Techniques that bypass or trick the guardian of the central nervous system. Developing drugs that can penetrate into the brain requires some creativity. The approaches vary, each technique comes with its benefits and drawbacks, making it appropriate for some patients or drug types but not others. In some diseases, you might want the drug everywhere; […]
Gene therapies for disorders such as X-linked Severe Combined Immunodeficiency (SCID, sometimes known as “bubble boy disease”), and Spinal Muscular Atrophy (SMA) have, for the first time, shown remarkable safety and efficacy results in clinical trials. FDA approved Trikafta, a gene-based therapy creating hope for children with cystic fibrosis and Zolgensma, a gene therapy drug, […]
Proof of Mechanism (PoM) Usually in Healthy Volunteers, Phase 1 study Essential for the selection of appropriate dose for PoC, disease model and biomarkers Investigate drug concentration at the target site of action Investigate drug engagement with target molecular receptor or enzyme (e.g. Receptor binding Investigate if drug biological response after target binding match the […]
