Health Care's New Frontier: Gene Therapy Cures 10 Babies
Editor's note: Today, we're featuring another essay from our friend and tech investor Matt McCall...
Matt, a chief investment analyst at our corporate affiliate InvestorPlace, is an expert at identifying disruptive trends that can shift entire industries – before the rest of the world catches on. He has recommended 120 different stocks that have more than doubled in his career... including one medical stock that booked an astounding 3,600%-plus gain.
And that could be only the beginning...
In today's Masters Series, Matt details an incredible new development in the biotech sector that he believes is the future of medicine... explains how it can cure previously "uncurable" diseases... and reveals what he expects from the companies at the forefront of this groundbreaking treatment...
Health Care's New Frontier: Gene Therapy Cures 10 Babies
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 clearly shows the breakthrough possibilities of cutting-edge treatments that "edit" genes.
The data is extremely encouraging. For example, scientists from St. Jude Children's Research Hospital and UCSF Benioff Children's Hospital announced the cure of 10 newborn babies who were infected with the rare "bubble boy" disease.
Scientists extracted 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.
"Bubble boy" disease is somewhat well-known because it was 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 ("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 decade or so, a small group of scientists working at different institutes has 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 10 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 "crisper."
The therapy 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 (or genes). Once mutations are found, Cas9 is used to identify the sequence where the mutated gene can 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 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 United States. Another 2 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 an estimated 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 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.
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 can 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 $4.7 billion. Simple math tells us that it would be a 23-bagger to get to a market cap of $110 billion.
To be clear, we are still years away from being able to fully cure diseases using CRISPR, but now is the time to buy.
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. And I have no doubt it will create incredible wealth for smart investors who get in early.
Best regards,
Matt McCall
Editor's note: CRISPR/Cas9 could change the way we look at medicine forever... curing potentially thousands of previously uncurable diseases in the process. And Matt says now is the time to invest in this life-altering breakthrough...
Matt has identified four companies that could skyrocket as this new gene-editing technology goes mainstream... And he says investors who get in during the early stages could make an absolute fortune.
That's why he put together a special report with all the details – including a full breakdown of how CRISPR/Cas9 works... and why he believes these companies could make you 5 to 10 times your money or more as this technology takes off. Until tomorrow, watch Matt's presentation right here.