A novel therapeutic biologic for treatment of autoimmunity

Many autoimmune diseases are characterized by constitutive production of autoreactive antibodies that drive the pathogenesis. These are life-long diseases without known cure, and current treatment options include injection of large doses of steroids or pooled antibodies. Thus, there is an urgent need for safer and more specific therapies. Our laboratory is at the international forefront of studies on how antibodies are transported within and across cells, and we have used this in-depth knowledge to develop a specific lead therapeutic candidate. The candidate is a biologic that induces rapid degradation and clearance of antibodies in a state-of-the-art humanized mouse model. Very encouraging data have strongly motivated us to further develop and test the candidate as part of his project. In this PoC project, we have carried out extensive work where the candidate has been humanized and several formats have been tested in biochemical and cellular experiments, as well as in a leading mouse model that have confirmed the effect of the candidate. We have also mapped the development pipeline of all similar strategies, which is growing rapidly. This confirms the interest and the need for new strategies to treat a number of autoimmune diseases. We have made a robust plan for essential work that must be in place to make commercialization likely, and will apply for funding to reach this level of maturity. A plan to secure intellectual property rights has been made.

Participants have gained knowledge about state-of-the-art methodologies and learn how to translate research results into product candidates, together with a subcontractor and Inven2. They have interacted with experts and stakeholders, and, as such, learned by example. This has broadened the understanding of the PoC process and strengthen the interactions between parties. Participants have gained new skills regarding the different steps included in the project, as for instance mapping of the product development pipeline and the market. As almost 3% of the global population has an IgG-driven autoimmune disease it affects the society at large. The product that is under development may have great social value as it will be specific and lower side effects and symptoms with less need for hospitalization and assistant from heath care workers. The PIs team has established routines on how to interact with industrial partners on projects involving confidentiality. The project has been successful, where a humanized candidate has been developed that shows preferred effect in a state-of-the-art mouse model. The robust data obtained strongly motivate the team to take the next steps toward commercialization of the humanized candidate.