Selecta Biosciences (SELB) Announces Presentation of Encouraging Preclinical Data on SVP-Rapamycin in Gene Therapy

October 20, 2016 8:11 AM EDT
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Selecta Biosciences, Inc. (Nasdaq: SELB) announced that encouraging results from preclinical studies conducted in collaboration with Federico Mingozzi, Ph.D., Head of Immunology and Liver Gene Therapy at Genethon in Evry, France were presented at the Annual Congress of the European Society of Gene and Cell Therapy in Florence, Italy in a poster titled, “Antigen-specific modulation of capsid immunogenicity with tolerogenic nanoparticles results in successful AAV vector re-administration”. The new preclinical data elucidate the mechanism by which SVP-Rapamycin mitigates undesired immune responses. Further, the data demonstrate therapeutic benefits of co-administration of SVP-Rapamycin with an AAV8 gene therapy vector expressing Factor IX, a coagulation protein deficient in patients with Hemophilia B.

“These newly presented results underscore the unique advantages of SVP-Rapamycin in gene therapy,” said Dr. Takashi Kei Kishimoto, Ph.D., Chief Scientific Officer of Selecta. “Based on both these preclinical data with AAV and our clinical data showing that a single dose of SVP-Rapamycin mitigates antibody responses to a highly immunogenic enzyme for more than 30 days, I believe that our approach will help to enhance the development of the field. The key advantages of using SVP-Rapamycin would be the ability to re-administer gene therapies, when protein expression levels are not sufficient, and to prevent the type of immune attacks that can reduce expression or damage transduced organs.”

SVP-Rapamycin demonstrated successful mitigation of both humoral (antibody) and cellular immune responses that are associated with gene therapy using adeno-associated viral (AAV) vectors. Antibodies against AAV develop with the first dose of gene therapy and can prevent re-administration of therapy, which may be important in pediatric applications and diseases where sufficient protein expression cannot be achieved with a single dose. Cellular immune responses observed in clinical trials of gene therapies have been associated with an increase in liver enzymes and a loss of transgene expression in patients. Further, the data presented demonstrate that SVP-Rapamycin mitigates immunogenicity to gene therapy via the induction of T regulatory cells and prevention of germinal center formation.

Selecta is advancing gene therapy programs for two rare genetic metabolic disorders, Methylmalonic Acidemia (MMA) and Ornithine Transcarbamylase Deficiency (OTC). For both of these gene therapy applications, SVP-Rapamycin is designed to enable treatment of patients in childhood before the occurrence of disease-associated developmental and neurological issues. The prevention of antibodies and cellular immune responses by SVP-Rapamycin should allow for multiple doses of gene therapies in order to maintain beneficial therapeutic protein expression as the patients age.

Selecta’s lead product candidate, SEL-212, applies SVP-Rapamycin to pegsiticase, a pegylated uricase. SEL-212 is designed to be the first non-immunogenic version of uricase, an immunogenic enzyme which targets uric acid. SEL-212 demonstrated clinical activity in a Phase 1b clinical trial and is being developed for patients with chronic refractory and chronic tophaceous gout.

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