeva1, Isabelle Newkirk1, Deborah Doctor1, Karan Chawla2, Saurabh Chattopadhyay2, Dhyan Chandra3, Nageswararao Chilukuri1 and Venkaiah Betapudi1,4* 1Neuroscience Branch, Research Division, United States Army Medical Research Institute of Chemical Defense, Aberdeen, MD, United States 2Department of Medical Microbiology and Immunology, University of Toledo College of Medicine and Life Sciences, Toledo, OH, United States 3Roswell Park Comprehensive Cancer Center, Buffalo, NY, United States 4Department of Physiology and Biophysics, Case Western Reserve University, Clev...
Source: Frontiers in Oncology - Category: Cancer & Oncology Source Type: research
St. Jude is trying to establish a gene therapy program to bring next-level treatment options to patients in under-resourced countries.
Source: bizjournals.com Health Care:Physician Practices headlines - Category: American Health Authors: Source Type: news
The mainstay of hemophilia management has been the regular, prophylactic infusion of missing coagulation factors VIII/IX. This approach is limited by the need for frequent intravenous infusions, high cost, limited availability, and the development of inhibitory antibodies to factors VIII/IX. Numerous recent breakthroughs are addressing many of these limitations. These include the development of extended half-life factors that require less frequent infusions and the development of various novel agents that can be given subcutaneously and infrequently, including FVIII-mimetic antibody and downregulators of natural anticoagul...
Source: Hematology/Oncology Clinics of North America - Category: Cancer & Oncology Authors: Source Type: research
Condition:   Hemophilia B Intervention:   Biological: PF-06838435/ fidanacogene elaparvovec Sponsor:   Pfizer Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
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