In May, the FDA approved onasemnogene abeparvovec (Zolgensma) for treating spinal muscular atrophy in children under two years of age. The list price of Zolgensma was set at  per dose, making it the most expensive drug ever.
In May, the EMA approved betibeglogene autotemcel (Zynteglo) for treating beta thalassemia for people twelve years of age and older.
In July, Allergan and Editas Medicine announced phase I/II clinical trial of AGN-151587 for the treatment of Leber congenital amaurosis 10. This is the first study of a CRISPR-based in vivo human gene editing therapy, where the editing takes place inside the human body. The first injection of the CRISPR-Cas System was confirmed in March 2020.