ASPIRE Gene Therapy for Hemophilia Landing Page

2018 Awards in Gene and anti-TFPI Therapies For Hemophilia Global

Background

The 2018 Advancing Science through Pfizer: Investigator Research Exchange (ASPIRE) Gene and anti-TFPI Therapies for Hemophilia Global 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 and anti-TFPI therapies 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 2018 ASPIRE Gene and anti-TFPI Therapies in Hemophilia Global Research Awards Program is designed to support laboratory and clinical research in pathogenesis, complications, management, and clinical outcomes of these therapies for hemophilia.

Mission

To support research through a competitive grants program that advances medical knowledge in the area of Gene Therapy and anti-TFPI Therapy for Hemophilia.

Areas of Research Focus

  1. Basic Science of Gene Therapy for Hemophilia
    • Basic science, tropism, transduction efficiency & tolerability of Adeno-associated virus
    • AAV antibody Titer assessment, reduction, tolerance
    • Role of immunosuppression in managing transaminitis
  2. Basic Science of TFPI & Anti-TFPI Monoclonal Antibodies
    • Regulation of Coagulation
    • Basic biology of TFPI interactions with Protein C, ATIII, & Protein S
    • Cross talk among regulators (e.g. Protein S being a co-factor for both Protein C and TFPI)
    • Role of different TFPI pools in regulation of coagulation
    • Pharmacology resulting from concomitant treatments (especially antifibrinolytics) added to anti-TFPI
  3. Patients with MILD Hemophilia A or B
    • Natural history of mild Hemophilia
    • Arthropathy: presence, development, clinical burden & Joint damage in mild Hemophilia
    • Quality of Life/Work analysis in mild Hemophilia
    • Clinical profile & healthcare utilization in mild Hemophilia
    • Cost of Care, including non-hemophilia related healthcare utilization in mild Hemophilia

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 2 to 3 awards will be made.