KTH Royal Institute of Technology
Sequestrins - New small protein drugs targeting neurodegenerative disorders
The Swedish Brain Foundation
This application is motivated by very positive and encouraging recent preclinical results (described below) from a novel therapeutic approach in the field of Alzheimer’s disease (AD). The overall objective is to explore how small affinity
proteins can be utilized as new types of protein drugs for neurodegenerative disorders. Affinity proteins have indeed proven suitable for targeting applications, e.g. to detect cancer tumors, but also as capturing agents for blocking of
protein:protein interactions and even to sequester proteins in the circulation. This proposal intends to explore a novel and challenging biotherapy strategy. Very small affinity proteins, termed sequestrins, that are developed in-house, will be
investigated as in vivo capturing agents aimed towards future treatment of neurodegenerative disorders, with an initial focus on AD. A sequestrin is a new type of protein scaffold, based on head-to-tale-linked affibody molecules, aimed to act as
“molecular tweezers” capturing aggregation-prone peptides and proteins.