Advancements in CRISPR Medicine: Meeting Highlights and Global Accessibility Challenges

The Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of seven new medicines, including Casgevy, the first medicine using CRISPR/Cas9 gene-editing technology, for the treatment of transfusion dependent beta-thalassemia and severe sickle cell disease. Other recommended medicines include Skyclarys for Friedreich's ataxia, Velsipity for ulcerative colitis, and four generic medicines. The CHMP also confirmed the non-renewal of a conditional marketing authorization for Blenrep. Additionally, extensions of therapeutic indications were recommended for four medicines, and positive opinions were adopted for two medicines intended for use outside the EU. The CHMP has also recommended the suspension of marketing authorizations for certain generic medicines tested by Synapse Labs Pvt. Ltd due to irregularities in study data.