Genetic Therapy Inc. says the National Institutes of Health has approved trials for treating Gaucher's disease, a metabolic disorder, by genetically altering a patient's blood.
The Gaithersburg-based biotechnology company received approval to remove blood from within or around a patient's bone marrow, genetically alter it, and return it to the patient in a healthy state. The treatment would use a delivery system designed by Genetic Therapy called a retroviral vector.
The company closed yesterday unchanged at $20.25 a share.
Genetic Therapy hopes that once the treated blood cells are returned to the patient, they will combat the disease by producing a continuous supply of healthy blood cells.
The human gene therapy might provide those inflicted with Gaucher's disease with a "more effective, longer-lasting and lower-cost treatment," said James Barrett, Genetic Therapy's president and chief executive.
Although approved by the NIH's DNA advisory committee, the trials cannot begin until the company receives clearance from the U.S. Food and Drug Administration, Genetic Therapy said.
The trials will be led by researchers at the National Institute of Neurological Disorders and Stroke, at the National Heart, Lung and Blood Therapy Institute, and at Children's Hospital at the University of California in Los Angeles.