Non-invasive intranasal delivery of nucleic acid therapeutics for retinal disease using a novel peptide carrier

About the Project

Pathological retinal neovascularisation is a leading cause of vision loss globally which is characteristic of diseases such as diabetic retinopathy, age-related macular degeneration, and retinopathy of prematurity, collectively affecting >300 million people worldwide. Anti-VEGF therapies are the current gold standard for such conditions, but these require frequent intravitreal injections which are associated with complications and high treatment burden for patients. To address these limitations, this project proposes a non-invasive intranasal delivery strategy using a novel cell-penetrating peptide to transport nucleic acid therapeutics to the retina. This approach exploits the trigeminal and olfactory nerve pathways to deliver therapeutics directly to the retina, as an alternative to invasive intravitreal injections, with a more patient-friendly approach.

Briefly describe the training that will be provided through the research project

Right from the start the PhD student will be involved in academic research designed to have translational/clinical application. This dual approach spans:

1. Research Skills: the academic supervisors will ensure excellent training in nanoparticle formulation, systematic physical characterisation, in vitro cell and molecular biology techniques and potentially in vivo skills.
2. Record keeping & monitoring: Monthly meetings with the student will take place with electronic records. Students must also complete a 3-month initial review and annual progress review to proceed to years 2 & 3. The annual progress review involves written work, presentation and/or mini viva. However, at each of these meetings, the primary supervisor will also be present ensuring that the maximal training benefit can be derived from these processes.
3. Additionally, there will be opportunities to present at academic meetings, building professional networks, personal development on courses for animal licenses, advanced statistics, skills which are all relevant to subsequent employment opportunities.

Briefly outline the expected impact activities

Impact activities include but are not restricted to presenting the research to academic and industry peers through scientific conferences and students from different disciplines through the Graduate School. The student will also engage with patients, clinicians and key stake holders through a series of webinars/focus groups to understand how they can feed and shape the research plan. Other impact activities relate to commercialisation though IP protection processes, competitor analysis and engagement with clinical collaborators.

Funding Notes

This project is not funded; applications are welcome from self-funding candidates.

References

Retinal disease, Intranasal drug delivery, Cell-penetrating peptides, Nucleic acid therapeutics, Nanomedicine