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. The Foundation recognizes that this timeline can be long. 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. This will best enable reviewers to assess feasibility of the proposed project. Examples of expenses allowed will include:

• Clinical-grade vector production and quality control
• Research subject assessments
• Late-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 study
• Research 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.

Eligible Countries:

Sponsor Institute/Organizations: Cooley's Anemia Foundation

Sponsor Type:

Address: Cooley's Anemia Foundation; 330 7th Ave Ste 200; New York, NY; United States

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