2022 Global Hemophilia ASPIRE Competitive Grant Program

Funding Agency:
Pfizer Inc.

Pfizer Global Medical Grants (GMG) supports the global healthcare community’s independent initiatives (e.g., research, quality improvement or education) to improve patient outcomes in areas of unmet medical need that are aligned with Pfizer’s medical and/or scientific strategies. Pfizer’s GMG competitive grant program involves a publicly posted Request for Proposal (RFP) that provides detail regarding a specific area of interest, sets timelines for review and approval, and uses an expert review panel (ERP) to make final grant decisions. Organizations are invited to submit an application addressing the specific gaps in research, practice or care as outlined in the specific RFP.

Projects that will be considered for Pfizer support will focus on the following areas:

Basic and Clinical Science of Gene Therapy for Hemophilia: 1. What are the mechanisms determining tropism, transduction, safety, and tolerability of vectors for gene therapy? 2. What is the prevalence and impact of AAV neutralizing antibodies? Which strategies can be used to circumvent preexisting antibodies, with the ultimate goal of either re-dosing or expanding potential eligibility for gene therapy? 3. What mechanisms underpin hepatic and/or immunologic adverse effects of gene therapy? What management strategies can be used to mitigate risk or treat adverse responses (e.g., immunosuppressive regimens)? 4. What mechanisms influence the durability and/or interpatient variability of response to gene therapy? What factors predict response and are there biomarkers that can be identified to guide decision-making? 5. With a focus on optimal long-term surveillance, what innovative approaches can be used to provide long-term follow-up in persons receiving gene therapy (e.g., digital approaches; optimized registry design, maintenance of engagement)?

Basic and Clinical Science of Tissue Factor Pathway Inhibitor (TFPI) and anti-TFPI monoclonal antibodies: 1. What are the mechanisms involved in the interaction and crosstalk between TFPI, protein C, antithrombin III and protein S? 2. What is the impact of concomitant treatments (e.g., antifibrinolytics and hemostatic treatments used in combination with anti-TFPI) on hemostasis? 3. What is the role of TFPI in pathological states associated with enhanced thrombogenesis? How can laboratory investigation be optimized? 4. What is the potential role of anti-TFPI in bleeding disorders other than hemophilia A &B?

Burden of disease: Clinical Hemophilia A and B: 1. What is the natural history of hemophilia in the modern era, where prophylaxis has gained broad adoption? What gaps remain with respect to optimal treatment and adherence? Natural history of hemophilia with inhibitors? 2. What unmet need remains with respect to arthropathy in the current treatment era? What is the clinical burden, impact, and natural history of arthropathy? 3. What unmet needs remain with current standard of care treatment? What is the impact on patient-relevant outcomes such as quality of life, treatment preference, work productivity, and quality of care? What is the unmet need in patients with inhibitors? 4. With a focus on optimal long-term surveillance, what innovative approaches can be used to provide long-term follow-up in persons receiving hemophilia treatment (e.g., digital approaches; optimized registry design, maintenance of engagement)?

Deadline: May 3, 2022 (was March 1)

Agency Website

Eligibility Requirements

Geographic Scope • All countries except for Africa/MiddleEast, Asia-Pacific, and Latin America (a separate program)

To be eligible: • The institution and principal investigator (PI) must be based in one of the eligible countries noted above. • Only organizations are eligible to receive grants, not individuals or medical practice groups. • The applicant (PI) must have a medical or postdoctoral degree (MD, PhD, or equivalent), an advanced nursing degree (BSN with a MS/PhD), or a degree in Pharmacy, Physiotherapy, or Social Work. • Applicant must be affiliated with a host institution

Amount Description

• Individual basic science projects requesting up to $90,000/year for 1 to 2-year projects will be considered. Pfizer anticipates awarding up to 1 grant

• Individual clinical science projects requesting up to $110,000/year for 1 to 2-year projects will be considered. Pfizer anticipates awarding up to 1 grant

Funding Type

Grant

Eligibility

Faculty

Category

International Opportunities
Medical
Medical - Basic Science
Medical - Clinical Science

External Deadline

March 1, 2022