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Partnership focused on Anc80-AAV-based gene therapy for treatment of methylmalonic acidemia
February 16, 2017
By: Tim Wright
Editor-in-Chief, Contract Pharma
Lonza Houston, Inc. a provider of viral gene and cell therapy manufacturing, and Selecta Biosciences, a clinical-stage biopharma focused on developing biologic therapies for rare and serious diseases that avoid unwanted immunogenicity, have entered into a manufacturing agreement. Lonza will produce an Anc80-AAV-based gene therapy product for Selecta’s proprietary program for the treatment of methylmalonic acidemia (MMA), a rare inborn error of metabolism, and may in the future produce other Anc80-based products for which Selecta holds exclusive options. The partnership will leverage Lonza’s expertise in the development of robust and industry-scale manufacturing platforms for viral-based products. Data shows that Anc80-AAV, an in silico-designed synthetic gene therapy vector, has the potential to provide superior gene expression levels in retina, liver, muscle, cochlea’s outer hair cells and other tissue targets in preclinical studies, as well as reduced cross-reactivity as compared to naturally occurring adeno-associated viral vectors (AAVs) that are currently in clinical development. “This agreement with Selecta Biosciences continues to demonstrate Lonza’s leadership position in the cell and gene therapy space,” said Andreas Weiler, head of emerging technologies business unit for Lonza’s pharma and biotech segment. “Lonza will utilize our extensive cGMP manufacturing knowledge and world-class quality systems to help Selecta Biosciences develop promising novel therapeutics for patients impacted by MMA and other devastating diseases.” Werner Cautreels, president and chief executive officer, Selecta, said, “ “We at Selecta are focused on combining novel and proprietary viral vectors with our immune tolerance Synthetic Vaccine Particles (SVP) to enable the first non-immunogenic gene therapies, providing the potential for repeat dosing. We view Lonza—one of the industry’s largest contract manufacturers of biologics and a leading supplier in gene therapy—as an ideal partner. They already have invested in developing various expression technologies, and they share our excitement about Anc80. We look forward to working with them to bring the first Anc80-based program into the clinic as a potential treatment for patients afflicted with MMA.”
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