Date: 2012-04-06
Type of information: Granting of a patent
Product name: EGT-101 - adeno-associated viral vector serotype 9 containing the human sulfamidase gene
Compound: adeno-associated viral vector serotype 9 containing the human sulfamidase gene
Therapeutic area: Rare diseases - Genetic diseases
Action mechanism: gene therapy. Adeno-associated viral vector serotype 9 containing the human sulfamidase gene is a medicine that works by delivering genes into the body (gene therapy). It is made up of a virus that contains the gene for producing the missing enzyme, N-sulfoglucosamine sulfohydrolase. The type of virus used in this medicine (adeno-associated virus) does not cause disease in humans.
When injected into the brain, the virus is expected to carry the gene mainly into the brain cells. These cells are then expected to produce the missing enzyme so that it can break down the accumulated heparan sulphate and help to relieve the symptoms of the disease.
Company: Laboratorios del Dr. Esteve (Spain)
Disease: mucopolysaccharidosis type IIIA (Sanfilippo A syndrome)
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Orphan status USA: 2011-06-01
Orphan status UE: 2011-06-21
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