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NHF 70th: Pursuing discovery of the next generation of therapies

An interview with one of the recipients of NHF's Innovative Investigator Awards

NHF 70th: Pursuing discovery of the next generation of therapies

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.

Spark Reports Progress on Hemophilia A Gene Therapy Candidate

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.

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