Hemophilia Federation of America is a national nonprofit organization that assists, educates and advocates for the bleeding disorders community.
HFA does not recommend, endorse or make any representation about the efficacy, appropriateness or suitability of any specific products, treatments, or opinions. Please consult your physician before use of any treatments. We recently had Jackson鈥檚 six-month comprehensive visit at the hematologist. A comprehensive visit is an appointment in which many bleeding disorders specialists meet with […]
Featured Story: Federal court rules that the ACA is unconstitutional: Texas v. Azar On Friday, Dec. 14, we learned a federal judge had ruled in the case of Texas v. Azar聽that the Affordable Care Act is unconstitutional in its entirety. This news has alarmed many. HFA staff posted a number of updates to tell the […]
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 […]
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 […]
The following is a press release from uniQure. uniQure, a gene therapy company advancing transformative therapies for patients with severe medical needs, announced the expansion of its research pipeline with novel AAV gene therapy approaches to treating Hemophilia A, Fabry disease and Spinocerebellar Ataxia Type 3 at the Company鈥檚 Research & Development Day held this […]
The field of gene therapy is a hot topic of discussion. With multiple clinical trials ongoing, people with hemophilia are at the forefront of gene therapy research. Has anyone ever asked you about your thoughts and opinions about gene therapy? Here鈥檚 your chance to make them known! Hemophilia Federation of America Research is asking people […]
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 […]
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. […]
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