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Compugen Ltd. (NASDAQ: CGEN), has announced that CGEN-15001, an Fc fusion protein drug candidate derived from a novel immune checkpoint protein discovered by Compugen, has been shown to be highly efficient in preventing the development of disease in a well-accepted animal model of autoimmune type I diabetes, known also as juvenile diabetes. The study was performed as part of an ongoing collaboration with Stephen Miller, Professor of microbiology-immunology at Northwestern University Feinberg School of Medicine.
The study utilized non-obese laboratory mice that spontaneously develop autoimmune insulin dependent diabetes. Administration of CGEN-15001 to the mice began at 10 weeks of age, prior to manifestation of any clinical symptoms of the disease, and was continued for a period of only 2 weeks. The mice were then monitored for blood glucose levels for an additional 4 months after the last treatment.
Results of the study demonstrate that treatment with CGEN-15001 prevented development of diabetes throughout the 4 months of follow-up. Animals treated with CGEN-15001 maintained normal blood glucose levels compared with untreated animals that developed high blood glucose. The sustained duration of response following a short-term treatment with CGEN-15001, also observed previously in other disease models of autoimmunity, suggests that CGEN-15001 turns off the autoimmune process and may potentially lead to restoration of immune tolerance and resolution of autoimmunity, thereby arresting disease progression.
"We have already shown through various preclinical studies that CGEN-15001 has the potential to serve as an efficacious disease modifying therapy for multiple sclerosis and rheumatoid arthritis," said Anat Cohen-Dayag, Ph.D., Compugen's President and CEO. "The results being reported today show that this Fc fusion protein also has a remarkable effect in a well-accepted disease model of type I diabetes, a severe, life-long disease with no current cure. These findings highlight the potential of CGEN-15001 to treat multiple autoimmune conditions and the promise of developing new effective immune modulating therapeutic drug candidates based on Compugen's discovery platforms."
About CGEN-15001 and Immune Checkpoints
CGEN-15001 is a novel Fc fusion protein drug candidate consisting of the fusion of an IgG Fc domain to the extracellular region of CGEN-15001T. CGEN-15001T is a novel immune checkpoint discovered by Compugen through its predictive discovery infrastructure and has been shown to have significant potential as a target for cancer immunotherapy.
Immune checkpoints are inhibitory receptors, which are crucial for the maintenance of self-tolerance (that is, the prevention of autoimmunity) and for the protection of tissues from damage when the immune system is performing its intended function of responding to pathogenic infection. Autoimmune diseases, such as type I diabetes, multiple sclerosis and rheumatoid arthritis, are mediated by self-reactive T cells that have escaped immune tolerance to self-proteins.
To date, two soluble recombinant fusion proteins, both derived from the CTLA4 immune checkpoint, have been cleared for marketing in the U.S.: Orencia for the treatment of moderate to severe rheumatoid arthritis, and Nulojix for the prophylaxis of organ rejection in adults receiving a kidney transplant.
About Type I Diabetes
Type 1 diabetes, also known as insulin-dependent or juvenile diabetes, is a chronic disease in which the pancreas produces little or no insulin, a hormone necessary for control of blood sugar levels. The disease is caused by an autoimmune process whereby self-reactive T cells specifically attack and destroy the insulin-producing cells of the pancreas. Currently, type I diabetes has no cure, and treatment consists of life-long administration of insulin. Even with insulin treatment, type I diabetes may lead to serious complications, which can include: kidney failure, blindness, nerve damage, heart attack, stroke, and pregnancy complications.
Article adapted by Medical News Today from original press release. Source:
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Compugen. "Compugen drug candidate demonstrates high effectiveness in type I diabetes animal model." Medical News Today. MediLexicon, Intl., 11 Oct. 2013. Web.
11 Dec. 2013. <http://www.medicalnewstoday.com/releases/267293>
Compugen. (2013, October 11). "Compugen drug candidate demonstrates high effectiveness in type I diabetes animal model." Medical News Today. Retrieved from
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