WATERTOWN, Mass., July 26, 2021 (GLOBE NEWSWIRE) — Selecta Biosciences, Inc. (NASDAQ: SELB), a biotechnology company leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses, today announced the appointment of world renowned AAV gene therapy pioneer Jude Samulski, Ph.D., as a special advisor to assist with the development of their gene therapy programs. 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 (MMA) in the journal Molecular Therapy Methods & Clinical Development. The publication, entitled, “ImmTOR nanoparticles enhance AAV-driven transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia,” was led by Petr Ilyinskii, Ph.D., a senior director at Selecta.
“Overcoming immunogenicity is a huge challenge for the entire gene therapy field,” said Dr. Samulski. “The preclinical data presented in this study supports Selecta’s ImmTOR technology potential to be a game-changing technology. Ultimately this could directly translate into improved safety and efficacy for patients.”
Dr. Samulski is professor of pharmacology and has been the director of the University of North Carolina Gene Therapy Center for over two decades. He was awarded the first patent for AAV as a viral vector and was the first recipient of the American Society of Gene & Cell Therapy Outstanding Achievement Award for lifetime achievements in gene therapy. Dr. Samulski has advanced gene therapies into human clinical trials for hemophilia, Duchenne muscular dystrophy, giant axonal neuropathy, Pompe disease and heart failure, and is the president, chief scientific officer and co-founder of Asklepios BioPharmaceutical Inc. (AskBio), a biotechnology company focused on AAV-driven gene therapy.
Carsten Brunn, Ph.D., president and chief executive officer of Selecta, added, “A major barrier to current efforts in AAV-driven gene therapy is the inability to re-dose patients due to the generation of neutralizing antibodies formed against the vector after the initial dose. The data outlined in this publication demonstrate that ImmTOR, when co-administered with AAV vectors, can both enhance transgene expression and provide specific suppression of the adaptive immune response to AAV vectors to allow redosing. These data are of particular interest to our MMA program, and we look forward to building on these findings as we advance our lead candidate, MMA-101, into the clinic and expect to file an IND by the end of 2021.”
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.
About MMA
MMA is a rare metabolic disease that affects the body’s ability to metabolize certain amino acids and fats. The condition may lead to metabolic acidosis, hyperammonemia and long-term complications including feeding problems, developmental delays, intellectual disability and chronic kidney disease.
About Selecta Biosciences, Inc.
Selecta Biosciences Inc. (NASDAQ: SELB) is a clinical stage biotechnology company leveraging its ImmTOR™ platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses. With a proven ability to induce tolerance to highly immunogenic proteins, ImmTOR has the potential to amplify the efficacy of biologic therapies, including redosing of life-saving gene therapies, as well as restore the body’s natural self-tolerance in autoimmune diseases. Selecta has several proprietary and partnered programs in its pipeline focused on enzyme therapies, gene therapies, and autoimmune diseases. Selecta Biosciences is headquartered in the Greater Boston area. For more information, please visit www.selectabio.com.
