CRISPR Therapeutics Stock Jumps on Trial Breakthrough

By Anthony Summers
Senior Research Analyst

Emerging Tech

One of my favorite science fiction films of all time is Gattaca.

Released in 1997, it presents a thought-provoking vision of the not-so-distant future where the science of gene editing has affected all aspects of society.

While the film articulates the proposed benefits of gene editing – less disease, reduced crime, enhanced physical and cognitive aptitude, and greater societal achievement – it also depicts the potential pitfalls of a society that obsesses over genetic perfection.

Gattaca was great science fiction at the time of its release. But 20 years later, it has become reality.

Today, gene editing technologies are part of an incredibly fast-growing industry that promises to treat (and potentially cure) previously incurable diseases.

The enormous potential of gene editing treatments means investors can’t afford to ignore this trend. It’s why I’ve argued before that investing in the gene editing space in its early stages will prove to be profitable down the line.

But first, let’s address the obvious… As the field is still in its infancy, it’s prone to the risks associated with skepticism, regulatory setbacks and ethical concerns.

That’s certainly been the case for CRISPR-based gene editing treatments, which I’ve discussed in the past.

For example, consider CRISPR Therapeutics (Nasdaq: CRSP), which remains the front-runner in the U.S. field.

The company was co-founded by Emmanuelle Charpentier, half of a two-researcher team that includes Jennifer Doudna, who developed the CRISPR/Cas9 gene editing method primarily used today.

Last year, some controversy arose concerning two studies that proposed CRISPR-based treatments might increase the risk of cancer.

As a result, not only did shares of CRISPR Therapeutics take a hit, but nearly all gene editing stocks in the market dropped.

Those studies were immediately dismissed as inconclusive. In October 2018, the FDA decided to lift its hold on clinical trials for CTX001 – a gene editing treatment being developed by CRISPR Therapeutics and Vertex Pharmaceuticals (Nasdaq: VRTX).

Just a few months later, we’re already seeing positive results…

On Monday, CRISPR Therapeutics and Vertex Pharmaceuticals announced that one trial patient suffering from beta thalassemia has become the world’s first person treated with CTX001.

This person is part of a Phase 1/2 clinical study of patients with rare and inheritable blood diseases, including beta thalassemia and sickle cell.

Shares of CRISPR Therapeutics popped following this announcement…

But this isn’t just a milestone for the companies involved. It’s also a major morale boost for companies researching the promise of CRISPR-based treatments.

Even so, just as the film Gattaca predicted, ethical concerns have surfaced…

Last year, Chinese scientist He Jiankui claimed to have created the world’s first genetically engineered HIV-resistant twin babies.

The intentions were obviously good. In the absence of a cure for AIDS, a preventive approach seemed reasonable and even desirable.

But these actions ignited international condemnation and concerns about the danger of human experimentation – especially on embryos.

The Chinese government even released preliminary regulations placing gene editing and related technologies under the oversight of the State Council’s health department.

I have no doubt that more ethical controversies will arise as time goes on. And investors should be aware of the risks that future regulatory actions could have on their investments.

But I’m confident that the positive impact that gene editing is slowly but surely unleashing on the world will outweigh those risks.

Good investing,

Anthony