Therapeutics Development Award Program
By supporting early to mid-phase drug development activities with both financial and technical support, the Cystic Fibrosis Foundation's Therapeutics Development Award (TDA) program is designed to stimulate and de-risk CF drug development of novel therapies for cystic fibrosis. The program provides companies with funding and other resources to support studies that may include drug discovery; pre-clinical safety; pharmacokinetics/pharmacodynamics (PK/PD); and clinical studies in people with CF through Phase 2 development.
Applications will be considered in any therapeutic area that could benefit people with CF but are particularly encouraged in areas related to treating CF infections, CFTR modulation, mucus clearance (mucolytics and airway surface hydration), inflammation, chronic lung allograft dysfunction, CF-related diabetes, CF liver disease, and nutrition.
To be funded through this mechanism, organizations are expected to provide internal funds that at least match the level of CF Foundation support. Generally, Foundation support may not be used for manufacturing and controls (CMC production), to conduct clinical trials in only healthy human subjects, or to support senior personnel engaged in administrative roles without specific approval by the Foundation.
The TDA program provides two mechanisms of support:
- Component I awards provide funds up to $600,000 over two years for early exploratory, discovery, and/or preclinical development studies.
- Component II awards are considered for more advanced programs that generally have identified a clinical candidate and are moving into investigational new drug (IND)-enabling studies such as PK/PD, GLP toxicology, and Phase 1/2 clinical trials in CF patients. Component II awards are typically supported at a level of up to $3 million-$5 million for two or more years and require substantial matching funds. Only direct costs are covered.
Award pay-out is typically milestone-based. If a TDA contributes to successful regulatory marketing approval, the CF Foundation will receive reimbursement plus a multiple and/or a percentage of net sales. Terms are negotiated prior to finalizing the award. If a program is unsuccessful, no payback is expected.
Industry Award for Path to a Cure Program
The Path to a Cure (PTAC) initiative is the Foundation's ambitious research program to support development of treatments for the underlying cause of the disease and, ultimately, a cure for every person with CF.
Although significant breakthroughs are helping people with CF live longer and healthier lives, there are still many individuals who will not benefit from CFTR modulators. Therefore, the Foundation is prioritizing efforts to identify innovative therapeutic approaches for these people with CF.
Industry programs supported through the PTAC initiative will focus on two core strategies to address the underlying cause of CF:
- Restoring CFTR protein when none exists, including for people with CF who have nonsense mutations
- Fixing or replacing the underlying genetic mutation to address the root cause of CF through gene replacement/transfer, gene editing approaches, or stem cell therapy
The Foundation intends to allocate $500 million through 2025 for these efforts and is challenging potential collaborators to submit proposals that will accelerate the pace of progress for CF drug discovery and development in areas that include, but are not necessarily limited to, the following priority areas:
- Preclinical: Identify viable readthrough compounds and other strategies to support development of treatments for people with nonsense mutations. Develop platforms for gene delivery and editing strategies.
- Targets: Develop methods to target appropriate cell(s) in the lung, GI tract, and other tissues affected by CF that can be used for CF genetic-based therapies.
- Delivery: Develop vectors that efficiently target and deliver nucleic acid cargo to the correct cells in the correct tissue without inducing an immune response (allowing for re-administration).
- Editing: Develop effective gene editing methods, amenable to clinical development, to correct mutations in the CFTR gene.
- Clinical Studies: Advance treatments to the clinic with a high potential to benefit people with CFTR nonsense and rare mutations that are not correctable by CFTR modulators.
Any commercial programs developing therapies or therapeutic platforms with the potential to advance these goals will be seriously considered. The amounts of awards given through the PTAC initiative are expected to be substantially higher than those through the TDA program -- up to tens of millions of dollars. Contract terms are negotiated prior to finalization of the agreement.
In addition to funding, the Foundation offers partners a range of resources to de-risk CF drug discovery and development. This includes access to a robust community of leading academic researchers, preclinical CF-related model systems, infrastructure for preclinical development, and access to the world's largest network of CF clinical trial sites. Additionally, consulting advice on trial design is supported through the Therapeutics Development Network Coordinating Center.
Learn more about the Path to a Cure.
Policies and Guidelines
Please review the appropriate Policies and Guidelines for complete submission information:
For those who seek support through the TDA or PTAC program and want to ensure the proposal is within the scope of research that supports the CF Foundation's mission, investigators should submit a letter of intent (LOI) for initial CF Foundation review in advance of a full proposal.
Both LOI and full submissions are accepted throughout the calendar year, although it is highly unlikely that submissions not received by October 31 will be funded in that calendar year.
Applicants are to submit LOIs and full applications online at awards.cff.org.
For More Information
For further information or to discuss the potential relevance of studies for this program, please contact Lindsey Beaman, email@example.com, and/or Maria Wang, firstname.lastname@example.org.