Selecta Biosciences' gene therapy program is centered around leveraging the ImmTOR platform to enhance the efficacy and safety of gene therapies. This involves co-administering ImmTOR with adeno-associated virus (AAV) vectors to mitigate the formation of anti-AAV antibodies, which can limit the durability and effectiveness of gene therapies. The lead therapeutic candidate in this segment is SEL-302, currently in the preclinical stage for methylmalonic acidemia (MMA). Research and development efforts focus on optimizing ImmTOR's immunomodulatory properties and expanding its application to other genetic disorders. The goal is to enable durable gene expression and improve patient outcomes by preventing immune-mediated clearance of the therapeutic transgene.

The biologic therapies segment focuses on developing treatments for chronic refractory gout and IgA-mediated diseases. SEL-212, a lead product candidate in Phase III clinical trials, combines ImmTOR with a pegylated uricase enzyme to address chronic refractory gout. The ImmTOR component aims to prevent the formation of anti-drug antibodies (ADAs) against the uricase enzyme, thereby prolonging its therapeutic effect. Additionally, this segment includes product candidates targeting IgA nephropathy, linear IgA bullous dermatitis, IgA pemphigus, and Henoch-Schonlein purpura. Research efforts are directed towards understanding the underlying mechanisms of IgA-mediated diseases and developing targeted immunomodulatory strategies to alleviate disease symptoms and improve patient quality of life. The ImmTOR platform plays a crucial role in enhancing the tolerability and efficacy of these biologic therapies.

This segment is dedicated to developing tolerogenic therapies for autoimmune diseases, including primary biliary cholangitis (PBC) and other autoimmune conditions. The approach involves utilizing the ImmTOR platform to induce immune tolerance to specific autoantigens, thereby suppressing the autoimmune response and preventing tissue damage. Research and development activities focus on identifying relevant autoantigens in various autoimmune diseases and designing ImmTOR-based therapies that can selectively target and modulate the immune system. The goal is to develop disease-modifying treatments that can provide long-term remission and improve the lives of patients with autoimmune disorders. This segment represents a significant opportunity for Selecta Biosciences to expand its pipeline and address unmet medical needs in the field of autoimmunity.