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Clinical Trials in Thalassemia Cell and Gene Therapy Application

Cooley's Anemia Foundation, Inc.

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.

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. Excluded from funding in this grant are lodging and other personal costs for research subjects to come to a treatment center.

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.

The research may not be conducted at a for-profit laboratory.

Letters of reference are not required.

AI Based Application Success Predictor

Although CAF does not publish a formal scoring rubric, the following factors are consistently important based on application guidelines and funder emphasis:

✅ 1. Strong Clinical or Translational Relevance to Thalassemia

Applications must focus directly on thalassemia or its complications — not generic hematology or unrelated biology.

Clinical focus and relevance to meaningful patient outcomes (e.g., reducing iron overload complications, improving fertility/family planning) are essential. Cooley’s Anemia Foundation

Predictor: Demonstrate clear patient relevance and direct links between study results and clinical impact for individuals with thalassemia.

✅ 2. Feasibility & Detailed Study Plan

Proposals should include a realistic timeline, achievable milestones, and a level of work that matches the funding (both time and budget).

For clinical trials, clear documentation of regulatory status (e.g., FDA review meetings, trial approvals) and practical plans for vector production, subject assessments, and safety/efficacy endpoints are emphasized. Cooley’s Anemia Foundation

Predictor: Clear study design with well-defined deliverables and justification for each stage of work.

✅ 3. Solid Preliminary Data or Rationale

For competitive clinical trials or ongoing research, reviewers look for evidence that the investigator understands key biological or clinical issues and that the project builds on a credible scientific basis.

In fellowship applications, a proposal that reflects thoughtful preliminary work or strong conceptual foundation (reviewed with mentor) strengthens competitiveness. Cooley’s Anemia Foundation

Predictor: Present preliminary results or a strong scientific rationale supporting hypotheses.

✅ 4. Investigator Experience & Environment

Fellowships are targeted to postdocs and junior faculty; strong mentorship and institutional support are described as important, especially in postdoc applications.

For clinical research and trials, documented access to appropriate clinical facilities, patient populations, and necessary research infrastructure is expected. Cooley’s Anemia Foundation

Predictor: Demonstrate that the investigator and support environment are capable of executing the project effectively.

✅ 5. Clinical Research Credentials and Ethics

Investigators must interact directly with patients or patient-related data for clinical research applications, and ethical approvals (IRB/human subjects) must be in place or clearly planned. Cooley’s Anemia Foundation

Predictor: Show preparedness for human research standards and ethical compliance.

✅ 6. Budget Justification & Responsible Use of Funds

CAF limits administrative costs (≤ 8–10 %), and research budgets should reflect direct research needs, such as personnel support, preclinical/clinical assays, and trial infrastructure. Cooley’s Anemia Foundation

Predictor: Prepare a tight, well-justified budget aligned with achievable project outcomes.

✅ 7. Competitive, Well-Written Letter of Intent and Full Proposal

CAF uses a two-stage process for many grants: a short Letter of Intent (LOI) followed by a full application if invited. Cooley’s Anemia Foundation

A strong LOI that succinctly outlines the problem, hypothesis, impact, and feasibility can drive further review.

Predictor: Craft concise, compelling LOIs that clearly communicate why your project matters and how it will succeed.

📌 Fellowship-Specific Success Predictors

For the Research Fellowship Program:

Focused thesis-driven project related to thalassemia biology or treatment. Cooley’s Anemia Foundation

Mentorship plan (postdoc must have an experienced sponsor). Cooley’s Anemia Foundation

Preference for candidates without major NIH R01 funding, favoring emerging investigators. Cooley’s Anemia Foundation

Institutional approval and demonstration of adequate research facilities are required. Cooley’s Anemia Foundation

Predictor: Show independence, clarity of research direction, and mentor support tailored to thalassemia research.

🧠 Summary: Key Success Predictors

Predictor	Why It Matters
Direct thalassemia focus	CAF funds disease-specific work only. Cooley’s Anemia Foundation
Feasible research plan	Match project scope to funding resources. Cooley’s Anemia Foundation
Evidence/rationale	Shows credibility and potential impact. Cooley’s Anemia Foundation
Qualified investigator & environment	Ensures project deliverability. Cooley’s Anemia Foundation
Ethics & patient focus readiness	Necessary for clinical research. Cooley’s Anemia Foundation
Strong budget justification	Aligns resources to research aims. Cooley’s Anemia Foundation
Clear LOI & proposal narrative	Drives reviewer interest early.

Specialties

Clinical Trials

Genetics & Genomics

Eligibility Requirements

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.

Sponsor Details

Sponsor Institute/Organizations: Cooley's Anemia Foundation, Inc.

Sponsor Type: Corporate/Non-Profit

Address: 330 7th Ave Ste 200 New York, NY 10001 (212) 279-8090

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