Cooley’s Anemia Foundation: Clinical Trials in Thalassemia Cell & Gene Therapy Grant

The Cooley’s Anemia Foundation is committed to improving the lives of individuals affected by thalassemia, particularly through funding clinical trials in Cell and Gene Therapy. With a yearly budget of up to $75,000, the foundation supports both phase I and II trials aimed at developing effective treatments. Applicants must provide a detailed timeline and ensure compliance with regulatory standards. This initiative seeks to enhance life expectancy and quality of life for thalassemia patients while striving for a universal cure.

Statement of Purpose
The Cooley’s Anemia Foundation is dedicated to serving people afflicted with various forms of thalassemia, most notably the major form of this genetic blood disease, Cooley’s anemia/thalassemia major.
We do so by funding medical research to advance treatment and curative approaches, by supporting and advising patients and their families and advocating on their behalf and by educating medical professionals and the general public.
The Cooley’s Anemia Foundation invites national and international applicants to apply for grants to facilitate clinical trials in Cell and Gene Therapy to advance a cure for thalassemia. Both phase I (safety) and phase II (efficacy) trials are eligible for support.
Funding
Total funding of up to $75,000 per year will be available (including indirect costs of 8%). With satisfactory evidence of progress and availability of funding grants may be renewed for a second year.

Eligibility

Applicants should state explicitly the status of their proposed trials; for example, whether the trial has been approved by the Food and Drug Administration (or equivalent processes at non-US sites) or whether review meetings at regulatory agencies have been scheduled.Studies proposed for this RFA must be accompanied by a realistic timeline of progress to human trials. While the funds may be used toward any aspect of launching a human gene therapy trial, the Foundation will request careful documentation of other support of the investigator(s) and the project, and a thorough justification of the allocation of costs to various support mechanisms.Examples of expenses allowed will include:Clinical-grade vector production and quality controlResearch subject assessmentsLate-preclinical studies and ex vivo studies on research subjects required for safety and efficacy assessments (including, but not limited to, evaluation of vector genome integration events, studies of transduction efficiency, studies of globin production by transduced hematopoietic stem cells from subjects)Patient care costs directly related to the studyResearch assistant or research nursing support.Assurance must be given that adequate clinical and other facilities (including, where applicable, laboratory and vector production facilities) exist and are available to conduct the research project and, in case of investigations involving human subjects, a full human subject protection plan should be included as an appendix.Support for investigator effort may not exceed 10% of the current NIH salary cap.Animal studies, if any, must be directly relevant to the specific proposed clinical trial.

Ineligibility

The research may not be conducted at a for-profit laboratory.Excluded from funding in this grant are lodging and other personal costs for research subjects to come to a treatment center.