Applications Invited for Translational Research to Develop Gene-Based Therapies for Cystic Fibrosis

Emily’s Entourage (EE) is a US non-profit organisation that supports research to develop therapies for people with cystic fibrosis (CF) who do not benefit from CFTR-modulators, including those with rare and nonsense mutations.

EE is inviting applications for the 2025 round of its Translational Grant Programme and is seeking proposals from researchers based anywhere in the world for translational research in identified high-need areas, including nucleotide-based therapies, innovative inhaled antimicrobial formulations and overcoming immune recognition for gene therapy delivery systems.

The grants award critical seed funding of up to $220,000 over two years to support researchers pursuing innovative strategies with the potential to accelerate the development of gene-based therapies and drug-delivery mechanisms for CF.

Areas of specific interest for the 2025 call for applications include:

  • NucleotideBased Therapies – support for innovative approaches for nucleotide-based therapies for CFTR correction, including, but not limited to:
    • Gene editing platforms (CRISPR, base editing, prime editing) formulated for inhaled and systemic delivery with enhanced lung deposition and cellular uptake;
    • Novel encapsulation technologies including lipid nanoparticles, polymeric carriers, and hybrid delivery systems optimised for nebulisation;
    • Immune-evasive formulation strategies incorporating immune-suppressive excipients, stealth coatings, and targeted delivery to specific lung cell populations;
    • Next-generation viral vectors (AAV, lentiviral, adenoviral) with reduced immunogenic profiles and enhanced lung tropism.
  • Novel Inhaled Formulations for Staphylococcus aureus and CF Pathogens – for research to address the current need for innovative inhaled antimicrobial formulations and delivery systems. Priority targets include MRSA, nontuberculous mycobacteria (NTM), and other high-burden CF pathogens. Proposals are sought for research into novel formulation approaches, including, but not limited to:
    • FDA approved antimicrobials formulated specifically for pulmonary delivery;
    • Advanced liposomal and lipid-based formulations of existing antibiotics for enhanced lung penetration and sustained release;
    • Sustained-release formulations enabling extended antimicrobial activity with reduced dosing frequency;
    • Combination inhaled therapies integrating antimicrobial-adjuvant combinations;
    • Novel inhaled antifungal formulations addressing fungal co-infections and emerging resistance patterns.
  • Understanding Immunogenicity in CF Nucleotide-Based Therapies – nucleotide-based therapies for CF encounter significant obstacles from immune recognition and safety issues that can substantially reduce treatment effectiveness. There is critical need for insights to advance research on safe, effective, and repeatable gene therapy delivery systems. Proposals are sought for research that overcomes current barriers to successful gene therapy administration, enabling patients to receive multiple treatments safely and effectively over time, including, but not limited to:
    • Understanding of pre-existing immunity and treatment-induced immune reactions;
    • Immunosuppressive approaches to reduce immune system activation;
    • Novel strategies to bypass immune recognition.

Eligible applicants are faculty, based at academic institutions anywhere in the world, who have a doctoral qualification and good track record of peer-reviewed publications and extramural funding. Collaborative research by investigators with experience in gene-targeting strategies within or outside of the CF field is strongly encouraged.

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