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February 16, 2017

Lonza to Manufacture Selecta Gene Therapy Candidate for MMA

  • Lonza’s viral-based therapeutics unit Lonza Houston has agreed to manufacture an Anc80-AAV–based gene therapy product for Selecta Bioscience’s proprietary program for the treatment of methylmalonic acidemia (MMA) and may produce other Anc80-based products for which Selecta holds exclusive options, the companies said today.

    The companies said their strategic manufacturing agreement—whose value was not disclosed—will apply to Selecta’s program Lonza’s expertise in developing robust and industry-scale manufacturing platforms for viral-based products.

    “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,” Andreas Weiler, Ph.D., head of the Emerging Technologies Business Unit for Lonza’s Pharma & Biotech segment, said in a statement.

    Anc80-AAV, an in silico-designed synthetic gene therapy vector, has generated preclinical data suggesting its potential to provide what the companies termed superior gene expression levels in the retina, liver, muscle, cochlea’s outer hair cells, and other tissue targets. Anc80-AAV has also shown reduced cross-reactivity compared to naturally occurring adeno-associated viral vectors (AAVs) now in clinical development, Lonza and Selecta said.

    Selecta exclusively licensed Anc80 for MMA from Massachusetts Eye and Ear® (MEE) in May 2016. The vector was developed by the laboratory of Luk H. Vandenberghe, Ph.D., director of MEE’s Grousbeck Gene Therapy Center and an assistant professor at Harvard Medical School. Under the license agreement, whose value was not disclosed, Selecta also has the exclusive option to develop gene therapies using Anc80 for additional predefined lysosomal storage, genetic muscular, and genetic metabolic diseases.

    Selecta focuses on combining novel and proprietary viral vectors with its immune tolerance Synthetic Vaccine Particles (SVP™) to enable the first nonimmunogenic gene therapies, providing the potential for repeat dosing.

    Selecta said it intends to combine Anc80 with recently discovered transgenes and Selecta’s SVP-Rapamycin to create a novel gene therapy candidate for MMA. The candidate will be designed to enable treatment of patients with and without pre-existing anti-AAV antibodies, prevent cellular immune responses that often reduce the expression levels of gene therapies, and provide the ability to administer repeat gene therapy doses to achieve sufficient levels of methylmalonyl-CoA mutase, the enzyme that MMA patients lack.

    To advance the MMA program, Selecta last year entered into a Collaborative Research and Development Agreement (CRADA) with MEE and the NIH’s National Human Genome Research Institute. The CRADA’s principal investigators are Dr. Vandenberghe and Charles Venditti, M.D., Ph.D., senior investigator and head, Organic Acid Research Section, Medical Genomics and Metabolic Genetics Branch.

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