Neurodegenerative diseases are devastating diseases with huge unmet medical needs. Decades of research have been invested in finding cures, however almost none have been successful so far. Antibodies are great therapeutic molecules as they can target different aspects of disease-causing processes with high specificity. Within this class, human-derived antibodies stand out for their unique properties and therapeutic potential. For example, they are naturally designed by the human immune system to fight toxic moieties and have matured in the human body, resulting in an expected higher efficacy and specificity compared to antibodies deriving from synthetic libraries or animal models. However, human subjects harboring target-specific antibodies for neurodegenerative diseases are extremely rare, down to 1 every 10,000 people. Thus, to identify such rare individuals an extremely large number of blood samples needs to be screened, which is rarely available to drug discovery.
Mabylon’s antibody discovery platform overcomes these limitations with access to one of the largest and most diverse libraries of blood samples deriving from up to 100,000 human subjects per year. The library further distinguishes itself by containing both diseased and healthy patient samples which, together with access to patient medical history, advanced data-analytics, and cutting-edge B-cell screening technology, overall increases the chance of finding protective, therapeutically relevant antibodies.
Mabylon’s lead molecule is an antibody against pathological TDP-43 for the treatment of
amyotrophic lateral sclerosis (ALS) and a second antibody for Alzheimer’s disease (AD) therapy.
For the SMEi phase 1 feasibility study, we investigated the technical validity, the market potential and competitor landscape of our primary lead candidate targeting TDP-43 and our antibody in AD. Moreover, we established partners for preclinical development and validated our licensing strategy. We have consolidated these results in a business plan to support future funding activities.
