CRISPR-Cas9 is a technology that allows scientists to change the DNA of an organism (including humans). This is likely one of the most important technological breakthroughs of the last 100 years and brings with it a lot of ethical questions but is incredibly powerful.
One of the first use cases of this technology will be to cure existing disease. While there may be ethical questions around changing DNA, it's hard to argue removing a lifetime of suffering from a curable genetic disease.
Beta Thalassemia is one of these diseases potentially requiring frequent blood transfusions. Someone suffering from this disease may not live a normal life span and treatment costs can be in excess of a million dollars per patient.
Sickle Cell Disease is another one which can cause chronic pain, vision problems, and frequent infection. Lifetime treatment costs are around a million dollars a patient.
Patients have been effectively cured from both of these diseases using CRISPR. This is incredible and likely just the beginning. It shows the promise of this technology is not just in early research anymore. It’s been used in live patients and it Works.
Some other diseases that Cas9 may be able to treat or cure include:
Transthyretin (ATTR) Amyloidosis
Hereditary Angioedema
AATD-Lung Disease
Hemophilia B
Hemophilia A
Acute Myeloid Leukemia
Leber CongenitalAmaurosis 10
UsherSyndrome 2a
Autosomal DominantRetinitis Pigmentosa 4
Various Cancers
Muscular Dystrophy
Myotonic Dystrophy
Cystic Fibrosis
How to Invest in the Space
I split my bets across the various companies that have access to the CRISPR patents and have research pipelines attacking the above diseases. My current investments include holdings in:
I also have investments in general genetic testing infrastructure including:
The cost of DNA sequencing has reduced dramatically from 100 million dollars to sequence the human genome to ~$1000.
I’m a big believer that the future of medicine is personalized, and that this space has a lot of growth ahead. To me, this is easily a trillion dollar space. This isn’t currently reflected in the market cap of these companies.
Disclaimer: This does not constitute financial, investment, tax, legal or accounting advice nor does it constitute an offer or solicitation to buy or sell any securities referred to. Individual circumstances and current events are critical to sound investment planning; anyone wishing to act on this article should consult with his or her advisor.