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Wolman disease

Transgenic chicken eggs target rare disease

Tecno_Galinha MarromDaniel BuenoThe first transgenic chicken approved by the Food and Drug Administration (FDA) – the agency that regulates the food and drug industry in the United States – will not be used for human consumption but rather as a biofactory for producing the enzyme sebelipase alfa in its eggs. This enzyme, in the form of an injectable drug made by the pharmaceutical company Alexion, is the first treatment for lysosomal acid lipase (LAL) deficiency, a rare genetic disorder also known as Wolman disease, which, in its most severe form, can kill babies in their first six months of life. The disorder leads to the build-up of fat in the liver, spleen, and blood vessel walls. Without lysosomal acid lipase – or if it fails to work properly – a person’s ability to break down esterified cholesterol and triglycerides is seriously diminished. Wolman disease strikes once every one million births. Named Kanuma, the drug is purified from the egg whites of the transgenic hens. The FDA verified that the enzyme does not affect the health of the animals. The company will raise the hens in a secure environment and they will not enter the food supply. The FDA designated Kanuma as an orphan drug, making it eligible for such financial incentives as tax credits.