Beta Cell Targeted Drug Delivery in Diabetes

Curative treatments of Diabetes remain elusive.

A major cause of failure in drug development, is the balance between sufficient drug exposure at the target receptor and the emergence of off-target effects.

Also, drug target receptors may be present in other tissues, where activation generates unwanted effects.A straightforward solution is to modify the drug with a guiding moiety, which in turn binds to a target exclusively expressed on the cells of interest.

Furthermore, the guiding moiety must have certain properties, including suitable size, stability, molecular handles for flexible functionalization as well as low off-target interactions and rapid blood clearance.

The proposed ZAM106 – DGCR2 drug delivery system clearly fulfills all the afore-mentioned criteria and offers a unique opportunity given the beta cell specificity of the target combined with a unique, small and versatile high-affinity Affibody molecule as guiding moietyAt the end of the project, we anticipate to have developed and validated a beta cell specific drug delivery system.

The guiding system will be designed with versatility in mind, to enable a broad application on different classes of potential T1D and T2D drugs, including small organic molecules, biologic drugs, cell therapies and gene therapies.

A beta cell specific drug delivery system would enable conceptually new anti-diabetic therapeutics and re-assessment of earlier drug candidate failures due to off-target and off-tissue effects and toxicity.