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  • Writer's pictureNick Allen

Gene therapy is here — where are we headed?

It’s no longer science fiction — gene therapy is here and is gaining steam fast. Last Friday, the FDA approved two gene-modifying treatments for sickle cell disease, adding to a collection of approximately 30 DNA-modifying drugs already on the market. Among last week’s approvals includes the first drug using CRISPR technology to be approved in the United States. The FDA isn’t showing any signs of slowing, either. Lead regulators have described the need for a strong push to get more of these drugs to market, comparing the mission to the COVID pandemic’s “Operation Warp Speed.”

Rather than try and predict exactly what the next decade will hold, I’ll describe two camps and what each is predicting. One is markedly conservative while the other is bordering on sci-fi. Assuredly, the actual outcome will fall somewhere between these two extremes. 

On the conservative side, individuals point to the obvious shortcomings of the already-approved therapies. These therapies are far from eradicating the diseases they treat. Take Casgevy, the newly approved sickle cell treatment, for example. While being hailed as a breakthrough (and I would agree that it is), it’s only “available” to patients with exceptionally severe sickle cell disease. I say “available” in quotes because even if one does meet the strict eligibility criteria, actually accessing the medicine is another story. The price tag on Casgevy is 2.2 million dollars, falling short of the record $3.5 million which is held by another recently-released gene therapy. On top of this, one has to endure a months-long treatment process that includes receiving high-dose chemotherapy.

People on this hesitant side of the aisle also point to the fact that pharmaceutical companies working on gene therapy are struggling, with many going out of business entirely. Despite price tags that seem sure to create profits, the ultra-expensive R&D process and narrow niche of patients that these drugs treat makes it so that even a slight hiccup can be destructive. On the day their treatment (Lyfgenia) was approved last week, Bluebird Bio stock lost 40% of its value. This is likely attributable to the fact that the FDA is requiring Lyfgenia to come with a warning label that it can potentially increase the risk of cancer in the patients it treats. For all of the aforementioned reasons, the more conservative prediction would be that gene therapy slowly crawls forward, tackling rare diseases one at a time and leading to marginal improvements.

Those at the pharmaceutical company Minicircle are trying to make these conservative estimates look silly. The controversial startup has no shame in taking the “move fast and break things” approach that can occasionally be successful in the tech world, but is typically frowned upon in the medical realm where lives are on the line. Their stated mission is sensible. On a podcast episode from 2020, CEO Mac Davis says that “Big Pharma…is centered around making extremely expensive drugs for extremely rare diseases that very few people have. I want to make affordable drugs for diseases that everybody has.” 

It’s not this mission, but rather their methods that are bringing about concern in the scientific community. Minicircle is running their clinical trials from an island off the coast of mainland Honduras, intentionally designed to be a low-regulation zone that encourages innovation. Additionally, their headline trial is a therapy designed to increase levels of the molecule follistatin. Increasing levels of follistatin effectively decreases the activity of another molecule, myostatin, which normally puts the brakes on muscle growth. If you’ve seen pictures of excessively muscular dogs and cows like those in Figure 1, these are animals that have a mutation which disrupts the genetic code for myostatin and leads to uninhibited muscle growth. Minicircle’s aim is to generate a similar effect, albeit to a lesser extent. Their website claims that this would have benefits both for those with muscular diseases (ALS, muscular dystrophy) as well as healthy individuals seeking to increase muscle mass, lower body fat, improve glycemic control, and live longer.

Figure 1: Animals with excessive muscle mass as a result of myostatin mutations (Source: PNAS

Many in the scientific community feel that Minicircle’s trials and therapies are specifically targeting wealthy individuals with the desire to bring about superhuman characteristics. There are even rumors out there that a handful of well-known celebrities and athletes have already received the follistatin therapy as part of Minicircle’s clinical trial. Those examining the ethics of gene editing have expressed fears about this scenario for years: individuals with power and money being able to take control over the genetic lottery that, up until this point, has simply been a game of random chance. Though experts in the field are doubtful that Minicircle’s therapies even work, the company’s claims and approach represent a stark contrast to the views of the most conservative scientists and regulators.

So what will be our course? Will we struggle towards incremental progress for the treatment of the most devastating rare diseases? Will companies like Minicircle have us all walking around with bulging muscles and living to the age of 150? The most reputable names in this field would remind us that neither will be the case. We’re likely to see exciting advancements in gene therapy over the coming decades, but we must proceed with caution and prioritize equitable advancements. Interested in hearing more about where this all started and where we’re going? Check out Dr. Eric Topol’s recent conversation with Dr. Fyodor Urnov, a pioneer in this space who offers a grand but realistic vision for the future.


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