$8.7 Billion SMA Drug Explained

Emily BurkeGene Therapy, Orphan Drugs, SMA

The Science Behind the Deal Earlier this week, news of the $8.7 billion acquisition of gene therapy company AveXis (Bannockburn, IL) by Novartis (Basel, Switzerland) made big biotech headlines. AveXis’ lead candidate, AVXS-101, is now in Phase III clinical studies for the treatment of spinal muscular atrophy (SMA). In this Weekly, we’ll take a look at the science behind these headlines by explaining exactly … Read More

Gene Therapy Cures

Emily BurkeBiologics, Gene Therapy, Genetics, Term of the Week

The Promise of Gene Therapy Unfolds In many ways, 2017 was the year of gene therapy in the United States. Patients and pharmaceutical companies celebrated the approval of not one, but three treatments for otherwise untreatable health conditions. Researchers have been working on developing safe, effective gene therapies for three decades. Early trials were plagued with safety issues. Consequently, the … Read More

Zinc Finger Nucleases

Emily BurkeGene Therapy, Genetics

CATCHING THE RIGHT BREAK How are ZFNs made? To start, zinc finger proteins (ZFP) are sequence-specific, DNA-binding proteins that activate gene expression. They are engineered to recognize unique sites within the genome. While ZFPs do not have the ability to cut DNA on their own, scientists can fuse a ZFP together with a DNA-cutting enzyme called nuclease—the “N” in ZFN. The marriage of ZFP … Read More