Hemophilia A (Severe) - Healing Genes
post-template-default,single,single-post,postid-1297,single-format-standard,mkdf-bmi-calculator-1.0,mkd-core-1.1.1,tribe-no-js,wellspring-ver-1.8,mkdf-smooth-page-transitions,mkdf-ajax,mkdf-grid-1300,mkdf-blog-installed,mkdf-header-standard,mkdf-no-behavior,mkdf-default-mobile-header,mkdf-sticky-up-mobile-header,mkdf-dropdown-slide-from-bottom,mkdf-full-width-wide-menu,mkdf-search-dropdown,elementor-default,elementor-template-full-width,elementor-kit-3486,elementor-page elementor-page-1297,elementor-page-2540

Hemophilia A (Severe)

Share This Post

Share on facebook
Share on linkedin
Share on twitter
Share on email

Dose-Ranging Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A

A Phase 1/2, Open-Label, Adaptive, Dose-Ranging Study to Assess the Safety and Tolerability of SB-525 (Recombinant AAV2/6 Human Factor 8 Gene Therapy) in Adult Subjects With Severe Hemophilia A

Phase 1 / 2

Doctors at five research hospitals seek male patients with severe Hemophilia A (with Factor VIII Inhibitors) to trial a new product, SB-525, an investigatory gene therapy. This therapy utilizes a harmless virus to deliver a functional copy of the factor VIII (F8) gene to the liver, which may permanently provide sufficient blood coagulation and reduce the need for transfusions.

The treatment requires a single intravenous injection of SB-525 and follow up will involve lab assessments and check-ins over the following year and for up to 3 years.


  • Be male and 18 years or older
  • Have congenital severe or moderately severe hemophilia A
  • Not have participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational product within the last 12 weeks


  1. Screening before the treatment
  2. A single infusion of the gene therapy drug will be administered
  3. Monitoring of the patient for a few hours after dosing, with periodic lab assessments required for 3 years.

Trials will take place at 13 locations across the United States. See the list of locations.
Contact: Medical Monitor  |  510.307.7266  |  [email protected]
Sangamo Therapeutics
Or go online:

Subscribe To Our Newsletter

Get updates and learn from the best

More To Explore

Vanquishing the Virus: 160+ COVID-19 Drug and Vaccine Candidates in Development

While President Trump continues to promote antimalarial drugs such as hydroxychloroquine sulfate and azithromycin to treat COVID-19—“What do you have to lose?” he exclaimed during the April 4 Coronavirus Task Force Briefing—the global community of drug discovery researchers and biopharmas is developing a much broader