It’s Risky Business

There’s a race on in the biotech world.

A number of companies have developed variations of gene therapy technology.

As all biotechs with a new platform will do, they’ve looked at a range of diseases they could help cure.

Biotech development is a risky business and many promising new therapies never make it market, failing along the way.

So when an upstart biotech chooses the first application for its technology, it chooses carefully the one with the lowest potential risk and the highest reward…

They are looking for the one that’s likely the fastest to show a new therapy works, that’s the easiest and cheapest to develop and with the highest likelihood for success.

Biotechs tend to choose the “low-hanging fruit,” as it were.

Stopping the Bleeding

A number of gene therapy companies are working on a therapy for the bleeding disorder known as hemophilia. The disease comes in two main variants: hemophilias A and B.

Both are caused by a defective gene that doesn’t code for the production of a blood clotting factor, leading to dangerous risk of a person bleeding excessively both internally and externally.

That makes hemophilia a case of “low-hanging fruit” for developmental gene therapy companies. The cause of the disease is well understood. It’s believed to be one of the more easily addressable indications for emerging gene therapy platforms. The disease is a serious one. And a significant number of patients need a therapy.

In gene therapy, it’s a crowded race to treat hemophilia. And investors are watching carefully.

Whoever gets to market first is likely to capture a large share of that market for good, provided they have a really effective and safe gene therapy.

Which leads me to the company I’d like you to consider today.

Billions of Dollars Up for Grabs

BioMarin (NASDAQ: BMRN) is a California based biotech that focuses on curing rare diseases using advanced gene therapy research.

The company currently has a pivotal hemophilia A therapy in development. Patients with hemophilia A are missing, or have low levels of a clotting protein called Factor VIII.

BioMarin’s new therapy, called Valrox, seeks to restore Factor VIII levels to normal in patients.

So far phase 1/2 trial data is promising with the therapy boasting a “97% reduction in mean annual bleed rates,” as reported by

Based on this data the company announced they’re eyeing to file for FDA approval “in the second half of next year,” as reported by

On the year, the stock has performed well considering the volatility we’ve endured throughout 2018.

Here’s the YTD for BioMarin (black line), as compared to the S&P 500 (red line) and Dow Jones (blue line).


There’s thousands upon thousands of folks who could benefit from this new hemophilia A therapy.

And to that end, gene therapy drugs are wildly expensive, meaning FDA approval in 2019 could open up the door for a massive financial boon for BioMarin and their shareholders.

For Technology Profits Daily,

Ray Blanco
Chief Technology Expert, Technology Profits Daily

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Ray Blanco

Ray Blanco is the editor of Technology Profits Confidential as well as Breakthrough Technology Alert, FDA Profit Alert, and Technology Profits Daily. Ray has been with Seven Figure Publishing since 2010. In 2019, his closed positions in Technology Profits Confidential outperformed the S&P500 by 50%.

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