Selecta Biosciences, Inc., a clinical-stage biopharmaceutical company developing targeted antigen-specific immune therapies, announced today that it obtained an exclusive license to Anc80, an in silico designed gene therapy vector, from Massachusetts Eye and Ear. Under the agreement, Selecta secures an exclusive license to the Anc80 technology for a rare genetic disease and has options for additional pre-defined indications in the areas of lysosomal storage diseases, genetic muscular diseases and genetic metabolic diseases. Anc80 is developed by the laboratory of Luk H. Vandenberghe, PhD, Director of the Grousbeck Gene Therapy Center at Mass. Eye and Ear and an Assistant Professor at Harvard Medical School. Anc80 has demonstrated the potential to provide superior gene expression levels in the liver compared to naturally-occurring adeno-associated viral vectors (AAVs) that are currently in clinical development. Anc80, a next generation engineered gene therapy vector, is synthetic in nature and has been shown to have reduced cross-reactivity with commonly used AAV vectors.
By combining Anc80 with Selecta’s investigational drug candidate SVP-Rapamycin (SEL-110), Selecta is seeking to advance a new gene therapy platform designed to avoid immunogenicity that restricts the application of gene augmentation, replacement and editing therapies. Gene therapy vectors are derived from viruses and are designed to insert a therapeutic transgene into the nucleus of targeted cells. Undesired immunogenicity can manifest itself in three ways. First, pre-existing neutralizing anti-drug antibodies (ADAs) induced following a natural AAV infection can block gene transfer. Today up to 50 percent of patients are ineligible for gene therapy due to the presence of pre-existing ADAs. Second, ADAs form in response to a first administration of a gene therapy vector and prevent effective subsequent doses of gene therapy. The ability to re-administer is important for therapeutic targets that undergo renewal which is the case in many pediatric indications. Lastly, cellular immune responses against the transduced cells can reduce efficacy and pose safety concerns. Selecta intends to combine Anc80 with SVP-Rapamycin (SEL-110), a proprietary immunomodulatory therapeutic, with the objective to address all three of these immunogenicity issues related to gene therapies.
“The combination of Anc80 with SVP-Rapamycin is very promising,” said Dr. Vandenberghe. “Anc80 is a potent gene therapy vector that is not known to circulate in humans, making it less likely to cross-react immunologically with naturally occurring AAVs. Selecta’s SVP-Rapamycin has shown the potential to mitigate the immune responses that are connected with the administration of viral vectors. The combination of Anc80 with SVP-Rapamycin offers the exciting prospect of broadening the potential of gene therapy in both the proportion of patients it can reach and the number of diseases amenable to this emerging therapeutic modality in medicine.”
“This agreement provides Selecta with the possibility of developing several proprietary gene therapies using a novel and differentiated vector,” said Werner Cautreels, PhD, CEO and President of Selecta. “The agreement with Massachusetts Eye and Ear is a cornerstone in our strategy to build a pipeline of proprietary products to treat rare and serious diseases by mitigating the immune responses that would otherwise compromise these therapies. In addition to gene therapy, our efforts are focused on the mitigation of deleterious immune responses to otherwise highly effective therapeutic enzymes and proteins.”