Emerging SARS-CoV-2 Mutations webinar available on-demand

There are approximately 7,000 distinct rare diseases affecting 350 million people worldwide, approximately 80 percent of which are caused by faulty genes. The US Food and Drug Administration (FDA) has more than 700 active Investigational New Drug Applications (INDs) for gene and cell therapies and in 2017, the FDA approved two cell-based gene therapies and it is anticipated that gene therapy will become a mainstay treatment for many rare diseases.

Transporters are involved in clinically significant drug-drug interactions (DDI). Such interactions can occur at multiple sites, including in organs important in drug absorption (e.g. intestine), elimination (e.g. liver and kidneys) and distribution (e.g. brain). Notably, some of these interactions can be “hidden”, occurring in a tissue with no discernible trace in the systemic circulation. In addition, for drugs that are both transported and metabolized, DDI will most prominent when they occur at the “rate-limiting step” in the clearance of the drug.

DNA defines who we are. And like anything else, sometimes there are mistakes – mutations in genes that can cause life-lasting conditions. But what if broken genes could be repaired? How many patients could we help in the years ahead? This is the promise and possibility of genome editing.

While the importance of ligand-receptor binding kinetics on drug action is becoming well established, focus to date has been almost entirely on the role of the dissociation constant on drug efficacy and duration of action. The association rate constant, in contrast, has been largely overlooked, with many investigators still considering it diffusion-limited and equivalent across a compound series. This GEN webinar aims to remedy that by turning the spotlight onto the undervalued and oft-ignored association rate constant.