Gene therapy has shown remarkable progress in treating various genetic diseases over the past few years. Its applications in treatment of the genetic disorders are extended beyond traditional therapies. These techniques are being explored as treatment of many genetic disorders including Duchenne muscular dystrophy, cystic fibrosis, Alzheimer’s disease, Parkinson’s disease, and Huntington disease.

Gene therapy holds significant promise as an innovative approach to treating andpotentially curing various diseases, particularly genetic disorders. We selected some key aspects of the promise of gene therapies.

In December 2023, the U.S. Food and Drug Administration approved two treatments,Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatmentof sickle cell disease (SCD) in patients 12 years and older.