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Alnylam Presents Preclinical Data on AAT Deficiency Drug


Alnylam Pharmaceuticals this week released new preclinical data from its alpha-1 antitrypsin deficiency program, which aims to treat liver disease associated with the rare genetic condition.

According to the company, it has generated data showing that it can achieve “robust RNAi-mediated silencing of AAT liver mRNA and serum protein in a transgenic mouse model of mutant AAT protein over-expression.”

The data were presented at the Annual Meeting of the American Association for the Study of Liver Diseases in Boston this month.

“Approximately 10,000 people worldwide have been diagnosed with severe AAT deficiency, a rare genetic disease that results in severe lung and liver pathology,” Saint Louis University School of Medicine researcher and Alnylam collaborator Jeffrey Teckman said in a statement. “AAT patients can develop early onset emphysema, as well as liver disease, including hepatitis, cirrhosis, and hepatocellular carcinoma. While lung disease associated with AAT deficiency has been addressed with AAT replacement therapy, there are limited treatment options for patients with liver disease.”

Specifically, Alnylam said that AAT-targeting siRNAs formulated in lipid nanoparticles at doses ranging from 0.03 mg/kg to 1 mg/kg could trigger up to a 90 percent knockdown of target mRNA in the liver and a greater than 80 percent decrease in serum AAT in the mice 48 hours after treatment.

“Furthermore, a 90 percent reduction in soluble protein monomers in the liver was observed at 1.0 mg/kg, with an 80 percent reduction seen at 0.3 mg/kg,” Alnylam said.

The company noted that it has identified a GalNAc conjugate-formulated siRNA targeting AAT that enables subcutaneous dose administration for further development.

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