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Selecta’s SEL-201 Nanoparticles Induce Immune Tolerance in Hemophilia A Model

Selecta Biosciences, Inc., a clinical stage biotechnology company developing a novel class of targeted antigen-specific immune tolerance treatments, today announced that data from a pre-clinical proof of concept study in Factor VIII-deficient mice were presented in a poster presentation at the 2013 American Society for Hematology (ASH) International Conference in New Orleans.

SEL-201 demonstrated the ability to induce immune tolerance in a model of hemophilia A, showing that SEL-201 has the potential to serve as an adjunct therapy to avoid harmful neutralizing antibody response to Factor VIII treatment severely affecting hemophilia A patients.

The presentation was made by Dr. Aihong Zhang, an employee of The Henry M. Jackson Foundation for the Advancement of Military Medicine, Inc. (HJF) and a scientist in the Department of Medicine laboratory of David W. Scott, PhD, at the Uniformed Services University of the Health Sciences (USU). Dr. Zhang and Dr. Scott are collaborating with Selecta under a Cooperative Research and Development Agreement among HJF, USU and the company to apply Selecta’s proprietary targeted tolerogenic Synthetic Vaccine Particles (t2SVP) to Hemophilia.

“Inhibitory antibodies to coagulation factors occur in approximately 25 percent to 30 percent of hemophilia A patients, representing a major medical complication for clinicians and the affected patients,” said David W. Scott, PhD, Vice Chair for Research and Professor of Medicine at USU. “SEL-201 has the potential to become the first drug to dramatically mitigate the immunogenicity of Factor VIII, which would be a breakthrough in the treatment of patients afflicted with neutralizing antibodies.”

Researchers at USU demonstrated that Selecta’s SEL-201 nanoparticles added onto standard Factor VIII treatment prevented the formation of neutralizing antibodies and led to durable tolerance without affecting the immune response to other antigens. Specifically, SEL-201 conferred antigen-specific and lasting immune tolerance for at least 166 days after the last treatment, while untreated animals developed high levels of neutralizing antibodies. SEL-201 was found to be effective when administering the treatment concomitantly or after priming with FVIII.

Selecta Biosciences is working to translate the findings to other approved biologic drugs where undesired immune responses, such as neutralizing antibodies, anaphylaxis and injection site reactions, are major complications.

“Anti-drug antibodies are a major issue for many biologic drugs and can affect both drug safety and efficacy. We have identified a significant number of approved and experimental biologics, where antigen-specific inhibition of drug-therapy-related immunogenicity would be highly beneficial and could be enabled by Selecta’s proprietary targeted tolerogenic Synthetic Vaccine Particles,” said Kei Kishimoto, PhD, Chief Scientific Officer at Selecta Biosciences. “In certain classes of biologics, we see tremendous potential to significantly improve the standard of care provided by these important biologic therapies.”

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