Guest Blog | CF Foundation’s Path to a Cure Programme
Guest blog by Oli Rayner, Business Development Consultant, Cystic Fibrosis Foundation
Since the discovery of the gene that causes cystic fibrosis in 1989, advances in our understanding of CF have been profound. CFTR modulators, which address the underlying cause of CF by modifying the CFTR protein, are beginning to change what it means to have cystic fibrosis. However, we still have a long way to go before everyone with the condition has an effective therapy to treat their particular mutations and, ultimately, a cure.
The global cystic fibrosis community is focused on completing this mission. In October 2019, the Cystic Fibrosis Foundation launched Path to a Cure, an ambitious $500 million research agenda to accelerate genetic-based treatments for the disease and develop a cure for every person living with cystic fibrosis.
Nearly every approved CF drug available today – more than ten therapies - was made possible with support from the Foundation. In addition to providing funding for basic science and clinical research, the Foundation supports therapeutic development through research tools, scientific advice, and an integrated international clinical trials network dedicated to CF. In this way, the Foundation can de-risk early stage studies and enable promising science to move forward in ways that would not otherwise be possible. Our Path to a Cure initiative builds on a unique venture philanthropy strategy that the Foundation has pioneered over the last 25 years to drive therapeutic development.
Some people with CF have mutations that do not respond to CFTR modulators. Even when people have a combination of mutations that do respond to CFTR modulators, they still experience chronic infections, pulmonary exacerbations, inflammation, and gradual decline, and suffer from non-lung complications of CF such as GI symptoms and CF-related diabetes. All of these require additional treatments over an extended period of time. Alongside the Path to a Cure initiative, the Foundation is actively investing in the development of therapies to address these needs. For example, the Infection Research Initiative is a $100m investment programme to improve the detection, diagnosis, treatment, and outcomes of CF-related infections.
The Foundation funds projects from the early research stage through Phase II clinical development via a flexible range of funding tools ranging from small grants to larger royalty-based awards and, in certain situations, invests directly in companies to encourage them to develop CF therapies.
Here in the UK, the Foundation works in collaboration with the CF Trust to ensure that promising therapeutic development opportunities are investigated and progressed for the benefit of people with CF. The CF Trust’s Clinical Trials Accelerator Platform is supported by the Foundation and works in close coordination with NIHR’s Translational Research Collaboration and the European Cystic Fibrosis Society’s clinical trial network.
As a consultant for the Cystic Fibrosis Foundation in the UK and continental Europe, my role is to identify therapeutic development opportunities and potential academic and biopharma collaborators with promising projects in genetic therapies and novel antimicrobial strategies, and I am eager to work with individuals and companies who may be undertaking relevant research in these areas.
In addition to identifying specific therapeutic development opportunities, we are interested in platform technologies that may not currently be directed towards CF but which have strong potential.
Please reach out me if you are working on a therapeutic development project that you feel could fit with the Foundation’s strategy and would benefit from funding support, specialist scientific/development advice, or connection to enabling resources.
Business Development Consultant
Cystic Fibrosis Foundation
For further information:
Infection Research Initiative: https://www.cff.org/Research/Research-Into-the-Disease/Research-into-CF-Complications/Infection-Research-Initiative/
Other Research Funding & Support Mechanisms: https://www.cff.org/Research/Researcher-Resources/Awards-and-Grants/
Therapeutic Development Pipeline: https://www.cff.org/trials/pipeline
Therapeutics Development Network (CF clinical trials network):https://www.cff.org/Research/Researcher-Resources/Therapeutics-Development-Network/