Codexis to develop enzyme therapeutic candidate for potential treatment of phenylketonuria (PKU)

Published: 4-Dec-2014

Preclinical studies will start next year

US biocatalysts specialist Codexis has developed a novel enzyme therapeutic product candidate for the potential treatment of phenylketonuria (PKU) through oral administration.

PKU is an inherited metabolic disorder in which the enzyme that converts the essential amino acid phenylalanine into tyrosine is deficient. As a result, phenylalanine accumulates in high levels in the brain causing serious neurological problems, including intellectual disability, seizures and cognitive and behavioural problems. Phenylalanine is found in meat, dairy products, fish, poultry and many fruits and vegetables.

Codexis has conducted studies in a PKU animal model that demonstrate proof of concept for the enzyme product candidate. In these studies, the product candidate was introduced into the stomach of the animal resulting in decreased blood phenylalanine levels.

The company has filed patent applications covering the composition of matter for its therapeutic enzymes and the use of these enzymes as a treatment for PKU.

The company is also seeking partners for its PKU programme to advance its development.

Preclinical studies are expected to begin next year.

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