What was the sequence of events?
*See what I did there?*
As explained in the first post, gene therapy is the introduction, removal, or altering of genetic material to manipulate how a group of proteins is produced in a cell.
Although the therapy itself is very recent, the science behind it has been known for decades.
In 1990 the first human patient was treated with gene therapy. She was a four-year-old already suffering from severe combined immunodeficiency and received treatment for adenosine deaminase, a congenital disease. After this milestone, diseases such as hemophilia, cystic fibrosis, and cancer have been treated with gene therapy.
Diagram demonstrating LUXTURNA altering genes in eyes.
In 2017, the FDA approved the first gene therapy called Luxturna which helps treat vision loss, especially hereditary cases. It is the first approved gene therapy, in the US, that targets a genetic disease. "I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses,” said FDA Commissioner Scott Gottlieb, M.D. “We’re at a turning point when it comes to this novel form of therapy and at the FDA, we’re focused on establishing the right policy framework to capitalize on this scientific opening. Next year, we’ll begin issuing a suite of disease-specific guidance documents on the development of specific gene therapy products to lay out modern and more efficient parameters — including new clinical measures — for the evaluation and review of gene therapy for different high-priority diseases where the platform is being targeted.”
With all this being said, and over 1,000 gene therapy clinical trials underway, gene therapy is still in the early stages of research.
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