Please present the disease that is the subject of the application include age of onset, mortality, morbidity, demographic etc. What is the disease health, clinical or product need you are seeking to address? Describe which organ(s) are to be targeted.
Please provide a summary including the following: Indication (disease targeted for the product), Biological activity (hypothesised biological function(s), Efficacy (proposed efficacy endpoints), Safety (potential safety risks associated with the product), Dosage form and Dosing, Route of Administration (proposed route of delivery for the product). TPP should capture key goals regarding clinical indication and target product quality characteristics.
What are the competing solutions in development and their shortcomings?
Please explain the scientific rationale for your proposed intervention, include data and experimental supporting evidence. This should include all pre-clinical data in models and toxicology data.
Describe the target gene providing as much detail as possible on structure and gene / part of the gene to be replaced or modified.
Provide a description of the proposed construct and vector. This should include the source of any material and ownership. If possible, any IP liabilities associated with the construct, including the vector, should be disclosed.
Explain the experimental design and provide a statistical justification. Specifically, animal numbers must be justified.
Explain the clinical trial design and provide a statistical justification. Specifically, participant numbers and access must be justified. Include proposed study design and delivery, proposed end-points, and potential safety risks and mitigations.
If yes, please detail your planned or predicted dose requirements (if known)
Do you produce R&D or GMP-grade viral vectors in-house or source them from elsewhere?
If produced in your lab / institution, please provide details of the process you currently use for making viral vectors including producer cell type, culture type (e.g. suspension or adherent), media and any other relevant factors.
If purchased elsewhere, please provide full details including the supplier’s name. We recognise there may confidentiality considerations for this question, please provide as much detail as possible.
If access is required, what IP does the proposal need access to? Detail institutions or individuals holding relevant background IP.
If not, why do you believe you will be able to access the required IP on reasonable terms?
Detail the organisations/individuals who will own any arising IP and any live, pending or envisioned agreements governing management or exploitation of that IP
How is the project going to be supported after the end of the project?
List potential sources of further funding and comment upon the project’s compatibility with the individual fund’s defined remits.
How is the project going to be supported after the end of the project? List potential sources of further funding and comment upon the project’s compatibility with the individual fund’s defined remits.
Outline the likely route to market/patient benefit. If licensing anticipated, list potential partners and describe the required data package to be offered for licensing?
Give details of previous awards that have supported the project (funder, grant title, amount awarded, grant period).
Please note any other issues not mentioned elsewhere in the application form.
