Intranasal delivery of nucleic acid therapeutics for neurodegenerative diseases using a novel peptide carrier

About the Project

Neurodegenerative diseases such as Alzheimer’s and Parkinson’s Disease represent a growing global health burden, with limited treatment options that effectively target the brain. One of the major challenges in developing therapeutics for these conditions is the blood-brain barrier, which restricts access of systemically administered drugs to the central nervous system. This project proposes a novel, non-invasive intranasal delivery strategy using a cell-penetrating peptide to transport nucleic acid therapeutics directly to the brain. By exploiting the olfactory and trigeminal nerve pathways, this approach bypasses the blood-brain barrier and enables targeted delivery to key brain regions implicated in neurodegeneration. The student will investigate formulation strategies, cellular uptake, and therapeutic efficacy of peptide nanoparticles in relevant in vitro models, with potential progression to in vivo studies. This project offers a patient-friendly alternative to invasive or systemic delivery methods and aims to advance the development of gene-based therapies for neurodegenerative disease.

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

Neurodegenerative disease, Intranasal drug delivery, Cell-penetrating peptides, Nucleic acid therapeutics, Nanomedicine, Blood brain barrier