Selecta Biosciences Makes Two Major Announcements

Selecta Biosciences, Inc. recently announced that, under the terms of an existing strategic global collaboration, Sanofi has exercised its option to an exclusive license to develop an immunotherapy for the treatment of celiac disease.

In celiac disease patients, the consumption of gluten-containing food induces harmful immune responses that can lead to abdominal pain and, in most severe cases, intestinal cancer. This new immune tolerance program expands activities within the Sanofi-Selecta collaboration, which is already successfully advancing a novel immunotherapy for a life-threatening food allergy. The products resulting from this collaboration will leverage Selecta’s proprietary Synthetic Vaccine Particle (SVP) platform, which has unique capabilities to engineer nanoparticles with the structure and composition to produce immune tolerance by attenuating the overactive response to specific antigens.

“Sanofi and Selecta are working together to push toward the outer barriers of immunotherapy to deliver innovative solutions to patients. This area is constantly evolving, and with partners like Selecta, breakthrough medicines may be within our grasp,” said Kurt Stoeckli, Vice President and Head of Biotherapeutics, Research & Development at Sanofi.

Under the terms of the collaboration, Selecta is eligible to receive research support and several preclinical, clinical, regulatory, and sales milestones totaling up to $300 million for this new program in celiac disease. Additionally, Selecta is also entitled to up to double-digit tiered royalties as percentage of product net sales for any commercialized immunotherapy resulting from these efforts with Sanofi.

“We are very pleased that Sanofi and Selecta are now collaborating on three programs for immune tolerance,” said Werner Cautreels, PhD, Selecta’s President and CEO. “Both Sanofi and Selecta recognize the tremendous unmet medical needs in addressing the adverse immune responses leading to allergies and autoimmune diseases.”

In November 2012, Selecta announced that they had formed a strategic global collaboration to discover highly targeted, antigen-specific immunotherapies for life-threatening allergies. Under the agreement, 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. With the exercise of this option by Sanofi, Selecta and Sanofi now have two initiatives actively advancing immune tolerance treatments under the terms of the 2012 agreement. In October 2014, Selecta and JDRF announced another collaboration with Sanofi to research novel antigen-specific immune therapies for Type 1 Diabetes.

Selecta Biosciences and Genethon also announced an ongoing research collaboration with the goal of enabling repeat dosing for gene therapies. Based on preliminary results, the companies have identified three applications that might benefit from combining Genethon’s expertise in the development of gene therapy vectors and Selecta’s Synthetic Vaccine Particle (SVP) platform to prevent undesired immune responses. The companies plan to co-develop and co-own these next-generation gene therapies, each with the potential to meet significant unmet patient needs.

Under the terms of the proposed collaboration, Selecta and Genethon will apply Selecta’s SVP platform in an effort to eliminate the neutralizing antibodies and other undesired immune responses to the viral vector used in gene therapy. The combination of Genethon’s novel gene therapies with Selecta’s proprietary SVP would, for the first time, allow repeated systemic dosing of gene therapy vectors. Selecta’s SVP platform has unique capabilities to engineer nanoparticles with the structure and composition to produce targeted immune tolerance by attenuating the undesired immune response specifically to viral vectors. Using SVP offers the potential to expand the therapeutic range for gene therapies by maintaining the efficacy of the gene therapy over several doses. This is of particular interest in children, where organs that produce the gene therapy products are growing, and in applications where high amounts of proteins need to be supplied by the gene therapy.

“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,” says Fulvio Mavilio, PhD, Scientific Director of Genethon. “I’m excited by the tremendous potential of combining Genethon’s gene therapies with Selecta’s novel SVP platform.”

“Through this collaboration with Genethon, we are pioneering new ground, aiming to enable, for the first time, the ability to allow repeated systemic dosing of gene therapy vectors that can open many new disease applications for patients, particularly children,” said Peter Keller, Selecta’s Chief Business Officer. “Our collaboration with Genethon, a leader in gene therapies, is a good example of the value that antigen-specific SVP immunotherapies can create in novel classes of biologics and co-development deal structures could serve as a template for other SVP applications in such biologic therapies.”

Genethon and Selecta will initially focus their collaborative research and co-development efforts on gene therapies in development for muscular dystrophies and pediatric liver metabolic diseases that employ adeno-associated virus (AAV) vectors, which are a gene transfer platform of choice for many in vivo therapy applications. In recent years, the field of in vivo gene transfer with AAV vectors has seen a dramatic expansion in the number of indications approaching or in clinical testing, including promising results obtained in clinical trials of AAV vector-mediated gene therapy for hemophilia B, Leber’s congenital amaurosis, and others, along with the market approval of an AAV gene therapy drug for the treatment of lipoprotein lipase deficiency in Europe.

Genethon, created by the AFM-Telethon, has the mission to make innovative gene therapy treatments available to patients affected with rare genetic diseases. Having played a pioneering role in deciphering the human genome, Genethon is today, with more than 200 scientists, physicians, engineers, and regulatory affairs specialists, one of the leading organizations for the development of gene therapy treatments. For more information, visit www.genethon.com.

Selecta Biosciences, Inc. is a clinical-stage biotechnology company developing novel drugs that use immune modulating nanomedicines to generate targeted antigen-specific immune responses to prevent and treat disease. Selecta’s proprietary Synthetic Vaccine Particle (SVP) platform creates a novel paradigm in immunotherapeutics and vaccines, enabling completely new applications while offering the potential of improved efficacy and safety profiles.

Selecta’s immunomodulatory SVPs can induce antigen-specific immune tolerance, enabling them to be applied in a variety of therapeutic areas with large unmet medical need. The company is focused on three key near-term applications: inhibition of immunogenicity of biologic therapies, treatment of allergies, and treatment of autoimmune diseases. For more information, visit
www.selectabio.com.