Applications of gene therapy in clinical trials

History of gene therapy

CNS-directed and liver-directed gene therapies   

Transforming lives through gene therapy

Transforming lives through gene therapy

In preclinical research and in clinical trials, AAVs have also shown lasting therapeutic gene expression (for >8 years) after a single treatment.^9,12,13

Are there approved AAV gene therapies?

As of July 2020, there are two FDA-approved AAV-based gene therapies in the United States.⁴ One is for people with spinal muscular atrophy, an inherited genetic disease that affects special cells within the spinal cord.¹⁴ The other is for people with inherited retinal diseases.¹⁵ These approved therapies have shown that AAV-based gene therapy is a potentially viable platform for treating genetic diseases.