Hemophilia Federation of America is a national nonprofit organization that assists, educates and advocates for the bleeding disorders community.
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 […]
BioMarin is pleased to update the community regarding our gene therapy clinical trial program in hemophilia A. BioMarin’s investigational gene therapy for hemophilia A has not been approved for use; it is in ongoing clinical trials evaluating its safety and efficacy. Clinical Trial Overview BioMarin’s valoctocogene roxaparvovec, is currently being studied in adults with severe […]
The following is an excerpt from a press release from uniQure. Read the update in its entirety here. Today uniQure announced a clinical hold by the U.S. Food and Drug Administration (FDA) in its phase III HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients. Per uniQure’s statement: “The clinical hold was initiated following […]
The following is an excerpt from a press release from Pfizer. Read the full press release here. Pfizer Inc. and Sangamo Therapeutics Inc., a genomic medicines company, announced updated follow-up data from the Phase 1/2 Alta study of giroctocogene fitelparvovec (SB-525 or PF-07055480), an investigational gene therapy for patients with severe hemophilia A. These data […]
On October 30th, 2020, the Institute for Clinical and Economic Review (ICER) held a public meeting to discuss its evidence report on therapies for hemophilia A. ICER is an independent and non-partisan research organization that evaluates the clinical and economic value of prescription drugs, medical tests, and other healthcare and healthcare delivery innovations. For this […]
The following is an excerpt from an update from Takeda. Read the update in its entirety here. Takeda recently informed HFA of it’s decision to suspend screening and further enrollment in the TAK-754 and TAK-748 clinical studies, effective immediately. TAK-754 is an AAV8 gene therapy for the treatment of hemophilia A currently in Phase 1/2 clinical development, while […]
The following is information from the World Federation of Hemophilia. A joint World Federation of Hemophilia (WFH) and National Hemophilia Foundation (NHF) statement* is issued on two deaths reported in orphan disease gene therapy clinical trial (*on behalf of the WFH Coagulation Product Safety, Supply, and Access Committee): We have become aware of two […]
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 that uniQure and CSL Behring have entered into a licensing agreement providing CSL Behring with exclusive global rights to etranacogene […]
The following is an excerpt from a press release from Pfizer. Read the press release in its entirety here. Pfizer Inc. and Sangamo Therapeutics Inc. announced updated follow-up data from the Phase 1/2 Alta study of giroctocogene fitelparvovec (SB-525, or PF-07055480), an investigational gene therapy for patients with severe hemophilia A. All five patients with […]
The following is an update on the Spark and Roche gene therapy program. “Together, Spark and Roche remain fully committed in our objective to meet the needs of the hemophilia community by developing gene therapies for hemophilia A that, with the lowest effective dose and optimal regimen to manage the immune system response, demonstrate safety, […]
By Najat Bouchkouj, M.D., Bindu George, M.D., and Karen Jackler, MPH A person with hemophilia can experience aspects of their disease that even the most qualified doctors and scientists can’t fully appreciate. This is why the Center for Biologics Evaluation and Research at the Food and Drug Administration is working to hear and understand the […]
Click Below to Translate Page to Spanish. HFA and NHF co-hosted a Safety Summit in Washington, D.C., over three days at the end of January. The summit was convened in recognition and response to community concerns arising from a series of drug safety reports in 2019. Those safety incidents rocked the hemophilia community – and […]
The following is an announcement from BioMarin. BioMarin announced an update to the community regarding our ongoing gene therapy clinical trial program in hemophilia A. BioMarin’s investigational gene therapy for hemophilia A has not been approved for use; it is in ongoing clinical trials evaluating its safety and efficacy. Clinical Trial Overview BioMarin’s investigational gene therapy valoctocogene roxaparvovec […]
The following is an excerpt from a press release from Sangamo. Read the press release in its entirety here. Sangamo Therapeutics Inc., a genomic medicine company, and Pfizer Inc. announced updated follow-up results from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy in patients with severe hemophilia A. The data showed that SB-525 […]
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’s ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene […]
The following is an excerpt from a press release from Sangamo. Read the press release in its entirety here. Sangamo Therapeutics Inc., a genomic medicine company, and Pfizer Inc. announced updated results from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy for severe hemophilia A. The data showed that SB-525 was generally well-tolerated […]
The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure announced updated clinical data in patients treated in the company’s 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. […]
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, presented new preclinical data on its gene therapy candidates for the treatment of hemophilia A and for the treatment of Fabry disease during back-to-back oral presentations at the 22nd American Society […]
The following is an excerpt from a press release from Spark Therapeutics. Read the press release in its entirety here. Spark Therapeutics , a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, announced today that it has entered into a definitive merger agreement for Roche to fully acquire Spark […]
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’s 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 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 from its ongoing Phase I/II trial of AMT-060, and provided an update on AMT-061, the company’s next-generation gene therapy 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’s 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’s 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’s ability to stop bleeding and repair damaged blood vessels depends heavily upon […]
Note: The followed is excerpted from a press release from uniQure. Read uniQure’s full press release HERE. uniQure announced today plans to advance their development program for hemophilia B gene therapy into a pivotal study using an enhanced version of their 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. Read the press release from Spark Therapeutics in it’s entirety here. The company has disclosed preliminary initial data from the Phase 1/2 dose-escalation clinical trial for SPK-8011, 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. Read the press release from Spark Therapeutics in it’s entirety here. Spark Therapeutics recently announced that 10 participants in its ongoing Phase 1/2 clinical trial of 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. Read the press release from BioMarin in it’s 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 presented […]
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