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, […]
Drug Development
2019 Successful Clinical Plan and Drug Development Stories
January 27th, 2020 | Drug DevelopmentThe BBCR team designs Proof of Concept (PoC) Trials and Proof of Mechanism (PoM) studies with the drug clinical plan and regulatory strategy in mind.
December 19th, 2019 | Drug DevelopmentProof 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 […]