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Isis Selects Development Candidate, Isis-AATRx, to Treat AATD-Associated Liver Disease
Date:10/11/2011

therapies for patients with AATD-associated liver disease, and liver transplantation is the only available option for patients who develop severe liver dysfunction.  Symptoms of AATD-associated liver disease can manifest as early as infancy, and it is the most common genetic disease for which pediatric liver transplantation is performed.  It is estimated that 10-15 percent of all liver transplant candidates have AATD.  

"AATD is an under-diagnosed disease that affects the lungs and, in some patients, can also lead to severe liver damage, end-stage liver disease and liver cancer.  Unfortunately, patients with AATD-associated liver disease currently have no therapeutic options and are only treated to alleviate the symptoms associated with liver damage and scarring.  In cases of severe liver damage, liver transplantation is the only option," said Jeff Teckman, M.D., Professor of Pediatrics and Biochemistry and Director of Pediatric Gastroenterology and Hepatology at Saint Louis University.  "We are encouraged by this therapeutic approach, which could, for the first time, offer an effective and selective treatment for patients with liver disease due to AATD."

In 2010, Isis entered into a strategic alliance with GSK to develop RNA therapeutics for rare and infectious diseases.  With the selection of ISIS-AATRx, Isis has earned $53 million in upfront fees and milestone payments and is eligible to receive license fees and milestone payments, totaling nearly $1.5 billion, in the event all six programs are successfully developed for one or more indications and commercialized through to pre-agreed sales targets.  

ABOUT AATD and ISIS-AATRx

AATD is a genetic disorder in which the patient does not produce normal AAT, a protein primarily produced in the liver that protects lung tissue from damage.  Patients with AATD inherit a mutant gene from one or both parents to produce misfolded AAT protein, wh
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SOURCE Isis Pharmaceuticals, Inc.
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