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As Gene Therapies Disrupt Big Pharma, Here’s How to Get Your Piece of the Action

4 | By Michael A. Robinson

A doctor injects a “corrective” gene into a virus. Then he uses that virus to introduce the new gene and modified DNA into a patient’s diseased cells. If the treatment is successful, the cells’ gene-reading machinery will build RNA messengers and protein molecules – i.e., disease fighters.

It’s a process that could be a cure for hundreds of genetic-related diseases.

That’s gene therapy in action.

When biotech researchers get gene therapy to work dependably, it will be a boon for humanity – and for investors.

For decades, Big Pharma has run on the same basic plan – use chemicals to create disease-fighting drugs. However, many of the drugs they end up creating carry severe side effects or are highly addictive.

But now, after a quarter century of failed experiments and numerous setbacks, gene therapy finally has the potential to make patients well again without addictive or harmful drugs.

Researchers worldwide report successful gene-therapy trials – and new and innovative uses for the technology – seemingly every week. The first federally approved gene therapy, the severe pancreatitis treatment Glybera, hit the market in late 2014 as. And I think the next half decade will bring dozens more gene-therapy products along those same lines.

Now, finding a way to invest in this newly disruptive biotechnology is tough. Most of the companies working on gene therapies are privately held or very risky, illiquid clinical-stage biotech companies that I just can’t recommend to beginning investors.

However, I have located a great backend way to play this breakthrough field. Its stock has soared 25% in just the past five months or so – and it shows no sign of slowing down. Plus, it pays a healthy 2% dividend.

Let me show it to you…

Broken Hearts to Bright Horizons

Gene therapy has broken a lot of hearts – and few wallets – over the years. The promise of gene therapy has long seemed just over the horizon.

But now, several of the world’s biggest drug firms are either directly involved or are forming pacts with those clinical-stage biotechs I mentioned.

This is a technology that could have a big impact on the $1.2 trillion market for global prescription drugs. If gene therapy captures just 20% of drug sales over the next 5 to 10 years, that would be a $240 billion market.

The Genetic Science Learning Center at the University of Utah defines gene therapy as a way to fix “a genetic problem at its source.” That means gene therapy has the potential to treat any disease linked to your DNA.

That’s not every ailment, but it covers everything from breast and colon cancer to hemophilia and sickle-cell disease.

With gene therapy, physicians “correct” a copy of a defective gene so that diseased tissues and organs work properly once again. In other words, gene therapy doesn’t treat symptoms – it solves the underlying genetic problem.

Like I said, though, gene therapy isn’t a universal platform, at least not yet with current technology.

First off, plenty of diseases are not genetic. Plus, some genetic disorders are just too complex for gene therapy to address.

But this is the dawn of a new industry – one that offers hope for millions of patients and that will end up handing investors who get in early outsized gains…

The Way to Play It

Earlier this week, scientists at the National Institutes of Health’s National Eye Institute revealed that they’ve used gene therapy as part of treatment to regenerate damaged optic nerves. They say this method could help millions of patients with vision loss or blindness due to glaucoma.

And like I told you just this past weekend, researchers at the University Medical Center Göttingen in Germany say they have found a way to supercharge cochlear implants – cutting-edge hearing aids – through a combination of gene therapy and tiny light-emitting diodes (LEDs). That’s a sign gene therapy is joining what I’ve been calling the “Convergence Economy” – when multiple tech innovations intersect in such a way that the sum of their parts is much greater than their individual additions.

Then there’s Glybera, which I told you about in the introduction to today’s report. That’s the gene-therapy advancement that’s led me to today’s recommendation.

Let me explain…

UniQure NV (Nasdaq: QURE) is the firm that formulated Glybera, the first gene-therapy drug on the market. It’s a treatment for lipoprotein lipase deficiency (LPLD), a rare genetic disorder that causes inflammation of the pancreas – pancreatitis.

Because that disease affects just 1 million people around the world, Glybera is quite pricey — about $1 million per year.

But we’re just getting started here – and researchers are working on gene therapies that will have much lower prices because they will serve much larger patient groups.

UniQure itself has a series of other gene therapies in clinical trials, such as one for hemophilia, a genetic condition that afflicts 400,000 people worldwide. Other targets include Parkinson’s disease; acute intermittent porphyria, which causes severe pain and mental symptoms; and Sanfilippo syndrome, an inherited metabolic condition that causes sufferers to have a hard time processing sugar.

As much as I like uniQure’s science and its product pipeline, it’s a loss-ridden, clinical-stage biotech firm that’s just too volatile for retail investors.

That’s why I’m sending you in the direction of Bristol-Myers Squibb Co. (NYSE: BMY), a high-quality “backend” play on gene therapy.

BMS and uniQure teamed up in April 2015 in a deal that provides BMS with exclusive access to uniQure’s gene-therapy platform that targets several cardiovascular diseases. The collaboration includes uniQure’s proprietary gene therapy for congestive heart failure that’s intended to restore the heart’s ability to synthesize S100A1, a calcium sensor and master regulator of heart function.

Beyond cardiovascular diseases, the two also agreed to potentially partner to use gene therapy to fight at least 10 other genetic disorders.

Every time uniQure discovers a new gene-therapy target, it will receive another $15 million from BMS. In fact, BMS has already sent $200 million uniQure’s way during this partnership – which gives it a 4.9% equity stake in the smaller company. If uniQure meets all of this deal’s goals, it stands to receive $2 billion from BMS.

For its part, BMS gets partial sales rights for any drugs that come out of those research efforts.

By investing in BMS, you get any upside from gene therapy – but also the safety that comes from Bristol-Myers’ huge portfolio of drugs and a global reach.

All About BMS

Bristol-Myers, based in New York City, is one of the world’s top Big Pharma firms, and its specialty is fighting cancer. It also manufactures treatments for HIV/AIDS, cardiovascular disease, diabetes, hepatitis, rheumatoid arthritis and psychiatric disorders

Its top products include the antipsychotic Abilify and the blood thinners Plavix and Eliquis.

It’s also adept at getting approvals for new drugs. Just in the last two years alone, the U.S. Food and Drug Administration gave nine new BMS cancer drugs the green light.

With roots dating back to 1858, this is a very well-run firm. Last year, BMS brought in more than $3 billion in levered free cash flow, and it has 26% operating margins.

Trading at around $77, this $128.4 billion market-cap company also pays a roughly 2% dividend.

And it’s a stock on the move. Since it hit bottom on Feb. 2, BMS shares have gained more than 25%. That’s more than double the S&P 500’s return over the same period. Plus, it’s trading close to its all-time high, a sign of key bullish support.

So, with BMS and its deal with uniQure, we get the upside that comes from a breakthrough new biotech field – gene therapy — and a stable, global leader in a powerful uptrend…

An uptrend that looks like it will keep going for a long, long time.

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4 Responses to As Gene Therapies Disrupt Big Pharma, Here’s How to Get Your Piece of the Action

  1. Aaron Ashcraft says:

    $GSK is also doing extensive research on gene therapy. Their researchers in Italy have cured 5 patients who suffered from inherited Severe Combined Immune Deficiency (AKA Bubble Boy Disease) by implanting stem cells into the bone marrow of patients to cure the inherited disease. This has not received extensive publicity due to the tiny number of people who inherit this rare disease, but it is acutely important as a step towards curing diseases like cancer. Our family has special interest in this development since our daughter has SCID.

  2. Barry Dietsche says:

    Michael, I have become a big believer in your Strategic Tech Investor. You are a good man, with good insight, who wants to share. Thank you so much.
    Barry D, Bloomer, Wisconsin, retired farmer and excavator

  3. Thomas Jarrett says:

    It would be a bit more compelling to compare Uniqure with a few other leading Gene Therapy research companies and compare their development pipelines. The above article does not offer a compelling argument to invest in BMS.

    It is an area I am interested in. There must be other Gene Therapy research companies with more compelling product and product applications.

  4. Todd Duncan says:

    It would be great, in the future, to refer to an imaginary doctor as “she or he,” rather than the simplistic “he.” More women are now enrolled in medical school than men. Not to mention that the 2 discoverers of Crispr, who will no doubt be honored by the Nobel Prize Committee, (and through their genius work, will drive profits for investors,) are women.

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