CAF Establishes New Gene Therapy RFA

December 16, 2008 – The Cooley’s Anemia Foundation announces a new RFA in the area of gene therapy for thalassemia.

The purpose of this initiative is to speed the application of recent translational advances in gene therapy to clinical trials. The foundation recognizes that there are many key areas worthy of support in the search for a cure for thalassemia with cell therapy and gene therapy. While several funding mechanisms are available for a range of studies on vector development, proof of concept studies in animal models, and transplantation, there remains a funding gap for launching human trials. Both phase I (safety) and phase II (efficacy) trials are eligible for support.

Funding and candidate eligibility:Total funding of up to $75,000 per year will be available (including indirect costs of 8%). With satisfactory evidence of progress the grant may be renewed for a second year. The research may not be conducted at a for-profit laboratory.

Because the purpose of the support is to facilitate launch of clinical trials, eligible studies proposed for this RFA must be accompanied by a realistic timeline of progress to human trials. The foundation recognizes that this timeline can be long. Applicants should state explicitly the status of their proposed trials, for example whether the trial has been approved by the recombinant DNA Advisory Committee (RAC), or Food and Drug Administration (or equivalent processes at non-US sites), or whether review meetings at regulatory agencies have been scheduled.


Applications (in Word format) can be downloaded by clicking here.

The completed application is due February 2, 2009. Applications should be accompanied by evidence of RAC approval for the protocol.

Completed applications should be sent to:

Gina Cioffi, Esq.

National Executive Director

Cooley’s Anemia Foundation, Inc.

330 Seventh Avenue, Suite 900

New York, NY 10001

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