Firms vie to treat genetic disease.Genzyme stands at the center of another enzyme-replacement story. The rare, fatal genetic condition in this instance is Fabry's disease Fa·bry's disease n. A sex-linked disorder of glycolipid metabolism characterized by a variety of progressive symptoms including fevers, hypertension, and purple skin lesions, with death resulting from renal, cardiac, or cerebrovascular complications. . In people with the illness, lack of the enzyme alpha-galactosidase A results in an abnormal buildup build·up also build-up n. 1. The act or process of amassing or increasing: a military buildup; a buildup of tension during the strike. 2. of certain lipids in blood vessel blood vessel n. An elastic tubular channel, such as an artery, a vein, a sinus, or a capillary, through which the blood circulates. blood vessel(s), n the network of muscular tubes that carry blood. cells. This causes episodes of severe pain and progressively damages the kidneys and heart. Genzyme scientists and collaborators from Mt. Sinai Medical Center in New York City New York City: see New York, city. New York City City (pop., 2000: 8,008,278), southeastern New York, at the mouth of the Hudson River. The largest city in the U.S. infused the missing enzyme into 29 adults with Fabry's disease. The treatment completely cleared the dangerous lipid in the hearts, kidneys, and skin of 20 patients. In all but one of the others, the treatment significantly slashed the lipid accumulation. Seeking approval for this therapy, Genzyme has submitted its data to the Food and Drug Administration. Investigators from Transkaryotic Therapies (TKT TKT Ticket TKT Transketolase TKT Twisted Kaiju Theater (web forum) TKT T Kang Taekwondo (New York) TKT Thin Kerf Technologies ) of Cambridge, Mass., and the National Institutes of Health have had similar success at treating Fabry's disease with alpha-galactosidase A. Like Genzyme, TKT has asked the FDA FDA abbr. Food and Drug Administration FDA, n.pr See Food and Drug Administration. FDA, n.pr the abbreviation for the Food and Drug Administration. for an exclusive license for the therapy. Adding to the corporate intrigue, Genzyme is suing TKT, claiming the company has infringed on its patents covering production of alpha-galactosidase A. TKT hasn't disclosed how it makes the enzyme, but the firm may be using its controversial technology known as gene activation. Most biotech firms produce a human protein by adding the gene that encodes it to animals or to cells growing in the laboratory. In contrast, TKT has developed methods to activate particular genes within human cells. With this strategy, TKT has sought to evade other companies' patents on several blockbuster drugs. |
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