Imagine being able to cure diseases the same way you fix a typo.
That’s a bit of an oversimplification because the process is more involved than a “delete” button on a keyboard. But it helps make clear the breakthrough possibilities of gene editing.
Last week was big for the industry. Scientists from St. Jude Children’s Research Hospital and UCSF Benioff Children’s Hospital announced the cures of 10 newborn babies with the rare “bubble boy” disease.
Scientists took out stem cells from the babies and used a modified HIV virus to deliver the “corrected” copy of the gene back into the patient. It cured the babies with no apparent side effects. This is huge. A previous trial was discontinued in 2003 after some children developed leukemia.
“Bubble boy” disease is somewhat well known because it has been featured in a movie, The Boy in the Plastic Bubble, which was released in 1976 and starred a young John Travolta. A boy suffering from the disease was also featured in a zany episode of Seinfeld in 1992.
It is officially called severe combined immunodeficiency disease (SCID), and it basically means that a baby is born with no immune system. Any germs in the air can be fatal, which is why sufferers are often isolated. It is rare for them to survive past their first birthdays, but this new gene therapy treatment could be the end of that.
Over the past six years, a small group of scientists working at different institutes have made incredible progress in this revolutionary new field of “gene editing.”
Gene editing is exactly what it sounds like. It’s the changing – or “editing” – of an organism’s genes to produce a desired outcome. While humans have tinkered with this idea for decades, it’s only in the past six years or so that CRISPR/Cas9 technology has radically changed what we can do in this field.
CRISPR is short for “Clustered Regularly Interspaced Short Palindromic Repeats.” But I’m more than happy to stick with CRISPR, which is pronounced like “crisper.”
The therapy is a gene editing technique that allows for the modification of any region of a genome. It can be performed on any species with accuracy and without causing harm to other genes. That’s why there are so few side effects.
Boiled down to basics, a protein (Cas9) is used to essentially cut the DNA, thus editing it. This is done to correct mutated genes and ultimately cure a disease.
The first step in the process is to have the genome mapped, and the next step is to search for the mutated gene(s). Once mutations are found, Cas9 is used to identify the sequence where the mutated gene with either be edited (Cut & Revise in the chart below), deleted (Cut & Remove), or replaced (Cut & Replace).
Picture the “find and replace” function in word processing software. Once the word is found in the document, you have the option to either delete, edit, or replace it – the same options available with CRISPR.
It’s really amazing stuff.
CRISPR is the secret weapon that will be able to eliminate diseases that currently have no cure. Take sickle cell disease, a disorder in which a person lacks enough healthy red blood cells to carry adequate oxygen throughout the body. There is no cure for this disease, which affects over 100,000 people in the U.S. Another two million people have the trait, and one in every 13 African-American babies is born with it.
Sickle cell disease is caused by one “misspelled letter” of DNA. If this can be replaced, it could essentially wipe out a disease that causes severe pain and often premature death – very similar to bubble boy disease.
According to the World Health Organization, there are 10,000 monogenic diseases like sickle cell – diseases caused by a mutation in just one gene. Think cystic fibrosis, hemophilia, Huntington’s disease, and many others.
Today, about 95% of the 10,000 monogenic diseases go untreated, and yet one in every 100 newborns has some type of monogenic disease from birth. Through gene editing, the removal or repair of that one gene can essentially cure thousands of diseases.
The Time to Invest in CRISPR Stocks is Now
Gene editing and gene therapy in general are in their very early stages. That makes now the time to invest. By getting in early, you can set yourself up for what I anticipate will be some of the biggest winners of your lifetime.
Take a look at this chart, which shows the total addressable market of monogenic diseases in the United States and abroad.
The $75 billion number on the left is just new diagnoses each year. Even more astounding is the $2 trillion market of people who are already living with diagnosed diseases.
If a single gene editing company is able to provide one-time cures for just 10% of the new diagnoses each year and only 1% of already diagnosed patients, it would lead to staggering annual sales of $27.5 billion.
The average market cap of the three leading gene-editing companies is approximately $1.36 billion. Simple math tells us that it would be an 81-bagger to get to a market cap of $110 billion, which would be a price-to-sales ratio of 4 – in-line with where the biotech industry has traded recently.
To be clear, we are still years away from being able to fully cure diseases this way, but today is the time to buy CRISPR stocks. By the time every magazine and newspaper is touting the life-altering ability of this new technology, it will be too late.
We don’t get many chances to invest in a once-in-a-lifetime, game-changing medical breakthrough. We need to take full advantage. Gene editing is the future of medicine.
It will save, lengthen, and improve the quality of billions of lives in ways we can only just now begin to even imagine.
Any such breakthrough has enormous financial implications, especially when it involves how long and how well we live. I have no doubt it will create incredible wealth for investors who understand those implications.
I recommend the leading gene editing companies in my Early Stage Investor service. They have gotten off to a solid start in 2019, up about 25% on average. But this industry is still in its infancy, so there is plenty of time to get in position with CRISPR stocks now. Click here to find out how.
Matthew McCall is the founder and president of Penn Financial Group, an investment advisory firm, as well as the editor of Investment Opportunities and Early Stage Investor. He has dedicated his career to getting investors into the world’s biggest, most revolutionary trends BEFORE anyone else. The power of being “first” gave Matt’s readers the chance to bank +2,438% in Stamps.com (STMP), +1,523% in Ulta Beauty (ULTA), +1,044% in Tesla (TSLA), +611% in Liquefied Natural Gas Limited (LNGLY), +324% in Bitcoin Services (BTSC), just to name a few. If you’re interested in making triple-digit gains from the world’s biggest investment trends BEFORE anyone else, click here to learn more about Matt McCall and his investments strategy today.
More From InvestorPlace
- 2 Toxic Pot Stocks You Should Avoid
- 7 Dividend Stocks That Could Double Over the Next Five Years
- 6 S&P 500 Stocks Ready to Break Out
- 5 Mining ETFs to Dig Into
The post A New Frontier for CRISPR Stocks: Gene Therapy Cures 10 Babies appeared first on InvestorPlace.