Gene therapy is the administration of genetic material to modify or manipulate the expression of a gene product or to alter the biological properties of living cells for therapeutic use.
Gene therapies offer the potential to treat diseases or conditions for which no or few treatments exist. They are being studied to treat cancer as well as genetic, infectious, and other diseases. FDA considers any use of CRISPR/Cas9 gene editing in humans to be gene therapy.
Gene therapy products are regulated by the FDA’s Center for Biologics Evaluation and Research (CBER). Clinical studies of gene therapy in humans require the submission of an investigational new drug application (IND) prior to their initiation in the U.S., and marketing of a gene therapy product requires submission and approval of a biologics license application (BLA). Clinical trials of gene therapies can be found at www.clinicaltrials.gov. FDA has also approved certain gene therapy products.
FDA has taken action against gene therapy products intended for self-administration and “do it yourself” kits to produce gene therapies for self-administration. The sale of these products is against the law.
Celebrity biohacker Josiah Zayner was investigated under by California state officials for practicing medicine without a license. Zayner’s company, The Oden, sells kits and tools intended to allow any consumer to genetically modify bacteria, yeast, animals, and even humans. Zayner attempted a DIY fecal transplant in an airport hotel, then tried to genetically engineer his skin.
“I want to live in a world where people get drunk and instead of giving themselves tattoos, they’re like, ‘I’m drunk, I’m going to CRISPR myself,’” said Zayner, who has a few tattoos of his own, in an interview with BuzzFeed News.
Before his biohacker stunts, Zayner spent two years as a researcher at the Mountain View, California’s NASA Ames Space Synthetic Biology Research Center, where he worked on Martian colony habitat design. He has a BA in plant biology from Southern Illinois University and a Ph.D in biophysics (2013) from the University of Chicago.
The California Department of Consumer Affairs informed Zayner in May 2019 of their investigation into a complaint against him for practicing medicine without a license. By September of the same year Zayner received notice that the investigation had been concluded, and “no further action is anticipated.”