Sovvenzioni per la traduzione

Il programma di sovvenzioni translazionali di Emily's Entourage (EE) fornisce finanziamenti fondamentali a ricercatori innovativi con strategie promettenti che accelerano la ricerca e lo sviluppo di farmaci per coloro che fanno parte dell'ultimo 10% della comunità FC che non traggono beneficio dai modulatori CFTR, compresi quelli con mutazioni rare e nonsense.

For the 2026 cycle, EE will prioritize proposals that turn decisively toward clinical development and funding the development of technologies that already show promise. EE is non looking to fund early screens intended to identify a lead or target identification or basic mechanistic studies without a defined therapeutic candidate.

Potential areas of interest include, but are not limited to, the following:

• Gene Editing for Specific CF Nonsense Variants: Adenine base editing (ABE) and prime editing offer the possibility of durable correction of disease-causing CFTR variants. Many common CF nonsense variants (G542X, W1282X, R553X, R1162X) arise from a C•G→T•A transition and are therefore chemically amenable to ABE. Priority questions include the minimum effective correction level in airway epithelium required for clinical benefit, elimination of bystander edits, editing efficiency across airway cell subtypes (basal versus differentiated cells), and the durability of correction in a continuously turning-over epithelium. 

• Delivery of Genetic Therapies to the CF Airway: Delivery remains the primary unsolved problem in CF gene editing: the target organ is the lung and the CF airway is an obstructed, chronically inflamed, biofilm-laden environment that traps and degrades nanoparticle and viral payloads. EE is particularly interested in LNPs (aerosolization stability, mucus penetration, lung tropism of  lipids, reduced immune activation, extended payload half-life and reduced dosing frequency) and engineered airway-tropic AAV capsids together with re-administration or immunosuppression strategies as well as systemic delivery strategies that have lung tropism.

• Antisense Oligonucleotide (ASO): Priorities include ASO lead selection and chemistry, GMP manufacturing, IND-enabling inhalation toxicology and off-target splice profiling, inhaled delivery to the CF airway, and the effects of combining an ASO with approved CFTR modulators. 

• Screening Assays for Immunogenicity of Genetic Therapies in the CF Airway: Because airway epithelial cells turn over continuously, repeat dosing will almost certainly be required for any nucleotide based therapy. LNP components and viral capsids may lead to inflammation/immune responses which may be amplified in the CF airway, already primed by chronic bacterial colonization and baseline inflammation. The field currently lacks validated, standardized assays to predict this immunogenicity. We are seeking proposals for screening assays that characterize and de-risk the immunogenicity of genetic therapies for CF, including but not limited to: innate immune activation panels in primary human CF airway epithelial cultures; assays performed under CF-relevant conditions (chronic inflammation, mucus, bacterial products) rather than in naive systems alone; and repeat-dose models that quantify loss of editing or transduction efficiency across sequential administrations.

• Small Molecules for Readthrough and Nonsense-Mediated Decay: Readthrough compounds and inhibitors of nonsense-mediated mRNA decay (NMD) are gene-independent, orally available approaches applicable across nonsense variants; the historical limitation has been potency. EE is interested in next-generation readthrough compounds, selective NMD modulators that have in vivo proof-of-concept. We are looking to fund tissue distribution studies in CF models, assessment of the breadth of activity across multiple CF nonsense variants, and layered combinations of readthrough plus NMD inhibition plus modulators that could raise functional restoration into the therapeutically relevant range.

• Phage Therapy for CF Pathogens with a particular focus on MRSA: Chronic airway infection continues to drive lung function decline and mortality, including in people on modulators. Bacteriophage therapy is among the most promising anti-infective strategies for CF, and priority questions include the optimal use of cocktail compositions versus sequential single-phage dosing for MRSA, inhaled delivery achieving therapeutic concentrations in the distal airway, phage–antibiotic combinations, emergence and management of phage resistance, and the data and protocols needed to move from compassionate use to formal clinical trials and timely patient access.
  
• Antibiotics and Anti-Infectives: Superbugs such as MRSA are recovered from roughly a quarter of people with CF and are associated with accelerated lung function decline. Areas of interest include next-generation inhaled formulation of antibiotics such as Vancomycin with sustained pulmonary half-life and reduced bronchospasm risk. Our major priority is inhaled formulations that meet the needs of pwCF with chronic infections, including tolerability, safety, and suitability for long-term use. 

Scadenze principali

• Applications will be accepted beginning October 1, 2026, and are due by November 15, 2026 at 11:59 pm (PT).
  
• Applicants will be notified in December 2026 if they are selected to move to the next round.
• Those applicants that move to the next round will have the opportunity to address concerns raised by external reviewers before a final funding decision is made. 
• Projected start date is April 2027.

Si prega di rivedere the 2026 grant application guidelines per i requisiti completi di presentazione.

Se avete domande o desiderate discutere il vostro interesse per le opportunità di finanziamento di EE, non esitate a contattarci qui sotto.