Read on Twitter
1. It’s hard to oversell the promise of CRISPR. It’s one of the most fundamental discoveries of the 21st century. It essentially leverages an infection-fighting mechanism in bacteria to edit DNA. This thread is a quick summary of the companies trying to use it to treat diseases.
2. There are two main camps behind the tech. One is led by Jennifer Doudna and Emmannuelle Charpentier who won the Noble prize in 2020 for their work. Second is led by Feng Zhang and his team at the Broad Institute at MIT and Harvard.
3. They have all built companies to productize the technology. The main ones are - CRISPR Therapeutics by Charpentier, Intellia Therapeutics by Doudna and Editas Medicine by Zhang. None of them have any material revenue. All of them hold a lot of promise.
4. CRISPR Therapeutics, $CRSP, is the biggest by market cap at ~14B. It has 300 employees. It’s relatively ahead in developing treatments. It’s already enrolling people for clinical studies for its treatments for sickle cell anemia and beta-thalassemia, and ...
... some immuno-oncological treatments. It’s also working with a partner, ViaCyte, on a stem cell therapy for diabetes.
5. Intellia Therapeutics, $NTLA, is valued at 4.8B and is backed by Bill and Melinda Gates Foundation. It already has its first patient dosed with a treatment. It expects application of human trials for ex-vivo treatment of acute myeloid leukemia and, later, solid tumors.
6. Editas Medicine, $EDIT, is valued at 4.13B. It’s also working on sickle cell anemia and beta-thalassemia, and on oncological treatments with partners. It’s working on some vision related problems which is unique among these companies.
7. $EDIT also employs Cas12a / Cpfl in addition to the Cas9 variant which is supposed to have better efficiency and accuracy for certain forms of gene repair.
8. Beam Therapeutics, $BEAM, is another company that came out of the Zhang camp. Valued at ~6b, it’s supposed to have a better technique for single base editing which minimizes “off-target edits” and avoids the unwanted consequences of double-stranded DNA breaks...
... Beam is also starting with the focus on sickle cell anemia. It licenses its tech to other companies to focus on specific problems.
9. One of which is Verve Therapeutics which recently showed it could lower cholesterol (LDL-C) levels in primates with a single gene editing treatment. Basically it inactivates a gene in the liver by changing a single base from A to G which increases the amount of LDL receptor...
... which in turn lowers the levels of LDL-C. This can cure a potentially fatal genetic heart disease called HeFH. https://www.vervetx.com/press-releases/verve-therapeutics-announces-data-demonstrating-durable-ldl-cholesterol-lowering-after-a-one-time-gene-editing-treatment-in-non-human-primates/
10. FDA approved the first gene therapy in 2017. This introduced genes temporarily in the cells to stimulate them to create specific proteins. Permanent gene-editing trials may not see approvals until 2023/24. It’s going to be some time before we start realizing the benefits.
11. Sickle cell anemia itself is expected to be a $4.8b / year market in the USA.
