Cathie Wood is the CEO/CIO of ARK Invest, which include the wildly successful and growlingly popular ARKK Innovation ETF and ARKG Genomic Revolutions ETF. In an interview with Bloomberg, Woods recently said: “The biggest upside surprises are going to come from the genomic space, and that’s because the convergence of DNA sequencing, artificial intelligence, and gene therapies are going to cure disease”. This interview helped propel many biotech stocks in the gene therapy space. Today we’re looking at two of our favorite CRISPR stocks:
Editas Medicine (EDIT) is a CRISPR/Cas9 gene editing company, along with CRSP, NTLA, BEAM. These CRISPR companies are perusing treatments of genetic diseases, by a process which uses a Cas9 protein to find a specific DNA sequence, and then cuts it at that location and inserts new DNA in place. This is of course a very crude summary, but can help provide context on the technology.
Editas has a large pipeline of several potential treatments and applications of its technology. Currently, one is in Phase 1/2 clinical trial and is attempting to target the mutant genes associated with genetic blindness. As of now, there are no cures or effective treatments for this type of blindness. This is also the first time an in-vivo CRISPR treatment has been done as part of a clinical study. The in-vivo CRISPR approach is administered directly delivered into the patient and the gene editing occurs within the person’s body. While ex-vivo, the other method of delivery for these type of treatments, relies on first taking the patients cells, and editing them outside the body, before transplanting them back into the body. If all goes well in trial, Editas will be the clear leader of in-vivo CRISPR treatments.
The company’s pipeline also includes EDIT-301, which targets to treat Sickle Cell Disease. EDIT-201 for the treatment of solid tumor cancers. Editas also has other treatments for both ex-vivo and in-vivo treatments and has partnered with other companies to develop and research these potential treatments.
Editas has secured enough cash to be run through 2023 - but make no mistake, this is a pre-revenue company.
Beam Therapeutics (BEAM) is up 275% since its February 2020 IPO and has more than doubled over the last month.
Beam is a very promising biotech company developing precision genetic editing medicines, utilizing the very hot CRSPR technology. The company was founded by David Liu, a pioneer in the field, who previously founded Editas(EDIT) - another leader of the CRISPR biotech space. Beam stands out in this field by its use of base editing which can target single edits in DNA with great precision with chemical reactions. Other companies in the field like CRSP, EDIT, NTLA use genetic editing which make double-stranded cuts in the DNA, and while that technology is farther ahead, has technical issues that still need to be overcome – primarily unintended cuts in the DNA which may have unintended consequences like cancer. But due to the greater efficiency in base editing technology, it generally has more precision and less issues with unwanted DNA changes to overcome. Though these issues are not completely unheard of with base editing either and more work is needed especially since this is a newer technology.
While there are many diseases that BEAM could use this technology to potentially address, the most immediate one is sickle cell anemia which is caused by a point mutation, exactly what base editing could one day treat more efficiently than the more involved gene editing approach. In fact, BEAM is currently pursuing two different base editing approaches to treat red blood cell disorders.
This site references only our opinion and is for information purposes only. It is not intended to be investment advice. Seek a duly licensed professional for investment advice. Disclaimer: We are long BEAM and EDIT stock and options.