Our therapy could help patients with a wide variety of diseases that require chronic dosing of a biologic. However, our initial focus is on the areas that will provide the biggest benefits for patients balanced with the shortest time to potential approval of the therapy. The target areas include diabetes, blood disorders, and lysosomal storage disorders.
Current therapies are unable to adequately address long-term sequalae such as joint damage and require multiple infusions every month for patients with blood disorders, such as hemophilia. Our therapy will encapsulate our proprietary cell line for blood disorders with our polymer chemistry to create a game changing therapy for Hemophilia patients potentially providing both improved efficacy and improved quality of life.
There are over 50 lysosomal storage disorders. While some diseases such as Fabry Disease and Gaucher Disease do have treatments available, the vast majority of these diseases do not have commercially available treatments. Our proprietary cell line for lysosomal storage disorders should enable the ability to create treatments for many of these diseases when biologically feasible, potentially providing new therapies for those not yet treated. For those LSD diseases with available treatments, we aim to provide better efficacy via a consistent delivery of therapeutic vs current commercially available treatments.
Our diabetes program underpins much of the foundation of the platform as demonstrated by our publications. Insulin-dependent diabetes is a very compelling application of our Afibromer™ technology, with the challenge of providing dynamic protein delivery in response to specific conditions in the body. This requires a complex clinical development strategy that is best handled with an industry partner where together we can leverage the significant resources required for success.