• CRISPR genetic editing just works…
  • Your smart watch could save your life
  • Thanks to AI, “trial and error” drug discovery is almost over

Dear Reader,

I was in Boston last week at MIT attending a biotechnology conference, the STAT Summit. It was the first year the event was held, and it was fantastic.

To attend, we had to fill out a brief application months in advance. The event was way oversubscribed. In fact, the executive editor of STAT said that he had to turn away more applications than there were executives and scientists in attendance.

The topics were incredible – everything from bionic limbs that patients can “feel” to an exposition of cell and genetic therapies, the use of artificial intelligence and machine learning in drug discovery, and future cures for cancer.

I’ve never seen so much excitement at a biotechnology event. Scientists and doctors tend to be a cautious bunch, sticking to what’s proven, not what’s possible. But not this time.

The event was very forward-looking, optimistic, and emboldened with the power of genetic editing technology and the support of artificial intelligence.

I’d like to share some insights from the conference with you today and tomorrow. You won’t find these stories in The Wall Street Journal or Bloomberg. They are exclusively for readers of The Bleeding Edge.

I hope you enjoy.

CRISPR genetic editing works…

One of the most incredible presentations at the STAT 2019 Summit came from Vertex Pharmaceuticals (VRTX) CEO Jeffrey Leiden. I mentioned this insight briefly in our special weekend edition of The Bleeding Edge.

For background, Vertex has been working with CRISPR Therapeutics (CRSP) on a therapy for sickle cell disease and beta thalassemia. Bad mutations in the hemoglobin gene cause these blood disorders.

This therapy is CTX001. It utilizes CRISPR genetic editing technology. As a reminder, CRISPR is like software programming for DNA. It allows scientists to “cut out” harmful genetic mutations and replace them with healthy DNA.

Leiden talked about how two patients have been treated with CTX001 so far – one with sickle cell disease and the other with beta thalassemia. And the results have been phenomenal.

The beta thalassemia patient has not needed a blood transfusion in nine months. This is amazing considering patients with this disease typically need blood transfusions every 2–4 weeks.

And the sickle cell patient has not suffered a crisis in four months.

In the case of the beta thalassemia patient, 99.8% of her hemoglobin is now of the correct type. That means CRISPR completely eradicated the blood disorder.

In the case of the sickle cell patient Victoria Gray, whom we’ve talked about before, her fetal hemoglobin has increased to 47%. In general, 25–30% of fetal hemoglobin is enough to cure sickle cell disease. So CTX001 has eradicated her disease as well.

Now, what was so striking about Leiden’s talk was how confident he was. Leiden stated directly, “We have essentially cured these diseases. We have cracked the biology of sickle cell and beta [thalassemia].”

There’s no ambiguity at all. And that’s telling.

For a CEO to state explicitly that a therapy has cured diseases after only two patients have been treated is amazing. I’m not sure I’ve ever seen that before.

Of course, it is not FDA approved yet. The next step for Vertex is to expand the clinical trials to another 45 patients. I’m expecting identical results compared to the first two patients.

So this is proof that CRISPR works, and it works amazingly well. The buzz around this at the conference was incredible. We are going to see the technology move rapidly from here. In fact, with results this clear, I expect the process toward FDA approval will be years faster than normal. After all, anyone with these blood disorders will be clamoring for the treatment as soon as possible.

And, of course, the investment implications are staggering. We’re showing returns of more than 300% with one CRISPR company in my small-cap investing service, Exponential Tech Investor. And these are still early days.

And if investors take a stake in only one CRISPR company, it should be this one. This company’s trial results are fast approaching. And if the results are anything like what we saw with CTX001, the stock could soar in a matter of days or hours. Full details here.

The true power of digital health devices…

Another hot topic at the conference centered on research released in The New England Journal of Medicine last week. The research pertained to a study of atrial fibrillation (A-fib). This is a condition where the heart has an abnormal beat, which can lead to stroke or heart attack in severe cases.

What was unique about this study was that it was done on volunteers who use digital health devices, specifically the Apple Watch. Researchers simply pushed out a request to users through their device asking for participants… And they got 419,000 volunteers. That’s the power of having so many devices running at the edge of the network. We can think of these digital devices as real-time health sensors collecting data on patients no matter where they are throughout the day.

Researchers monitored these volunteers for about four months. When they detected an abnormal heartbeat, they sent a notice to the user’s device. The notice suggested the person go get an electrocardiogram (EKG) to confirm the reading. And the results were stunning…

Of these 419,000 participants, only 2,000 received a notice. Of those, 84% of them indeed had A-fib. There were not many false positives.

That’s incredible accuracy given these people were monitored just through their smart watch.

So this shows that digital health devices aren’t just novelties. They can be used for serious research. In fact, Apple has launched three more studies: one for physical mobility, one for menstruation, and one for hearing.

What’s more, these studies can be paired with genetic data. That’s what genetic testing company Invitae is working on. It launched an app to pair the heartbeat data from A-fib patients with their individual genetic data. This will determine if there are any potential genetic causes of A-fib.

This is the future we are heading toward. Very soon, wearable health devices will monitor our vital signs and alert us at the earliest signs of trouble. Think of all the lives that will be saved and the improved quality of life. This is an exciting development.

What AI will do for the biotech industry…

Our last insight from the STAT 2019 Summit today comes from a panel on artificial intelligence (AI). The big question posed to the panel of experts was “How long will it take for AI to find new drug candidates without human involvement?”

Hal Barron, chief scientific officer at pharma giant GlaxoSmithKline, jumped right in. His first reaction was that we’ll see this happen within one to two years. The comment came as quite a surprise to the audience.

A few minutes later, he rephrased his comment… probably building in just a little more cushion… “I think we’ll have drug targets discovered in one to two years… And I would predict within the next two to three years, we’ll have examples of drug targets discovered by machine learning that unravel biology.”

So Barron believes that we are about to unleash AI on drug discovery. This is big news for the industry.

Take the A-fib study we just talked about, for example. A company could put an AI to work on that data set and task it with finding therapies. The AI would then comb through the data and find the highest probability drug candidates.

This would lead to a rapid acceleration in drug development. Unlike human researchers, an AI does not need to eat, sleep, or play. It can work on drug discovery all day, every day.

And with AI-discovered therapies, we would likely see the FDA approval process speed up as well. That’s because AI will come up with more precise therapeutic solutions. There won’t be as much trial and error… which means we’ll see more concrete results in clinical trials. And that should lead to a faster trial process.

So industry leaders are very excited about what AI will do for the biotech industry. And this will have an immensely positive impact on human health as well.

I’m not kidding when I say we are on the cusp of curing all human disease of genetic origin.


Jeff Brown
Editor, The Bleeding Edge

P.S. I simply can’t stress this enough, CRISPR genetic editing works. It just works…

My longtime readers know I don’t do this often, but I have to insist. Every serious technology investor needs to have some exposure to key companies in this space. If you miss this opportunity, you will regret it for the rest of your life.

And one CRISPR company, in particular, is about to make headline news. I have good reason to believe that this company is on the verge of curing a debilitating disease just like CTX001 is doing with sickle cell and beta thalassemia. And when that happens, I believe shares could shoot as high as 1,000% quickly. Every serious technology investor needs to own shares in this company right now. Go right here for the full details.

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