BBDF is dedicated to collaboratively developing, funding, and managing fundamental, preclinical, IND-enabling, translational and clinical research to improve the lives of children and young adults with CLN3 disease. Since 2008, BBDF has been the largest funder of juvenile Batten (CLN3) disease research, ranging from basic discovery research to translation into therapies. To inform our research initiatives, BBDF gathers information from one-on-one meetings, broad group discussions, scientific peer-reviewed publications, conference proceedings, drug pipelines, and tailored merit reviews into the foundation’s decision-making processes. Input comes from across the Research and Development spectrum including academia, biotechnology, the pharmaceutical sector, and clinicians, caregivers, and patients.
Our research funding decisions are based on these research objectives:
1) Supporting basic research to discover new mechanisms of disease, targets, and treatments
2) Supporting projects which facilitate execution of clinical trials such as expanding patient registries
3) Identifying biomarkers and other innovative clinical endpoints
5) Supporting projects 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 that will lead to disease mitigation and 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 regularly interacts with 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), and other related rare genetic diseases. 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 foundation 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. 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. LOIs are reviewed internally. We advise the applicant to contact BBDF’s Principal Scientist to discuss their ideas prior to submission. Download and complete the 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
Proposal forms, adapted from the US NIH RO1 format, can be found here. Due to the nature of unique and cutting-edge ideas, not all ideas can be adequately described using a standard application. If this is you and you are invited to submit a full proposal, please contact BBDF’s principal scientist for further instructions. We employ a two-stage process to enhance the fidelity of the reviews. Initially, full proposals are 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. Full proposals are then reviewed by multiple domain experts recruited ad hoc for the subtopic/approach being considered for funding. We believe that tailoring 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 grant applications based on two key attributes:
- 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?)
Once an LOI has been accepted and a proposal is drafted, BBDF advisors work with applicants to ensure that the proposed work meets our collective scientific and therapy-minded goals. BBDF will then enter into a research agreement that includes a detailed project description, milestones, deliverables, budget, and reporting structure. The lead investigator must conduct the research as specified in the grant contract and deliver progress reports to receive milestone-based payments.
We look forward to working with the Batten community to advance research to find new treatments and hopefully a cure for Batten Disease