The evolution of gene therapies
The earliest experiments in the field took place in the 1960s and saw major advances in the 1980s and 1990s. Scientists have learned more about gene therapy with each new discovery. Today, gene therapies are being studied and prescribed for people living with many different conditions, in many parts of the world.
First successful transformation of human cells with genomic DNA
Investigation of adenovirus for potential use in gene therapies
First gene therapy treatment of patients with adenosine deaminase deficiency
Setback in treatment due to safety spurs development of new vectors
Development of novel AAV as gene therapy vectors
Adeno-associated virus (AAV) vectors have been used to target muscle and nerve cells, and are preferred for their safety and efficacy features.
First regulatory approval of a gene therapy product
Gene therapy shows successes in hemophilia B, Leber congenital amaurosis, and adrenoleukodystrophy
First AAV gene therapy treatment approved in the U.S. for a rare genetic eye condition
AAV gene therapy approved for spinal muscular atrophy
Continued expansion of gene therapy clinical trials
AAV gene therapy for hemophilia B approved in the U.S.
Faster progress every day
Since the arrival of the first gene therapy back in 2003, the gene therapy timeline has become crowded with more and more discoveries leading to new research and new treatments. Gene therapies are now being made available to people with genetic disorders including spinal muscular atrophy and Duchenne muscular dystrophy, and with cancers such as leukemia and many other conditions.
See the range of gene therapies Sarepta is developing.
