The most promising triple-combination therapy, ELX/TEZ/IVA (Trikafta), has been approved by the FDA for all CF genotypes with at least one F508del copy present or a mutation in the CFTR gene that is responsive based on in vitro data, potentially covering 90% of North American CF patients, many of whom had no approved therapeutic options (https://www.fda.gov/news-events/press-announcements/fda-approves-new-breakthrough-therapy-cystic-fibrosis, (accessed on 1 January 2020)) [6,7,8,9,10,11]. The gene discussed is CFTR; the disease is cystic fibrosis.