ASPIRE Gene Therapy for Hemophilia Landing Page

Pfizer Rare Disease announces the 2017 ASPIRE GENE THERAPY FOR HEMOPHILIA competitive grant research awards program

Background

The 2017 Advancing Science through Pfizer: Investigator Research Exchange (ASPIRE) Gene Therapy for Hemophilia Research Awards Program is a competitive grants program that reflects the commitment of Pfizer Hemophilia Rare Disease group to support ongoing basic science and clinical investigation in gene therapy for hemophilia.

Ongoing basic science research and clinical research are critical to deepen understanding of disease mechanisms and to ensure advancement of management strategies for hemophilia and related comorbidities. In an era of increased competition for research funding, the 2017 ASPIRE Gene Therapy in Hemophilia Research Awards Program is designed to support laboratory and clinical research in pathogenesis, complications, management, and clinical outcomes of gene therapy for hemophilia.

Mission

To support research through a competitive grants program that advances medical knowledge in the area of Gene Therapy for Hemophilia.  The 2017 program will focus on Hemophilia B.

Areas of Research Focus

  1. Patients with Mild Hemophilia B
    • Natural history of disease
    • Arthropathy: presence , development, clinical burden & Joint damage
    • Quality of Life/Work analysis
    • Clinical profile & healthcare utilization
    • Comorbidities: prevalence, severity, management
    • Cost of Care, including non-hemophilia related healthcare utilization
  2. Basic science of Adeno-associated virus 8 (AAV8)
    • Basic science, tropism, transduction efficiency & tolerability
    • AAV antibody titer assessment, reduction, tolerance
    • Role of immunosuppression in managing transaminitis & expression levels
       

Available Awards

Selection of research proposals will be performed by an independent, external expert panel comprised of nationally known academic clinicians. Project duration should be 1-2 years and should be approximately $125,000/year, inclusive of overhead costs (capped at 28%). It is anticipated 3 awards will be made.