- In January 2024, CRISPR Therapeutics AG and Vertex Pharmaceuticals announced the U.S. FDA approval of Casgevy (exagamglogene autotemcel), marking the first-ever approval of a CRISPR-based gene-editing therapy for the treatment of sickle cell disease and transfusion-dependent β-thalassemia. This milestone signifies a transformative breakthrough in genetic medicine, offering a one-time curative approach for patients with severe genetic blood disorders
- In March 2024, Intellia Therapeutics and its partner Regeneron Pharmaceuticals reported promising Phase 1 clinical trial data for NTLA-2002, a CRISPR/Cas9-based in vivo gene-editing therapy aimed at treating hereditary angioedema (HAE). The early data revealed substantial reductions in HAE attacks, suggesting a potential for durable, single-dose treatment in a traditionally underserved therapeutic area
- In April 2024, Editas Medicine announced successful in vivo preclinical results of its CRISPR-edited cell therapy for rhabdomyosarcoma, a rare and aggressive pediatric cancer. The company’s lead candidate demonstrated targeted tumor regression and minimal off-target effects, advancing Editas’ oncology pipeline toward clinical trials
- In February 2024, Synthego, a U.S.-based genome engineering company, launched its Automated Cell Engineering Platform, which leverages AI and CRISPR to streamline and scale gene-editing workflows for both research and therapeutic development. This innovation aims to reduce CRISPR experiment timelines by up to 50%, accelerating the path to discovery and commercialization
- In November 2023, Beam Therapeutics received IND (Investigational New Drug) clearance from the U.S. FDA for BEAM-101, a base-editing CRISPR therapy for the treatment of sickle cell disease. Unlike traditional CRISPR approaches, BEAM-101 uses base editing technology to mimic a naturally occurring genetic variant associated with reduced disease severity, reflecting a more refined and potentially safer gene-editing strategy
- In December 2023, Caribou Biosciences reported clinical trial progress for CB-010, its lead CAR-T cell therapy candidate engineered using CRISPR genome editing. Early Phase 1 results indicated durable anti-tumor activity in patients with relapsed/refractory B cell non-Hodgkin lymphoma. This marked one of the first allogeneic CAR-T therapies to enter clinical trials using CRISPR to enhance cell persistence and reduce immune rejection
