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Gene Therapy in Clinical Trials for Alpha-1 Antitrypsin Deficiency (AATD) Treatment

For the past few decades, clinical trials related to alpha-1 antitrypsin deficiency (AATD), have focused on getting more replacement therapy on the market. New areas of AATD research focus on gene therapy for lung disease and RNA interference for liver disease. These advancements in AATD treatment could lead to much better control of these diseases.

Gene therapy has been slow to earn acceptance as a safe treatment option for AATD and other illnesses, but has been “picking up steam” as of late according to Bruce Given, MD. In February of 2015, the first human was dosed with a new RNAi treatment that administrators of the clinical trials hope will prove efficacious and eventually make it to the marketplace. The trial is currently taking place in healthy adults with normal alpha-1 antitrypsin production, but plans to test in AATD patients are progressing.