Hemophilia Federation of America is a national nonprofit organization that assists, educates and advocates for the bleeding disorders community.
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 […]
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. The company has disclosed preliminary initial data from the Phase 1/2 dose-escalation clinical trial for SPK-8011,聽a novel bio-engineered adeno-associated viral (AAV) vector utilizing the Spark200 capsid and containing a codon-optimized human factor […]
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 […]
Note: The following is an edited version of a press release from BioMarin.聽Read the press release from BioMarin聽in it鈥檚 entirety here. BioMarin Pharmaceutical Inc. announced an update to its previously reported interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. The updated results were聽presented […]
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 […]
Note: The following is an edited version of a press release from Shire.聽Read the press release from Shire聽in it鈥檚 entirety聽here. Shire has announced聽the submission of an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for SHP654, also designated as BAX 888, an investigational factor VIII (FVIII) gene therapy for the […]
Dimension Therapeutics聽announced it will聽discontinue the development of DTX101, an investigational AAVrh10-based gene therapy product in development for the treatment of moderate/severe-to-severe hemophilia B. The decision followed the review of the emerging DTX101 Phase 1/2 clinical study data, including the data as of the beginning of聽May 2017, and the observation that the data would not meet […]
Sangamo Therapeutics, Inc. and Pfizer Inc. recently聽announced an exclusive, global collaboration and license agreement for the development and commercialization of gene therapy programs for Hemophilia A, including SB-525, one of Sangamo’s four lead product candidates, which Sangamo expects will enter the clinic this quarter. Click here to read the full press release from Sangamo Therapeutics.
uniQure recently presented data at the American Society of Gene and Cell Therapy (ASGCT) 20th聽Annual Meeting in Washington, D.C.聽on successful repeated hepatic gene delivery with their聽AAV5 vector following a proprietary immunoadsorption procedure in non-human primates (NHPs). The results described successful sequential transduction with the two reporter transgenes and highlighted the progress made in readministration protocols […]
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 […]
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聽here. Spark Therapeutics has announced that its Phase 1/2 trial for the investigation of gene therapy for hemophilia A (factor聽VIII deficiency) has begun. This trial will initially be enrolling participants at the Children鈥檚 Hospital […]
______________________________________________________________________________ Note: The following is edited from a press release from UniQure. Read the full press release in it鈥檚 entirety聽here. UniQure announced that AMT-060, 聽its proprietary, investigational gene therapy聽in patients with severe hemophilia B, has received Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA).聽 This designation is based on results from the […]
______________________________________________________________________________ Note: The following is edited from a press release from UniQure. Read the full press release in it鈥檚 entirety here. UniQure announced new and updated results during the American Society of Hematology (ASH) meeting聽in relation to its ongoing, dose-ranging Phase I/II trial of AMT-060. AMT-060 is UniQure鈥檚 proprietary, investigational gene therapy聽for patients with severe […]
______________________________________________________________________________ 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 […]
______________________________________________________________________________ Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in it鈥檚 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, […]
The following is excerpted from a press release from PRNewswire. Read the press release in it’s entirety聽here. Sangamo BioSciences, Inc.聽announced earlier this week that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SB-FIX, the company’s zinc finger nuclease (ZFN)-mediated genome editing product candidate for the treatment of hemophilia B. Sangamo […]
Note: The below is an edited version of a press release issued by BioMarin Pharmaceutical Inc. The original release can be read in its entirety聽here. BioMarin Pharmaceutical announced today positive interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. Nine patients with severe hemophilia […]
Note: The below is an edited version of an article originally published on PR Newswire. The original release can be read in its聽entirety聽here. Sangamo BioSciences, Inc. announced the presentation of preclinical data that supports the clinical development of its new proprietary gene therapy for the treatment of hemophilia A. This new therapeutic comprises an adeno-associated […]
Note: The below is an edited version of an article originally published on Globe Newswire. The original release can be read in its entirety聽here. uniQure N.V. yesterday announced updated clinical data from its ongoing Phase I/II trial of AMT-060, its proprietary, investigational gene therapy, in patients with severe hemophilia B.聽 The updated data show that […]
Note: The below is an edited version of a press release by BioMarin Pharmaceutical Inc. The original release can be read in its entirety here. BioMarin Pharmaceutical Inc. announced on Tuesday, March 1, that BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A, has been granted orphan drug designation by […]
Note: The following is an edited form of a press release from the University of North Carolina School of Medicine. The original release can be read聽here. A single injection. That鈥檚 all someone with a factor VII deficiency would need for a life-long cure, thanks to a new gene therapy treatment developed in a collaboration of […]
In our family, we are open to participating in research, thinking, 鈥渋f it can help our kids or help a future family or patient then we are all for it.鈥 So when the opportunity arose to be enrolled in a trail to test long lasting factor, we jumped on it. We did our homework and […]
This post first appeared on聽Vector,聽a blog of Boston Children’s Hospital an was written by聽Ellis Neufeld, MD, PhD,聽a hematologist at Dana-Farber/Boston Children鈥檚 Cancer and Blood Disorders Center. 聽 ________________________________________________ From new longer-acting drugs to promising gene therapy trials, much is changing in the treatment of聽hemophilia, the inherited bleeding disorder in which the blood does not clot. […]
Research showed that bleeding events were drastically decreased in animals with Hemophilia B. Using a viral vector to swap out faulty genes proved safe and could be used for the more common Hemophilia A. _______________________________ A multi-year, ongoing study suggests that a new kind of gene therapy for Hemophilia B could be safe and effective […]
Biogen Idec, Fondazione Telethon, and Ospedale San Raffaele have entered into a worldwide collaboration to jointly develop gene therapies for the treatment of both hemophilia A and B. The agreement will combine聽San Raffaele – Telethon Institute for Gene Therapy鈥檚 (TIGET)聽extensive expertise in creating new gene therapy strategies and developing them from the bench to bedside […]
Pfizer Inc. announced on Monday, December 8, 2014 聽two strategic decisions to expand the company鈥檚 rare disease research and development activities through the establishment of a gene therapy platform to investigate potential treatments for patients. First is an agreement with Spark Therapeutics to develop SPK-FIX, a program incorporating a bio-engineered AAV vector for the potential […]
On November 19, St. Jude Children’s Research Hospital announced the results of their ongoing gene therapy partnership with University College London and the Royal Free Hospital. The report indicates success in the trial, which used a modified adeno associated virus (AAV) 8 to deliver genetic material for making Factor IX in Hemophilia B patients. In […]
[glossary_exclude] A technique developed by Stanford researchers could provide a safer, longer-lasting method of replacing faulty, disease-causing genes with working copies. The ability to pop a working copy of a faulty gene into a patient鈥檚 genome is a tantalizing goal for many clinicians treating genetic diseases. Now, researchers at the Stanford University School of Medicine […]
Dimension Therapeutics, a Cambridge biotechnology startup, has struck a deal worth up to $252 million with the pharmaceutical giant Bayer HealthCare to develop a gene therapy to treat hemophilia, a rare disease that prevents blood from clotting. The goal of the prospective treatment, which is not yet in clinical trials, is to repair a faulty […]
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