Selecta Biosciences today said it was expanding its three-year-old alliance with Sanofi focused on developing antigen-specific immunotherapies—with the potential to receive up to $300 million—and launching a separate ongoing research collaboration with Genethon aimed at enabling repeat dosing for gene therapies.
Selecta said Sanofi had exercised its option for an exclusive license to co-develop an immunotherapy for the treatment of celiac disease. The immunotherapy will be the second therapeutic program co-developed by the companies that will use Selecta’s Synthetic Vaccine Particle (SVP™) platform; the first is a new immunotherapy for an undisclosed “life-threatening” food allergy, and is in the pre-clinical phase.
SVP is designed to engineer nanoparticles with the structure and composition to produce immune tolerance by attenuating the overactive response to specific antigens. Selecta has identified three key near-term applications for the platform: inhibition of immunogenicity of biologic therapies, treatment of allergies, and treatment of autoimmune diseases.
SVP will also be deployed by Selecta and Genethon under their new gene therapy partnership, designed to eliminate the neutralizing antibodies and other undesired immune responses to the viral vector used in gene therapy. The partners plan to combine the platform with Genethon’s expertise in developing gene therapy vectors.
Based on preliminary results, the companies said they have identified three applications that might benefit from the combination—which they said would, for the first time, allow repeated systemic dosing of gene therapy vectors. Repeat dosing could prove particular helpful, according to Selecta and Genethon, in gene therapies for children, since organs that produce the gene therapy products are growing, and in gene therapies where high amounts of proteins are required.
“Gene therapies that can be applied repeatedly would exponentially increase the number of highly beneficial applications of gene therapies including muscular dystrophies and pediatric liver metabolic diseases,” Fulvio Mavilio, Ph.D., Genethon’s scientific director, said in a statement.
Genethon and Selecta said they will initially focus their collaborative research and co-development efforts on gene therapies for muscular dystrophies and pediatric liver metabolic diseases that employ adeno-associated virus (AAV) vectors.
Selecta said it is eligible to receive research support, as well as payments of up to $300 million for the celiac disease program. Payments will be tied to preclinical, clinical, regulatory, and sales milestones. Selecta is also entitled to up to double digit tiered royalties as percentage of product net sales for any commercialized immunotherapy co-developed through the immune tolerance collaboration with Sanofi.
Selecta and Sanofi launched their up-to-$900 million strategic global collaboration in 2012, with the goal of discovering highly-targeted, antigen-specific immunotherapies for life threatening allergies. Sanofi obtained a first exclusive license to develop an immunotherapy designed to abate acute immune responses against a life threatening food allergen and an option to develop two additional candidate immunotherapies for allergies and celiac disease.
Separately, in October 2014, Selecta and JDRF announced a collaboration with Sanofi to research new antigen-specific immune therapies for type 1 diabetes.