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VERTEX SIGNS EXCLUSIVE AGREEMENT TO DEVELOP COMPOUNDS FOR SICKLE CELL ANEMIA AND BETA THALASSEMIA

 CAMBRIDGE, Mass., Aug. 11 /PRNewswire/ -- Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) announced today that the company has acquired exclusive worldwide rights from the Children's Hospital Oakland (CHO) to develop, manufacture and market compounds to treat hemoglobin disorders such as sickle cell anemia and beta thalassemia.
 Sickle cell anemia and beta thalassemia (including "Cooley's anemia") are caused by genetic defects that produce a defective or incomplete adult form of hemoglobin, the oxygen-carrying protein in the blood. The compounds licensed by Vertex comprise a class of small, organic compounds which increase the level of healthy, fetal hemoglobin in sickle cell anemia and beta thalassemia patients. The compounds are believed to act as transcriptional activators to reactivate a dormant gene for production of fetal hemoglobin, a gene which is normally turned on during fetal development and during the first six months of life, but which is usually turned off after infancy. Reactivating this natural but dormant gene corrects many of the effects of these genetic diseases. One of the compounds has demonstrated significant potential as a treatment for hemoglobin disorders in preliminary clinical studies conducted at a CHO unit, the Children's Hospital Oakland Research Institute (CHORI), and these preliminary clinical results were published in the Jan. 14, 1993 issue of the "New England Journal of Medicine."
 "The agreement provides an opportunity for Vertex to develop products with demonstrated clinical promise in an area where there is an important medical need," said Dr. Joshua Boger, president and chief executive officer of Vertex. "Currently, there are no truly effective drug treatments for sickle cell anemia or beta thalassemia, both of which are potentially life-threatening disorders. We expect to initiate a clinical program expeditiously, expanding on preliminary animal and human studies which have already been conducted by CHORI researchers." Dr. Boger added that compounds affecting transcription have been the subject of Vertex's research efforts in other fields, and new approaches to transcriptional control will have applications in other diseases.
 In the 1993 "New England Journal" article, CHORI researchers reported in a short-term study in which intravenous administration of one of the licensed compounds reactivated the fetal hemoglobin gene, resulting in increases in fetal hemoglobin levels. The compound was well tolerated and safe. A separate 1991 study published in the "New England Journal" had established an important link between increased fetal hemoglobin and improvement in patients' clinical condition. Vertex intends to initially pursue development of two compounds, one that will be administered intravenously and another that will be administered orally. There are an estimated 60,000 sickle cell anemia patients and 10,000 beta thalassemia patients in the United States.
 Vertex is a leader in the use of structure-based rational drug design, a scientific approach to drug discovery which uses atomic level information to design synthetic compounds that interact with protein targets involved in disease processes. Vertex is using advanced techniques of biology, chemistry and biophysics to create novel, small molecule compounds that the Company plans to develop as orally deliverable drugs to treat major human diseases. Vertex is concentrating on the development of drugs in the areas of immunology, cancer, inflammation and antiviral therapy.
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 /CONTACT: Stephanie K. Marrus, director of Corporate Communications, Vertex Pharmaceutical, 617-499-2490; Robert Gottlieb, senior vice president, Feinstein Partners Inc., 617-577-8110; Steve Tiger, Children's Hospital Oakland, 510-428-3367/
 (VRTX)


CO: Vertex Pharmaceuticals Incorporated; Children's Hospital Oakland ST: Massachusetts IN: MTC SU:

DJ -- NE002 -- 1488 08/11/93 08:00 EDT
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Publication:PR Newswire
Date:Aug 11, 1993
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