U.S. Patent Granted to Amorfix for ALS Therapies Targeting Misfolded SOD1

Mutations in the gene encoding superoxide dismutase (SOD1) account for approximately 20% of familial ALS cases. The disease-causing mutations in SOD1 increase the protein’s propensity to misfold and aggregate, although it is not completely understood how exactly the mutant forms cause disease. Work from Neil Cashman’s laboratory has shown that SOD1 mutations can affect wild-type SOD1 and trigger misfolding of the native protein, as well as transfer the misfolded state from cell to cell (see Oct 2011 News and Dec 2012 News). The company founded by Dr. Cashman, Amorfix Life Sciences Ltd, has now announced that it has been granted broad patent protection for antibody targeting misfolded SOD1 for treating ALS. Amorfix has previously entered into a licensing deal with Biogen-Idec for developing these monoclonal antibodies to treat ALS and these are currently in preclinical development.

Mutations in the gene encoding superoxide dismutase (SOD1) account for approximately 20% of familial ALS cases. The disease-causing mutations in SOD1 increase the protein’s propensity to misfold and aggregate, although it is not completely understood how exactly the mutant forms cause disease. Work from Neil Cashman’s laboratory has shown that SOD1 mutations can affect wild-type SOD1 and trigger misfolding of the native protein, as well as transfer the misfolded state from cell to cell (see Oct 2011 News and Dec 2012 News). The company founded by Dr. Cashman, Amorfix Life Sciences Ltd, has now announced that it has been granted broad patent protection for antibody targeting misfolded SOD1 for treating ALS. Amorfix has previously entered into a licensing deal with Biogen-Idec for developing these monoclonal antibodies to treat ALS and these are currently in preclinical development.

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