Date: 2017-02-16
Type of information: Production agreement
Compound: Anc80-AAV-based gene therapy
Company: Lonza (Switzerland) Selecta Biosciences (USA - MA)
Therapeutic area: Rare diseases - Genetic diseases
Type agreement: manufacturing bioproduction
Action mechanism: gene therapy. Anc80 has been developed by the laboratory of Luk H. Vandenberghe, PhD, of Mass. Eye and Ear and Harvard Medical School. Anc80 is an in silico designed predicted ancestor of AAV1, AAV2, AAV8 and AAV9. In preclinical studies, Anc80 has been shown to be a potent gene therapy vector that has demonstrated the capability of yielding superior gene expression levels in the retina, liver, muscle, cochlea's outer hair cells and other tissue targets. Selecta exclusively licensed Anc80 for methylmalonic acidemia from Massachusetts Eye and Ear® (MEE) in May 2016 . Under the license agreement, Selecta also has the exclusive option to develop gene therapies using Anc80 for additional pre-defined lysosomal storage, genetic muscular and genetic metabolic diseases. Selecta intends to combine Anc80 with recently discovered transgenes and Selecta's SVP-Rapamycin to create a novel gene therapy candidate for MMA. This candidate is intended to a) enable the treatment of patients with and without pre-existing anti-AAV antibodies; b) prevent cellular immune responses that often reduce the expression levels of gene therapies; and c) provide the ability to administer repeat gene therapy doses to achieve sufficient levels of methylmalonyl-CoA mutase (MUT), the enzyme that MMA patients are lacking.
Disease: methylmalonic acidemia
Details: * On February 16, 2017, Lonza and Selecta Biosciences have entered into a strategic manufacturing agreement. Under the terms of the agreement, Lonza will produce an Anc80-AAV-based gene therapy product for Selecta's proprietary program for the treatment of methylmalonic acidemia, a rare inborn error of metabolism. The companies may in the future produce other Anc80-based products for which Selecta holds exclusive options.
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