We’re the leading resource for those affected by bleeding disorders. You can count on our organization to ensure your voice is heard. It’s with your help that we can make a difference. Learn more and get involved.
-
Our programs are designed to provide relevant information to support people with bleeding disorders and their families with their ability to make informed choices.
Take a look. -
Fundraisers and events help us raise awareness and generate crucial resources in support of the bleeding disorders community. Join us at our next event and see how you can get involved. Find something to do.
-
Our grassroots network is an integral part of our larger community. Team up with us and see what a huge difference a little bit of your time can make.
Take action.
News & Events
An interview with one of the recipients of NHF's Innovative Investigator Awards
An interview with one of the recipients of NHF's Innovative Investigator Awards
Sangamo Therapeutics, Inc. recently announced positive preliminary data from the Phase 1/2 clinical trial designed to evaluate SB-525, the company’s gene therapy program for people with hemophilia A, or factor VIII (FVIII) deficiency. SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer for the development and commercialization of hemophilia A gene therapy programs.
The therapy is administered via a one-time intravenous infusion, which is designed to elicit the production of therapeutic levels of factor VIII.
Spark Therapeutics recently reported preliminary phase 1/2 data for SPK-8011, the company’s investigational gene therapy candidate for hemophilia A. The therapy is administered via a one-time intravenous infusion, which is designed to elicit the production of therapeutic levels of factor VIII (FVIII), a protein that is normally deficient in individuals with hemophilia A. Spark Therapeutics’ proprietary bioengineered adeno-associated viruses (AAVs) act as delivery vehicles, or vectors, to carry the genetic codes that prompt the FVIII production.
View Archives
