Selecta Biosciences Announces the Publication of Data Evaluating ImmTOR with AAV Vectors for the Treatment of Methylmalonic Acidemia
July 26, 2021 at 08:00 am EDT
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Additionally, Selecta has published a peer-reviewed online article describing pre-clinical data from its study investigating the efficacy of co-administration of ImmTOR nanoparticles with AAV vectors in transgene expression in methylmalonic acidemia in the journal Molecular TherapyMethods & Clinical Development. In the study, performed in collaboration with the National Human Genome Research Institute, researchers evaluated the therapeutic efficacy of co-administration of ImmTOR and an AAV vector in a mouse model of MMA. After the initial dose, immediate increases in transgene expression and reduction of plasma methylmalonic acid, a marker of therapeutic activity, were observed in mice treated with both ImmTOR and AAV. Repeated administration of AAV vectors enabled by ImmTOR resulted in increased vector transduction and further decreases in plasma methylmalonic acid that was dose dependent. The combination was well-tolerated and led to near complete inhibition of neutralizing antibodies to the AAV vector. These data support the use of ImmTOR in combination with AAV-driven gene therapy to mitigate the current detrimental impacts of immunogenicity to AAV, potentially enabling re-dosing and elevated transgene expression at the initial dose.
Cartesian Therapeutics, Inc. is a clinical-stage company. The Company is engaged in developing messenger ribonucleic acids (mRNA) cell therapies for the treatment of autoimmune diseases. It is leveraging its proprietary technology and manufacturing platform, RNA Armory, to develop mRNA cell therapies for autoimmune diseases. Its lead asset e, Descartes-08, is an autologous mRNA chimeric antigen receptor T-cell therapy (CAR-T) directed against the B cell maturation antigen (BCMA), that the Company is developing for the treatment of autoimmune diseases. Descartes-08 is in Phase II clinical development. Descartes-15 is a next-generation, autologous anti-BCMA mRNA CAR-T. Using its proprietary technology and manufacturing platform, it designed Descartes-15 to be more resistant than Descartes-08 to recycling of the CAR upon multiple antigen exposures. It is developing Descartes-33 to deliver a combination of therapeutic proteins that target key drivers in the pathogenesis of autoimmunity.