Skip to main content
Premium Trial:

Request an Annual Quote

Alnylam Adds AAT Deficiency Drug to Pipeline


NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals announced this week that it has formally added ALN-AAT, a subcutaneously delivered therapy for alpha-1 antitrypsin (AAT) deficiency-associated liver disease, to its drug-development pipeline.

AAT deficiency-associated liver disease is caused by accumulation of mutant AAT protein in liver tissue with subsequent liver injury, fibrosis, cirrhosis, and potentially hepatocellular carcinoma, according to the company.

Recently released preclinical data showed that ALN-AAT triggered rapid, potent, dose-dependent, and durable knockdown of mutant AAT protein in the livers of transgenic mouse models, while improving liver histopathology. Treatment also resulted in significant improvements in liver pathology and function, along with reductions in the incidence of liver tumors.

Alnylam said that it anticipates filing an investigational new drug application for ALN-AAT in mid-2015. The drug is one of the company's genetic medicines programs and covered by its recently announced partnership with Genzyme.

The Scan

Positive Framing of Genetic Studies Can Spark Mistrust Among Underrepresented Groups

Researchers in Human Genetics and Genomics Advances report that how researchers describe genomic studies may alienate potential participants.

Small Study of Gene Editing to Treat Sickle Cell Disease

In a Novartis-sponsored study in the New England Journal of Medicine, researchers found that a CRISPR-Cas9-based treatment targeting promoters of genes encoding fetal hemoglobin could reduce disease symptoms.

Gut Microbiome Changes Appear in Infants Before They Develop Eczema, Study Finds

Researchers report in mSystems that infants experienced an enrichment in Clostridium sensu stricto 1 and Finegoldia and a depletion of Bacteroides before developing eczema.

Acute Myeloid Leukemia Treatment Specificity Enhanced With Stem Cell Editing

A study in Nature suggests epitope editing in donor stem cells prior to bone marrow transplants can stave off toxicity when targeting acute myeloid leukemia with immunotherapy.