Editor’s Note: As Bill often says, our usual beat in the Diary is money. But every weekend, we like to feature new, and profitable, ideas from Bill’s network of investing experts.
Today, Jeff Brown, Bill’s top technology analyst, pens our weekend Diary. He reveals what could be “the biggest medical breakthrough since antibiotics,” and shows how you can profit.
Emily Schaller was diagnosed with cystic fibrosis (CF) when she was 18 months old. At that time, those born with CF typically did not live beyond their eighteenth birthday.
Today, Emily is 35 years old.
Cystic fibrosis is a rare, life-threatening genetic disease. It is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene.
The CFTR gene is the cystic fibrosis transmembrane conductance regulator gene.
When there is a mutation of the CFTR gene, it leads to dysregulation of fluid transport in the lungs, pancreas, and other organs.
More simply, there is a poor flow of salt and water into or out of the cells in these organs.
It leads to a buildup of thick mucus that can cause chronic lung infections and progressive lung damage. And when the mucus clogs the pancreas, the human body cannot properly digest food, which leads to weight loss and malnutrition.
An estimated 75,000–80,000 people around the world are living with CF. And sadly, it eventually leads to an early death.
But that could soon change.
A revolutionary piece of technology is on the horizon. It has the potential to cure Cystic Fibrosis, as well as thousands of other genetic diseases, permanently.
And of course, smart investors can potentially make a fortune as this technology rewrites how we think of medicine.
The End of Disease
The technology I’m referring to is genetic editing. Genetic editing is a type of genetic engineering where DNA can be inserted, deleted, or replaced. In essence, it’s a technology that will allow scientists to fix “typos” (mutations) in our DNA, the sort of typos that can cause terrible diseases like cystic fibrosis.
Specifically, I’m referring to a type of genetic editing known as CRISPR-Cas9. CRISPR-Cas9 stands for “clustered regularly interspaced short palindromic repeats – CRISPR associated protein number 9.”
It’s a mouthful, I know. Most people just refer to it as “crisper,” which is much simpler.
In August 2012, a research paper about CRISPR-Cas9 was published and took the science community by storm. It demonstrated the potential of the CRISPR-Cas9 genetic editing technology to literally cure all human diseases that are caused by genetic mutations.
Very few know this, but there are more than 6,000 diseases caused by genetic mutations. Worse, 95% of them have no approved therapy or treatment. To understand the scale of the problem:
- About 3–4% of all babies will be born with a genetic disease or major birth defect
- 1% of all babies will be born with a chromosomal abnormality
- More than 20% of infant deaths are caused by birth defects or genetic conditions
- About 10% of adults in hospitals are there due to genetically related problems
- About 30% of children in hospitals are there due to genetically related problems.
Gene editing can be used to “fix” or improve the genetics of plants, animals, and even humans. To say that this technology is revolutionary is an understatement. It empowers the human race to do things like:
- Provide human gene therapy for serious genetic diseases that have never had treatment
- Have the ability to run screens for drug targets – accelerating new drug development
- Make pest-resistant crops to improve yields and feed the planet
- Improve the health of livestock
- Tackle major diseases like malaria at the source, by effectively sterilizing mosquitos through genetically restricting their ability to carry the disease.
Tech Titans Recognize CRISPR’s Potential
You may be thinking that this sound a bit like science fiction.
But I assure you, this technology is very real. And some of the biggest players in tech are pouring money into this research for philanthropic reasons.
Billionaire, and Facebook’s first president, Sean Parker founded the Parker Institute for Cancer Immunotherapy in 2016.
In June of that year, an advisory committee at the U.S. National Institutes of Health (NIH) approved a proposal by the Parker Institute at the University of Pennsylvania to use CRISPR genetic editing techniques to help find a cure for cancer.
CRISPR technology will be used to modify human immune cells so that those cells can “kill” cancer.
Those human trials will begin sometime this year. In fact, I’m expecting that a green light will be given for the trials any day now. Which means we can expect provisional results on the effectiveness of this U.S. based trial at the University of Pennsylvania before the end of 2018.
But Parker isn’t the only Silicon Valley figure getting involved with CRISPR technology.
Bill Gates, founder and former CEO and chairman of Microsoft, has invested close to $75 million through the Gates Foundation to eradicate malaria. The purpose of the investment is to apply CRISPR genetic editing technology towards solving this problem.
The Gates Foundation has been focused on eradicating malaria by utilizing what is called a “gene drive.” A gene drive can either remove all female mosquitos from the population or remove the genes that enable mosquitos to transmit the virus.
The original targets for testing a solution in the field were originally forecast by the Gates Foundation for 2029. With this new investment and Gates’ personal belief in the potential of CRISPR, he has stated publicly that the technology might now be ready in one to two years.
The Sky Is the Limit
The CRISPR-Cas9 genetic editing platform is the most revolutionary development in biotechnology since antibiotics. And it will lead to curing all diseases caused by genetic mutations.
I estimate the total market opportunity for therapies derived from CRISPR-Cas9 genetic editing to exceed $1 trillion. Essentially, the sky is the limit.
One exciting company in this space is Illumina (ILMN), which is the world’s leader in genetic sequencing equipment. Genetic sequencing and analysis is a critical part of finding the genetic mutations that cause specific diseases.
While genetic editing is still in its infancy, the progress made every month on research associated with CRISPR technology is simply staggering. Hands down, this is one of the most exciting investing opportunities I’ve seen in my 20-plus year career in high technology.
Editor, Exponential Tech Investor
P.S. Genetic editing represents the biggest medical breakthrough since antibiotics. It has the potential to cure countless diseases. And of course, early investors have the chance to make a fortune. I recently identified three small-cap technology stocks that are primed to soar as genetic editing rewrites medicine. Details here.