Applications should be based, at a minimum, on a strong non-clinical data package, with an established path to clinical development. Therapeutic nomination may be hypothesis-driven or unbiased, e.g. in silico prediction, but should be supported by a strong mechanistic rationale, as well as robust experimental validation. Activities may include further pre-clinical validation, studies to support progression to clinical development and clinical proof of concept, delivered within a suitable budget. It is the expectation that most projects will be preclinical and aimed at providing data to justify progression and prioritisation of candidates for clinical trials. Where there is already sufficient justification for a clinical trial, such proposals are in remit although limited to early phases.

Proposals should target the natural pathway of disease progression to prevent or slow the development of chronic wounds, fibrosis, and creation of the tissue environment for initiation of cancers, and might include novel evidence-based approaches to treating infection, and / or reducing inflammation, including through immune modulation, addressing the detrimental microenvironment of chronic wounds or intervening in the progressive alterations in cell fate associated with chronic tissue disruption. While the priority is interventions in disease course up to and including cancer initiation, well-evidenced novel approaches to combatting established or metastatic cancer will be considered.

Although most types of Epidermolysis Bullosa have evident skin involvement, it is a systemic disease with mucosae and internal tracts affected; treatments that address the systemic disease are especially encouraged.

Applicability of the proposed solution to other inflammatory / infected / fibrotic conditions which may or not also be prone to cancer initiation are welcome, although this is not a requirement for the proposed research.

Treatments should be applicable to young patients, and where appropriate, infants to newborns, to enable intervention before tissue damage accrues.

• A new therapy for Epidermolysis Bullosa that slows or prevents the natural pathway of disease progression and thereby contributes significantly to near-future clinical options for treating people with Epidermolysis Bullosa.
• The use of the proposed therapeutic is well validated in a relevant disease model (in vivo / in vitro) and / or human studies.
• The development of the therapeutic is complemented by relevant biomarkers that will improve the use, relevance, and progression of the therapeutic in patients.
• The strategy for clinical development, including addressing IP, regulatory approval, and potential involvement of commercial organisations in further development and delivery.

Timeline

A two-stage process will be used to select projects, with an initial expression-of-interest (EOI), followed by an invited full application.

Funding call status: Closed to EOI. Full applications by invitation only.

Funding type: Grant