Posted by on January 11, 2021 10:10 am
Categories: CRISPR-Google

CRISPR, Editas, Intellia: Gene Editing Stocks To Play The Next Revolution In Medicine – Forbes

Gene editing has emerged as a promising biotech theme. The technology is used to insert, edit, or delete a gene from an organism’s genome, helping to replace the defective genes responsible for a medical condition with healthy versions. This technology is being used to develop treatments for a range of diseases from cancer to rare genetic conditions, that are otherwise hard to treat, and is also being considered for diagnostic purposes. While there are broadly three gene-editing technologies, “clustered regularly interspaced short palindromic repeats” or CRISPR, as it is popularly known, has emerged as the method of choice with most companies, considering that it is relatively inexpensive, simpler, and more flexible compared to other tools such as ZFN and TALEN.

While most gene-editing players remain in the clinical stage with a limited financial track record, funding has risen meaningfully and larger pharma companies are also partnering with these companies, considering that the treatments could be lucrative and the broader technologies may be highly scalable. While the upside remains large, investing in these companies is risky. Being a new technology that has never been used in humans before, there are risks of significant side effects or of the therapies not being effective. The economics of producing and selling these drugs also remains uncertain. These stocks are also volatile, seeing big swings as any new research or data on their potential or risk is outlined. Our indicative theme on Gene Editing Stocks – which includes names such as CRISPR Therapeutics, Editas Medicine, and others – has returned about 230% over the past 2 years, compared to the broader S&P 500 which is up by about 52% over the same period. Below is a bit more about these companies.

CRISPR Therapeutics AG is one of the best-known names in the gene-editing space. The company is working with Vertex Pharmaceuticals

to co-develop CTX001, an experimental gene therapy that has provided promising results for people with sickle cell disease, and transfusion-dependent beta-thalassemia – disorders that affect the oxygen-carrying cells in human blood. The company is also developing cancer therapy candidates independently. The company was profitable last year, due to collaboration revenues from Vertex.


Editas Medicine, another leading CRISPR-focused biotech company, with a flagship program, EDIT-101 is targeting the treatment of hereditary blindness. The company recently finished dosing for its first group of patients in earlier-stage human trials. The company also recently filed a request with the U.S. FDA to commence phase 1/2 study of EDIT-301 in treating sickle cell disease. The company also has multiple other pre-clinical drugs focused on genetic diseases.

Intellia Therapeutics is developing a drug for a rare and fatal disease known as transthyretin amyloidosis in collaboration with Regeneron. The drug is in phase 1 trials currently. The company is also working on ex-vivo Sickle Cell Anemia treatment with Novartis that involves editing cells outside the body before infusing them into the patient. The candidate is entering Phase 1/2 trails. While the company has 8 other candidates, they are still in the research or pre-clinical stages. [1]

Sangamo BioSciences focuses on multiple areas in the genomic medicine space, including gene therapy, cell therapy, in vivo genome editing, and in vivo genome regulation. The company pioneered the zinc finger nuclease gene-editing method. The company’s most advanced development is a treatment for Hemophilia A, which is being developed with Pfizer

and is in phase 3 trials. The company also has 4 candidates in the phase 1/2 stage and 13 in the Preclinical stage. [2]

While Gene Editing stocks might offer much upside, it comes with considerable risk at current prices. What if you’re looking for a more balanced portfolio instead? Here’s a high-quality portfolio to beat the market, with around 130% return since 2016, versus about 65% for the S&P 500. Comprising companies with strong revenue growth, healthy profits, lots of cash, and low risk, it has outperformed the broader market year after year, consistently.

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Published at Mon, 11 Jan 2021 15:00:00 +0000