Intellia Therapeutics ($NTLA) shocked the gene-editing field with the announcement of data from the first-ever human study using CRISPR gene editing therapy. Intellia and partner Regeneron ($REGN) announced data from a phase I study evaluating their CRISPR genome editing candidate, NTLA-2001, that showed promise in patients with transthyretin amyloidosis (ATTR). ATTR is a deadly disease that affects the nervous system, destroying nerves and eventually crippling and killing those who suffer from the disease. Remarkably, patients with ATTR were given a single dose novel treatment which led to an 87% reduction in TTR levels, going up to 96% reduction after a month. In comparison, the current treatments options for ATTR have show reductions of TTR levels of about 80%. This news made Intellia shares nearly double, while other CRISPR gene editing therapies saw their shares soar as well with investors flocking to the potential of the gene editing. The technology works by injecting patients with CRISPR-loaded nanoparticles, which when injected alter a gene in the organ to halt the production of the proteins causing the disease. However, this gene-editing breakthrough may have huge potential in treating other deadly illnesses such as Alzheimer’s or cancer. With CRISPR technology, scientists may be able to eliminate or repair the source of the disease within their patient’s bodies. Investors looking to invest in a company researching CRISPR genomic editing should look at Intellia Therapeutics ($NTLA), CRISPR Therapeutics ($CRSP), and Editas Medicine ($EDIT)
Intellia Therapeutics ($NTLA)
With Intellia being the first to prove the potential of CRISPR technology in humans, investor sentiment is at an all-time high. In the past, CRISPR technology was limited to editing cells outside the body or in the eye. Now in the most recent trials of NTLA-2001, researchers at Intellia were able to genomically edit nanoparticles, and then inject them back into the body with a single dose. This was the first time researchers were able to successfully target the gene causing the disease en vivo (inside the body). This milestone bodes very well for the future of genomic editing.
With that being said, trials have only been conducted on 6 people, and it’s too early to say that the CRISPR treatment will completely eliminate the symptoms of the disease. Currently, NTLA-2001 has shown to be effective in patients with transthyretin amyloidosis, which is a hereditary disease affecting approximately 50,000 people worldwide. However, the drug may have much more widespread applications for other single-cell amyloidosis diseases. Currently, an estimated 250,000-550,000 people suffer from some form of amyloidosis. Intellia and other CRISPR gene-editing companies’ technology looks very promising, but they will have to compete with other treatments for TTR amyloidosis. One thing that sets CRISPR technology apart, however, is its simple and effective usage in comparison to other treatments on the market. One of these competitors, Alnylam is a company with an RNA-based therapy that requires an infusion every three weeks. Another competitor, Ionis Pharmaceuticals requires regular injections as well, although they can be self-administered. Both treatments are priced at about $345,000 per year, and Alnylam ($ALNY) has added cost when taking into account medical office visits to get the infusions.
Intellia’s one-time dosage gives the company a clear edge over its competitor and may become the preferred option for patients and administrators, due to its fast and effective use. While amyloidosis treatments may seem like a niche market, Alnylam was able to generate $306 million in sales and Tegsedi was able to generate $70 million. Things look even better for Intellia when considering the premium patients will pay for a one-time infusion. Bluebird Bio ($BLUE) another gene therapy company, has a one-time treatment that costs about $1.8 million. All things considered, Intellia’s TTR amyloidosis treatment has the potential to make the company billions of dollars.
Intellia Therapeutics is looking to be an early leader in CRISPR gene editing however, investors will need to remember that these results are from a very early phase 1 trial and the company valuation has skyrocketed to a market cap of roughly $11 billion. The company is up 550% year to date and is already starting to correct, down 10% from all-time highs. With that being said this technology could be ground-breaking for treating a multitude of diseases, especially those that are based on a singular gene. The company also has a very intriguing pipeline, with NTLA-2001 showing really good early results in their in vivo category. On the other hand, their CRISPR technology is looking really promising in autoimmune diseases in their ex vivo pipeline. These are drugs made using CRISPR outside the body. Intellia is currently running clinical trials for QTQ923 which is a CRISPR-based treatment for sickle cell disease, which affects about 70,000 to 100,000 Americans. The company is also getting close to running early clinical trials on NTLA-5001, which is a treatment for Acute Myeloid Leukemia (AML). There were 20,000 new cases of AML in 2018 alone, with the 5-year survival rate being under 30% making it one of the deadliest diseases in the world. Intellia is looking to use its CRISPR technology to precisely edit a patient’s immune cells and reinject them into the body. While this may sound like a long shot, if this technology is able to train the body to stop tumor antigens, it would be the biggest innovation in medicine of the 21st century. Intellia is still very early on and will need several years to conduct several years of trials and continue to innovate to get an edge on its competitors. However, if the company is able to stay at the top of CRISPR gene-editing technology the sky is the limit.
CRISPR Therapeutics ($CRSP)
CRISPR Therapeutics is a gene-editing company focused on developing transformative gene-based medicines for serious human diseases. The company’s lead pipeline candidate is CTX001, which has the potential to treat transfusion-dependent beta-thalassemia (TDT) and sickle cell diseases (SCD). The company struck a deal with Vertex Pharmaceuticals to collaborate on the treatment which is currently in mid-stage studies. CTX001 looks very promising with the gene-editing therapy showing consistent and sustained response to treatment in the ongoing clinical trial involving 22 patients tested so far. About 120,000 infants are born with SCD every year worldwide, with much of it being hereditary. Many born with the disease don’t end up getting symptoms, but those that do can suffer muscle breaking down, glaucoma, and in rare cases kidney cancer. CXT001 is very unique because it seeks to edit a patient’s own blood stem cells and reinject them into the body to stop symptoms. The results so far are very compelling, with none of the patients with TDT requiring blood transfusions and all of the SCD patients free of Vaso-Occlusive Crisis, which is the most frequent complication in patients. With the potential of CXT001 becoming a blockbuster drug, Vertex pharmaceuticals revised its agreement to pay $900 million upfront for a 60% cut of sales. With gene treatments being very new and innovative technology, a dose of CTX001 could be between $1-2 million dollars. If CRISPR could treat just 1% of the 70,000 people suffering from SCD this could be $700 million in revenue. CRISPR therapeutics lead candidate CTX001 has the potential to become the go-to treatment option for patients with SCD and TDT. However, they will need to continue to wow investors with compelling results if they want to beat out the countless other companies with similar products.
CRISPR also boasts 3 other candidates in their immuno-oncology pipeline that they currently own 100% of the rights to. One of these treatments that the company is developing is CTX-110, an immunotherapy treatment consisting of gene-edited, donor-derived cells for fighting cancer. During an interim phase 1 data release, the study found that four out of 11 patients with aggressive non-Hodgkin lymphoma developed complete responses after receiving CTX-110 infusions. In other words, a complete response means that there was a disappearance of all tumor activity after the treatment was given. In the coming months, the company would see its stock price nearly double with prices as high as $210 per share. Since then the stock has corrected and fallen 31% from all-time highs and 10% from the Intellia news which boosted the gene-editing market. Despite this, CRISPR is still up an impressive 65% year to date and may still have room to grow. The company is also independently developing cell therapies using CAR-T receptors to cure B-cell cancers, which if successful can be a major breakthrough in cell therapy research. Currently, all approved cell therapies need to be manufactured every time based on a patient’s own cells. However, this breakthrough could allow CAR-T therapies to be manufactured on large scale, resulting in lower cost and could propel the company to become the market leader in CAR-T therapies. If future trials can continue to bring good news, CTX110 can be a major revenue driver for the company. CRISPR Therapeutics has one of the most compelling pipelines in the gene-editing space, their own problem is they have yet to make any recurring revenue. The company also has 5 consecutive quarters of increasing its spending losing $348 million in 2020, with $220 million of that just in the last 2 quarters. The company is burning through cash quickly, but thankfully the revised agreement with Vertex will help alleviate some of that. While CRISPR products look promising, they are still years away from a product and there is no guarantee they will get FDA approval. With the company sitting close to an $11 Billion dollar market cap and a very volatile stock chart, this may scare off investors. With that being said, the company has tremendous potential, and if it’s able to get approval for CTX001 or CTX110 the stock will almost certainly go to all-time highs.
Editas Medicine, Inc ($EDIT)
Editas Medicine is a gene-editing company harnessing the power of CRISPR to develop a variety of treatments for people living with serious diseases. Unlike Intellia and CRISPR, Editas has an ocular focus, with a pipeline of gene-editing therapies aimed at eradicating the inherited retinal disease. The company’s lead pipeline candidate is EDIT-101, which utilizes CRISPR gene editing to treat LCA10, a rare genetic illness that causes blindness in children. The company is currently undergoing the first pediatric testing in its phase I/II BRILLIANCE study, which is evaluating EDIT-101 effectiveness in treating LCA10. Back in May, the brilliance study was the first CRISPR study to change DNA in a living human. A surgical procedure was done in an attempt to prevent blindness from a patient suffering from retinal dystrophy. The disease is a genetic disease that can cause complete blindness in patients suffering from it. During the procedure, scientists were able to successfully snip out abnormal DNA in the cells causing retinal dystrophy. This was a groundbreaking achievement for gene-editing proving in vivo, or within the body, treatments were possible. The company finished up initial dosing in its first group of patients in earlier-stage human trials. Editas expects to report initial data from its phase 1/2 study of EDIT-101 in September, which with good results should bode well for the stock price. The company also has an early study on EDIT-102 which is a treatment for Usher Syndrome (USH). USH is a condition that causes partial or total vision and hearing loss. It is estimated that 3-6% of all childhood deafness and 50% of children that are deaf and blind suffer from the disease. With promising results from EDIT-101 and the potential in EDIT-102, the company’s ocular focus could be groundbreaking in curing blindness in children. While the company’s main focus is on ocular diseases, Editas also recently commenced a phase 1/2 study of EDIT-301 in treating sickle cell disease. The drug had previously looked like a best-in-class for people living with sickle cell disease, driving the stock to $90 per share. However, investor sentiment quickly went down after a partial clinical hold, which the FDA wanted an improved potency test. This was quickly cleared up, but with other competitors and a stock correction, the company hasn’t been able to recover from all-time highs.
Sitting at a mere $3.3 Billion market cap, Editas has a much lower valuation when compared to peers. The company is down 45% from all-time highs and down 15% from the Intellia news boost. Still impressively the company is up nearly 40% in just a month and looks to be a lot cheaper than its peers. While the company is still losing money, its revenue numbers are looking good with $90 M in revenue in 2020 up 341% year to date. In contrast, this revenue is nearly double Intellia’s while boasting a market cap 3 times less. The company is still spending money at a high rate, similar to other gene-editing companies. However, funding has actually grown from about $140 million in December to $354 million in the first quarter of 2021. While the company may not have pipelines as lucrative as other gene-editing companies, the company has found a very strong and compelling niche in ocular research, especially in cases with children. Editas Medicine still has the potential to crash and burn; with even one failed clinical study investors will certainly flock to other lucrative gene-editing stocks. However, with the potential to cure hereditary blindness the company looks very promising.
CRISPR gene-editing technology is still very early on and relatively unheard of by most people. Still, if the technology can continue to innovate and produce impressive results, the gene-editing space could grow to be a trillion-dollar sector. The potential to edit genes on the fly and reintroduce them into the body to cure rare diseases sounds like something straight out of science fiction. It will be up to investors to figure out which company will create the first successful gene-editing treatment for humans. However, as positive research continues to come out it’s seeming like gene-editing could become too lucrative to pass up.
DISCLOSURE: The Sick Economist owns shares in $EDIT