Some Amazing Gene Editing Companies Rising to the Top Right Now!
18 million people are living with cancer everyday. 250 million people are living with sickle cell anemia everyday. 425 million people are living with diabetes everyday. And millions more and living with many other diseases. But, what if these diseases no longer existed?? What if they were all cured?!
Well, wait no longer, because that’s exactly what gene therapy treatments are trying to do right now! Read on to learn a little about three of these major biotech companies!
CRISPR Therapeutics
CRISPR Therapeutics is such a cool company! As some background, it is a public company whose IPO valuation is about $590.4 million. That’s crazy! That much money can buy 246,000 of the newest 16 inch Macbook Pros.
Imagine if all serious human diseases were solved. Millions of lives would be saved. And to add more ice to the coolness, CRISPR Therapeutics is using gene based medicines, including CRISPR/Cas9 technology, to create effective and efficient treatments for tons of diseases like diabetes, cancers, and hemoglobinopathies. Their innovative and sustainable ideas are revolutionizing the ways that we use gene editing and gene therapy.
Let’s talk about some of their coolest current projects. First, the treatment for sickle cell anemia (a single gene disorder that causes red blood cells to die quickly due to being misshapen and having poor functionality) has reached clinical trials. The CTX₀₀₁ program is an ex vivo treatment that uses CRISPR/Cas9 to edit a patient’s blood stem cells to increase fetal hemoglobin expression. With this improved hemoglobin production, symptoms should be permanently reduced or eliminated. The same process is also used for Beta Thalassemia (another single gene disorder that reduces the amount of hemoglobin produced by the body).
They are also using CAR-T cell therapies to combat cancer. They use a healthy donor’s T-cells that are edited to ensure safety, consistency, and efficiency. They add the CAR complex directly to the TRAC locus in the T-cell using CRISPR/Cas9, which allows the cell to identify and kill cancerous cells. They also use CRISPR/Cas9 to remove the T cell receptor and the class 1 major histocompatibility complex so that the patient’s cells and donor’s cells do not reject the other as a foreign substance. Their three products (CTX₁₁₀, CTX₁₂₀, and CTX₁₃₀) are all advanced and accurate ways to target cancer.
More than 100 million U.S. adults have diabetes. CRISPR Therapeutics could bring this number to 0. In an ex vivo treatment, they are using gene edited stem cells that act as pancreatic cells to restore the production of insulin!
Everyday this amazing company is working to advance the realities of the gene editing world, and they are definitely one of the coolest futuristic companies right now!
Editas Medicine
Editas is one of my favorite advancing gene editing companies! They use CRISPR/Cas9 and CRISPR/Cas12a (Cpf1) to develop treatments for genetic diseases. Their headquarters are in Cambridge, MA — the gene editing hub — and the company is growing rapidly!
They focus on three main disease sectors: ocular diseases, blood diseases, and cancers:
- Ocular Diseases: One very cool developing medine is the Edit-101 medicine that cuts out the CEP290 gene mutation that results in Leber Congenital Amaurosis (results in many irritating symptoms relating to the eyes). They use gene editing to repair the mutation in chromosome 12 safety and accurately. LCA is the leading cause of childhood blindness and there are no current treatments, so this developing medicine could be a great treatment. Usher Syndrome 2A is another genetic disease that results in blindness and deafness, and Editas is working to edit the USH2A gene in chromosome 13. The last ocular disease being worked on is Retinitis Pigmentosa, a rare genetic disease that results in gradual loss of vision. There are over 50 possible mutations and no current treatment, but using genome sequencing and targeting specific mutations, Editas could create a viable treatment option.
- Blood Diseases: Like CRISPR Therapeutics, they are working on Sickle Cell Anemia and Beta Thalassemia. Whether the blood cells are misshapen (Sickle Cell Disease) or hemoglobin is not produced (B-Thalassemia), body cells are unable to get enough oxygen and there is no current treatment. Using gene editing, the single gene mutations in these disorders can be reverted and symptoms will be gone!
- Cancer: Editas is using CRISPR medicines and stem cells to identify and destroy tumors in the body that lead to cancer. Using either a patient’s own cells (autologous treatment) or a healthy donor’s cells (allogeneic) and editing the cells to enhance their function, a patient’s immune system can be boosted and cancers can be detected and removed early!
They have already begun clinical trials in the ocular disease sector and are working to reach this stage for other diseases as well. To learn more about Editas, watch my quick presentation here: Ria Dani — Editas Presentation.
Intellia Therapeutics
Intellia Therapeutics is also a super innovative and interesting company! Similar to CRISPR Therapeutics and Editas, Intellia is developing gene editing treatments to positively impact people living with severe and life-threatening diseases. They are using in vivo and ex vivo treatments to treat diseases such as Hemophilia A and B, Sickle Cell Anemia, Leukemia, and other genetic diseases that haven’t been published yet.
One of their most developed treatments is the in vivo therapy for transthyretin amyloidosis (ATTR), a fatal disease from buildup of the amyloid protein in the body’s tissues and organs. Their process called NTLA-2001 uses CRISPR/Cas9 to restore normal functionality and this process has been funded and the initial trials have been successful.
They have also developed NTLA-2002, a in vivo treatment for hereditary angioedema (HAE). This rare genetic disease causes swelling from an excess amount of the bradykinin protein produced by the prekallikrein B1 (KLKB1) gene, so using their treatment, a knockout of this gene should reduce the severe swelling and other dangerous symptoms caused by the disorder.
Additionally, they have worked on creating an in vivo treatment for Hemophilia, a genetic disorder that causes a protein called Factor VIII (Hemophilia A) or Factor IX (Hemophilia B) not to be produced and secreted in sufficient amounts, resulting in blood not being able to clot. This disease results in internal bleeding and other dangerous symptoms, and the more common type, Hemophilia A, affects 1 in 5,000 men. With their partner, Regeneron, they are working to use CRISPR to begin treating Hemophilia B, and hopefully Hemophilia A soon.
- To learn more about genetic engineering being used for treatment of Hemophilia, check out my article (Using Gene Editing to Treat Severe Hemophilia A) or video (Treating Severe Hemophilia A Using TALENs And IPSCs)!
Their ex vivo treatment for Leukemia is also rapidly developing! Acute myeloid leukemia is a type of blood and bone marrow cancer that causes myeloid cells to inhibit the production of blood cells and platelets. The disease is rare, but the symptoms and current treatments (chemotherapy, radiation, etc.) are painful. Intellia is using engineered T-cells to identify the Wilms’ Tumor 1 so that the tumor can be identified quickly and accurately. Using TCR T-cells or Car-T cells, the patient’s immune system can locate and attack cancers.
Through exploration of new ideas and developing innovative solutions, Intellia is also working on creating treatments for various genetic diseases!
There are a ton of other incredible gene editing companies such as Mammoth Biosciences (https://mammoth.bio), Twist Bioscience (https://www.twistbioscience.com), and Sangamo Therapeutics (https://www.sangamo.com), for example. I focused on the top three in my area, but be sure to check out local and smaller scale gene editing companies as well to learn more about the crazy developments occurring right now!
This article was a little repetitive, but it’s a huge advantage to have all of these companies working towards almost identical missions. With so many revolutionary companies working to treat these genetic diseases, once thought to be untreatable, there is so much hope that we will soon reach a future where they are all cured! If you’re looking for a place to invest your money into, I highly recommend looking at gene editing and biotech companies — they’re creating a future we should all be looking forward to!
Thanks for reading :) If you ever want to chat or have any questions, be sure to reach out to me at my email, riadani27@gmail.com!
