Since 2008, Beyond Batten Disease Foundation (BBDF) has been the largest funder of juvenile Batten (CLN3) disease research, ranging from basic research discovery to the translation into therapies. Sitting at the nexus of Batten disease research worldwide, we are constantly building and refining our understanding of the pathogenesis of this disease and the corresponding drugs targeting it. Our research portfolio represents a carefully crafted approach for the following stages of research:

1) supporting fundamental discovery to improve and open up new approaches to treatment

2) creating, improving, and disseminating research tools with the potential to accelerate the development of treatments for Batten disease

3) advancing near-term therapeutic approaches that could lead to the rapid development of treatments

4) supporting projects in preparation for clinical trials such as advancing patient registries, identifying biomarkers and clinical endpoints, and building regulatory pathways to approval.

5) establishing therapeutic proof of concept that de-risks pharmaceutical investments in Batten disease

Foundation-Directed Research

The largest portion of the foundation’s portfolio is directed research. The foundation fills gaps in juvenile Batten disease research by funding high-impact projects and breakthrough technologies, advancing paradigm-shifting research that will lead to a cure. Much like a virtual pharmaceutical company, the foundation works with experienced scientists and project managers to identify, develop, coordinate, and monitor a portfolio of activities focused on the development of drug candidates.

BBDF’s Principal Scientist continually interviews leaders in research and development across the entire therapeutic pipeline for Batten, as well as for adult forms of neurodegeneration (e.g. Alzheimer’s and Parkinson’s disease), genetics, lysosomal, and other relevant disorders. To supplement this dialogue, BBDF creates specialty boards and steering committees to lead the development of its projects. A specialty board typically comprises ~200 years of combined experience specific to the proposed activity. A list of experts who have served on one or more of the foundations boards can be found here.

Investor-Initiated Research (Applying for a research grant)

In addition to directed research, BBDF accepts and awards research proposals. Because great ideas do not follow a schedule, we do this on a rolling submission basis. The award size varies widely, from $5,000 to $2 million with an average award of $150,000 over two years. The variability of award size highlights our flexible approach and willingness to explore all options that meet our mission: to eradicate Batten disease. All budgets are carefully reviewed along with the proposed body of work. Applicants may be asked to revise their budgets and/or allow BBDF to share a summary of the proposal with other funders for the purpose of gathering additional support. Sometimes, the foundation will offer the applicant additional funding to incorporate additional expertise or methods into his or her plan.

The Application and Scientific Review Process

Letter of Intent

Applicants and potential co-funders are asked to submit a Letter of Intent (LOI) that provides a preliminary overview of the project, resource, or therapy. LOIs are reviewed internally. It is in the best interest of the applicant to contact BBDF’s Principal Scientist to discuss their ideas prior to submission. Download a copy of the fillable LOI form. If your LOI is considered to be within the scope of our funding priorities, you will receive a call or an email with an invitation to submit a full proposal.

Full Proposal

Full proposals are reviewed by multiple domain experts recruited ad hoc for the subtopic/approach being considered for funding. We employ a unique two-stage process to enhance the fidelity of the reviews. Initially, full proposals are rapidly reviewed by BBDF’s principal scientist and one or more trusted advisors, to give the investigator feedback regarding anything that was considered unclear or that was flagged as potentially problematic. This gives the investigator a chance to clarify and rework the proposal prior to the formal review. BBDF then recruits individuals with expertise specific to each aim, research method, and/or anticipated outcome to conduct the formal review. We believe that the time-intensive tailoring of each review pays off by ensuring that each proposal we fund is in the best possible hands with access to the most advanced technologies. In fact, we often propose additional aims and funding at the request of our reviewers.

Scoring

The committee scores grants along two key dimensions:

  • Feasibility (how likely is the investigative team to prove their hypotheses?)
  • Significance (how important will the proven/disproven hypotheses be to the creation or development of therapy?).

By separately scoring these two dimensions, BBDF can strategically manage the risk-profile of our grant portfolio while maintaining laser focus on the end goal: — to eradicate juvenile Batten (CLN3) disease. Every proposal that is not triaged separately by all reviewers is discussed in-person or by teleconference.

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