Investing in Biotech: CRISPR Technologies

What is CRISPR?

Released in 2012, CRISPR-Cas9 (known as CRISPR) is a technology for gene editing- artificially altering specific genes of an organism. Although it is not the only technology to edit genes, it is the cheapest, easiest, and most precise.

How does CRISPR work?

CRISPR has two parts: CRISPR and Cas9. Cas9 is an enzyme that cuts strands of DNA- it is a pair of molecular scissors. CRISPRs are short sequences of DNA or RNA that tell the Cas9 where on the DNA to cut.

The CRISPR-Cas9 parts that are used by scientists were adapted from bacteria, who use CRISPR-Cas9 to fight viruses. In the lab, scientists attach RNA sequences (the CRISPR part) to a Cas9 enzyme. Using the RNA (CRISPR) sequence as a guide, the Cas9 finds the target part of the DNA and cuts it out. Scientists then hack the cell’s own DNA repair machinery to finish the process. For example, if they were looking to replace a gene, they will use the CRISPR-Cas9 to locate and cut out the gene, and then use the cell’s own repair machinery to insert the replacement gene.

What does this have to do with investing?

CRISPR is a promising candidate for curing, treating, and preventing many human diseases. Currently, there are over 1,700 CRISPR-related patents, and around 100 new ones are filed per month. CRISPR Therapeutics, Intellia Therapeutics, and Editas Medicine own most CRISPR patents regarding human therapeutics.

Gene editing cannot be used fully on an alive human, because we have way too many cells to edit them all. However, it can be used on a targeted group or type of cells in a person. For example, to cure a blood disease, scientists could take out some red blood cells from a person. In the lab, they would edit these cells, then re-inject them into the person. These cells would eventually multiply, which would produce more edited cells.

The only way to edit every cell of a person would be to do it before they are a person. The genes could be edited in the embryo stage, and then the edited genes would multiply to make the full person. The US bars the use of gene editing on embryos, so this is currently not an option.

CRISPR Therapeutics

Money raised: 162 million dollars

Promising treatments:

• Total, CRISPR has nine drug candidates

• CTX001 is a candidate to treat sickle cell and transfusion-dependent beta-thalassemia (TBT). In these diseases, red blood cells are malformed, and cannot deliver adequate oxygen to the body. In 2019, CTX001 successfully eliminated symptoms in a patient with sickle cell and in a patient with TBT.

• CTX110, CTX120, and CTX130 are candidates for a cancer treatment called CAR-T therapy.

Current share price (as of 11/26/20): $110.00

Market cap (as of 11/26/20): 7.772 billion. Market cap is the value of a company in the stock market- the number of shares times the price of each share.

Intellia Therapeutics

Money raised: 85 million dollars

Promising treatments:

• NTLA-5001 is a drug that could treat acute myeloid leukemia (AML). AML is a rare kind of cancer that causes the bone marrow to produce abnormal red and white blood cells.

• NTLA-2001 is a potential treatment for transthyretin amyloidosis, which causes the “loss of sensation in extremities and in internal organs.”

Current share price (as of 11/26/20): $34.45

Market cap (as of 11/26/20): 2.119 billion

Editas

Money Raised: 163 million dollars

Promising Treatments:

• EDIT-101 is a potential treatment for Leber congenital amaurosis, a disease that causes blindness. EDIT-101 successfully treated a blind patient in a clinical trial.

• EDIT-301 is a potential drug for treating sickle-cell disease and TDT (like CRISPR’s CTX001).

• EDiT-102 is being developed to treat Usher Syndrome 2a, which causes hearing loss and vision loss in teens and adults.

Current share price (as of 11/26/20): $29.02

Market cap (as of 11/26/20): 1.836 billion. Market cap is the value of a company in the stock market; the number of shares it has multiplied by the price of each share.

CRISPR’s December 30th, 2019 Stock Dive Explained

Between December 30th, 2019, and March 9th, 2020, all three companies’ stocks took a large dive. This massive dive happened because He Jiankui, a Chinese scientist, was sentenced to 3 years in prison for making “CRISPR babies.” Jiankui implanted “gene-edited embryos into two women.” The biological father of each embryo had HIV, while the mother did not. In an attempt to prevent the babies from getting HIV, Jiankui disabled the CCR5 gene- he disabled a gene that allows HIV infections to take hold. On December 30th, 2019, He Jiankui was sentenced to 3 years in jail for “illegal medical practices,” which caused the big dive in all three CRISPR stocks.