The originality of AC Immune’s Research and Development (R&D) Strategy is due to its combination of both an immunology and a chemistry platform technology to develop innovative therapies against conformational diseases, such as Alzheimer´s Disease. This strategy aims to produce new therapeutics along three axes - vaccines, antibodies and small molecules – and also to develop diagnostic tools for these types of diseases.
One of the challenges of conformational diseases comes from the fact that the proteins that need to be targeted are “self” proteins, coming from our own organism, and they do not elicit an immune reaction from the body. Moreover, the difference between a “good” and a “bad” protein is typically only expressed by a conformational change in the protein structure.
The immunological SupraAntigen™ Technology and the chemical Morphomer™ platform are designed to produce high affinity ligands, which bind specifically and with high-affinity to proteins in “sick” conformations.
Both platforms lead to new classes of therapies that “break” the pathological conformation of proteins by shifting the equilibrium via stabilization of the soluble form of the protein target and therefore render them harmless.
The initial focus of AC Immune´s R&D Strategy has been on addressing Alzheimer's Disease with misfolded Abeta as a first target. This has successfully generated two biologics and one small molecule now under clinical development. The technology platforms are also currently leveraged for addressing misfolded Tau as a second target in Alzheimer's Disease.
Misfolded Abeta Protein has recently been identified as a central point of all degenerative diseases. This aspect gives AC Immune the opportunity to leverage its products in other CNS and non-CNS disorders, while the focus on Alzheimer's Disease will remain unchanged. The leveraging strategy will deliver a stronger pipeline of compounds with different applications leading to a “natural” risk diversification of the product pipeline.