The coming era of gene therapies will be considerably more distributed and bottom-up than the advent of stem cell therapies. This will be a dynamic industry in which many small groups compete to set up distribution of mail order kits and clinics to provide widespread access to therapies. Regulators will attempt to suppress all of this, and will largely fail, as money talks and many regions will choose to host the businesses that offer gene therapies. This will come to pass because gene therapy technologies are many times cheaper, more easily managed, and capable of centralization and mass production than stem cell technologies. You might look at how medical tourism for stem cells progressed over the past twenty years, and expect the gene therapy industry to grow many times faster once the spark is lit. It will also be far more accessible to members of the public in its earlier stages: cost of the product drives the character of an industry.
There are several very promising targets for the first gene therapies, the best of which, in my opinion, are follistatin and myostatin, which control muscle growth, and are well studied. There are even a few natural human myostatin mutants, to accompany the many well-muscled myostatin mutants in other mammalian species, both natural and engineered. A number of other genes will be targeted in the first years of the industry, such as those that can dramatically lower blood cholesterol, and which also either have thriving human mutants or are already targeted by drug-based therapies. The only thing holding back an explosion of activity is the fact that current methodologies, even those based on CRISPR, are not yet up to scratch. They don't reliably introduce the therapy into a large enough number of cells in adults, and particularly into stem cells in order to make it truly lasting. When that changes, we'll all be in for an interesting ride.
Two companies say they'll continue offering DNA-altering materials to the public. The companies, The Odin and Ascendance Biomedical, both recently posted videos online of people self-administering DNA molecules their labs had produced. Following wide distribution of the videos, the FDA last week issued a harshly worded statement cautioning consumers against DIY gene-therapy kits and calling their sale illegal. A growing number of cases of DIY gene therapy are putting the health regulator in a difficult situation as individuals argue that no law stops them from self-administering the substances. In fact, there is a long history of scientists carrying out experiments on themselves, including some Nobel Prize winners. Last month, Josiah Zayner, CEO of The Odin, which sells DIY biology kits and supplies through its website, posted a video in which he injected himself with the gene-editing tool CRISPR during a biohacker conference.
The problem facing regulators is that interest in biohacking is spreading, and it's increasingly easy for anyone to obtain DNA over the internet. It's also easy to get hold of the recipes necessary to carry out gene editing using CRISPR, a potent new technique for modifying DNA. In October, Zayner's website began selling $20 copies of a DNA molecule containing the necessary genetic information to deactivate the human gene for a certain protein, myostatin, using CRISPR. Human DNA can be purchased through a number of other companies that cater to research labs. The difference is The Odin markets its DNA to amateur biologists. The materials sold by The Odin also can't be directly used to alter a person's genes. Instead, they contain DNA that would have to be produced in larger amounts, purified, and then delivered to the body using methods well beyond the skills of most consumers.
At least one other company appears to have begun offering finished gene-therapy preparations directly to patients for their own use. In October, an HIV patient was filmed injecting himself with a gene therapy designed to generate antibodies that he believed would help his body destroy cells infected with the virus. The material he used was supplied by Ascendance Biomedical, an until recently unknown startup company that promotes "decentralized" testing of new drugs. The company is also developing a herpes vaccine, as well as a follistatin gene therapy to boost muscle mass and reduce fat. Aaron Traywick, the CEO of Ascendance, says Ascendance plans to make both of those therapies available for self-administration by early next year.