CRISPR, CRSP, and Tomatoes

Jennifer Doudna received a Nobel Prize in 2020 for her role as a co-inventor of the CRISPR-Cas9 gene editing technology. Earlier this month Dr. Doudna spoke with the New York Times about her career, CRISPR, and how gene editing may impact our future.

We recommend you read her interview in full to glean some insight into the innovations already taking place in medicine and food production, as well as some of the key ethical issues surrounding gene editing.

First things first, though. What is CRISPR?

What is CRISPR?

CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. That is quite the mouthful! CRISPR is a bit like keywords in an e-book: in the same way that looking up 'Chapter 2' might help you find the beginning of that chapter, scientists can use keywords in the DNA of a cell to find specific portions of its genome. A more detailed discussion of CRISPR can be found here.

CRISPR-based tools can be used to identify and cut specific points in a DNA strand. Using these tools, scientists can pinpoint specific letters in the genetic code of a cell that they would like to modify, such as a mutation that causes a deadly disease, and edit them away. Powerful stuff.

Dr. Doudna won the Nobel prize for her work on CRISPR-Cas9, a landmark evolution that made straightforward gene editing possible. She and her co-authors published their invention in the prestigious Science journal in 2012, a mere ten years ago.

While new gene editing techniques are invented every year, CRISPR-Cas9 remains the basis for a number of potentially life-saving therapies. Further, a number of companies have been founded since 2012 to pursue various therapeutic modalities using CRISPR-Cas9.

Here is one example.

CRISPR Therapeutics

The aptly named CRISPR Therapeutics, (NASDAQ: CRSP) is a publicly traded company co-founded by Emmanuelle Charpentier, who received the Nobel Prize alongside Dr. Doudna.

CRSP is pursuing CRISPR-Cas 9 therapies across:

• Red blood cell disorders (hemoglobinopathies) such as β-thalassemia and sickle cell disease

• Immuno-oncology (engineering immune cells to kill cancer cells) targeting forms of leukemia, lymphoma, and solid tumors

• Regenerative medicine (using stem cells to repair diseased and damaged tissue)

• In vivo therapies (modifying organs of live patients to cure genetic diseases such as Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF)).

CRSP, like any drug development company, is realistically only likely to succeed in a small fraction of these development programs. Still, even one or two approved therapies would be monumental advances in modern medicine!

There are many other CRISPR-based companies pursuing novel therapies today. Some publicly traded examples include Intellia Therapeutics (NASDAQ: NTLA), Editas Medicine (NASDAQ: EDIT), and Caribou Biosciences (NASDAQ: CRBU). Please reach out to us if you would like to learn more about investable opportunities in this exciting field.

The potential of CRISPR goes beyond therapeutics, however. Dr. Doudna pointed out in her interview with the New York Times that CRISPR is already being used for food and agriculture, amongst other uses.

Here is a somewhat humorous example of the applications of CRISPR beyond the clinic. Much more to come.

The CRISPR Tomato

In September 2021, a CRISPR-edited tomato became the first gene-edited food approved and sold for human consumption.

A company called Sanatech Seed received approval in Japan to market a tomato that is genetically edited to contain high amounts of Gamma-Aminobutyric Acid (GABA). Dietary supplements and foods rich in GABA are popular in Japan, where it is believed to promote low blood pressure and help people relax.