Background & Purpose

Please click on the “RFA Announcement” link for complete information.

BACKGROUND

The JDRF Cures Program aims to halt the progression of T1D and ultimately achieve a cure through the development of disease-modifying therapies (small molecules and/or biologics) that promote the survival, function, and regeneration of endogenous insulin-producing beta cells (Link to Strategy Document Here).

Novel targets and pathways have recently been described that are able to promote beta-cell regeneration and survival. However, it has been empirically determined that many of these targets and pathways are not sufficiently specific or unique to the islet/beta cell and may present safety concerns if delivered systemically when non-pancreatic cellular targets could become deleteriously affected. Targeted drug delivery presents an opportunity to restrict a regenerative or survival therapy to the islet/beta cell and avoid or reduce deleterious effects on tissues or cells outside the pancreas.

Targeted drug delivery, also referred to as active or smart drug targeting, has most notably been successful in the oncology setting. It is likely that active drug targeting approaches, such as utilizing small molecule, biological (e.g., antibodies, aptamers, peptides, etc.), or nanomaterial drug conjugates to selectively bind to the target, could mitigate potential non-islet/beta-cell activities while increasing the potency of the therapeutic due to targeted delivery. In addition to traditional drug conjugate approaches, JDRF is also interested in gene delivery approaches via viral (e.g. AAVs, etc.) or non-viral (e.g. biomaterial scaffolds, etc.) platforms. The utility of newer generations of viruses with beta cell specific trophism are of particular interest.

To view the informational webinar that took place on August 3rd, please click here.