| HOME >> BIOLOGY >> TECHNOLOGY |
K-1001
HQK-1001 belongs to a class of compounds originally discovered at Boston University School of Medicine and licensed to the Company. These compounds, designated as Short Chain Fatty Acid Derivatives (SCFADs), have been shown to stimulate fetal globin expression and red blood cell production in the laboratory and in small clinical trials in patients with hemoglobin disorders, including sickle cell disease and beta thalassemia. HQK-1001 is an orally administered SCFAD, which has shown an excellent safety profile and biologic effects on fetal globin induction and red blood cell production in the laboratory, relevant animal models, and in healthy human subjects in Phase 1 clinical trials. Additionally, the compound has received Orphan Drug Designation in the United States and Europe for both sickle cell disease and beta thalassemia.
ABOUT SICKLE CELL DISEASE AND BETA THALASSEMIA
Sickle cell disease is a genetic disorder affecting the beta globin chain of adult hemoglobin, which results in distorted, rigid sickle red blood cells, which block blood vessels, causing lack of oxygen to tissues, acute episodes of pain (pain crises), lung injury (acute chest syndrome), and strokes. Infections are common, and chronic damage occurs in many organs, including the spleen, bones, kidneys, lungs, brain, and eyes. The sole drug which is approved to treat the disease is a cancer chemotherapy drug, hydroxyurea. The lifespan of sickle cell patients is markedly reduced in the U.S., where there are approximately 75-80,000 patients.
Beta thalassemias are among the most common genetic blood disorders worldwide, in which patients are unable to produce normal amounts of the beta globin chain of adult hemoglobin, with consequent rapid destruction of red blood cells and their progenitors, and moderate to severe, transfusion-dependent anemia. The standard therapies are transplantation and red blood cell transfusions, which cause iron overload an
'/>"/>
| SOURCE HemaQuest Pharmaceuticals Copyright©2009 PR Newswire. All rights reserved |
Related biology technology :
1. Merrimack Pharmaceuticals Acquires Hermes Biosciences, Inc.
2. Access Pharmaceuticals Provides Update on MuGard Commercial Launch in Europe
3. VIA Pharmaceuticals Completes Patient Visits in Phase 2 Trial of VIA-2291
4. Peregrine Pharmaceuticals to Report Second Quarter FY 2010 Financial Results
5. China Yongxin Pharmaceuticals Inc. to Distribute Medicines on the Essential Drug List
6. KTA Capital Arranges an AU$6.7 Million Private Placement by Neuren Pharmaceuticals
7. Arena Pharmaceuticals to Present at the Piper Jaffray 21st Annual Health Care Conference
8. Peregrine Pharmaceuticals Presents Promising Antiviral Data at 2009 Chemical and Biological Defense Science & Technology Conference
9. Onyx Pharmaceuticals to Present at the Credit Suisse 18th Annual Healthcare Conference
10. Optimer Pharmaceuticals Reports Third Quarter 2009 Financial Results
11. ISTA Pharmaceuticals Reports Third Quarter 2009 Financial Results
| Copyright © 2003-2012 Bio-Medicine. All rights reserved. ABOUT | CONTACT US | DISCLAIMER | PRIVACY POLICY | TERMS AND CONDITIONS |  |