Gene-therapy history
1962: Gene therapy is born
Professor William Szybalski shows that a genetic mutation can be corrected by adding DNA into an animal cell.1
1975: The Asilomar Conference
In light of global skepticism around genetic modification, scientists, journalists, and government officials from around the world meet in California to discuss the future of gene technology. Despite disapproval by some well-known members, the conference ends with the decision to continue gene technology research with the enforcement of a strict set of rules.3
1993: Discovery of CRISPR
Professor Francisco Mojica finds DNA in bacteria with unique repeating structures, later to be known as clustered regularly interspaced short palindromic repeats (CRISPR).4
1999-2000: Setbacks for gene therapy
Gene-therapy patient Jesse Gelsinger dies after an unexpected immune reaction to the gene’s delivery vehicle. The next year, 2 clinical trials of gene therapy in the UK and France result in 5 out of 20 male patients developing leukemia from uncontrolled mutations in their DNA.5-7
2000: National Gene Transfer Research Safety Symposia initiated
The first symposium is convened to discuss new safety protocols to enhance the protection of research participants in gene transfer clinical trials.8
2000: FDA and NIH plan new safety initiatives
The US Food and Drug Administration (FDA) and National Institutes of Health (NIH) cooperate to enforce more transparency in gene-therapy clinical trials, as well as adherence to existing guidelines, to improve safety for trial participants.9
2005: ZFNs studied for gene modification
Zinc-finger nucleases (ZFNs), first discovered in the African clawed frog, are shown to modify the x-linked severe combined immune deficiency (SCID) mutation in a human’s IL2Rγ gene, giving hope to the possible use of ZFNs in treating diseases.10-12
2011: Plant pathogens successfully modify human cells
Transcription activator-like effector nucleases (TALENs), engineered plant pathogens known for their high safety record in gene editing, are used for the first time in clinical trials to safely modify the human genome.13-15
2012: Discovery of third-generation gene-editing tool
Scientists Emmanuelle Charpentier and Jennifer A. Doudna reprogram the ancient defenses of toxic bacteria to make precise cuts to any DNA molecule for gene editing. These low-cost genetic snipping tools are aptly named CRISPR/Cas9 genetic scissors.16-18
2012: FDA approval of first HPC, cord blood
The FDA approves the first allogeneic hematopoietic progenitor cord blood therapy for use in patients with disorders affecting the hematopoietic system.19
2017: FDA approval of first gene therapy
The FDA approves the first gene therapy available in the United States for certain patients with acute lymphoblastic leukemia.20
2017: FDA approval of first CAR T-cell therapy
The FDA approves the first CAR T-cell therapy for adult patients with large B-cell lymphoma.21
2023-2024: FDA approval of the first ever gene-editing therapy
In 2023, the FDA approves the first ever gene-editing therapies for sickle cell disease, followed by an approval for β-thalassemia in 2024.22,23
2024-Present: FDA continues to evaluate gene therapies as new applications are submitted
Cell therapies, cord blood therapies, and gene therapies continue to evolve, and the FDA evaluates potential new therapies across various disease states.
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