Editas Stock: Tackling Rare Eye Disease With Crispr Technology (NASDAQ:EDIT)

Editas (NASDAQ:EDIT) I believe has potential to be huge in the Crispr gene editing space. What’s intriguing about this biotech is that it offers a lot of options in its pipeline in terms of variety. What I mean by that is the ability to use two types of Crispr technologies for starters. One type is Cas9 and then the other is CAS12a. Not only that, but this is further expanded upon with the ability to use in vivo gene editing and ex vivo gene editing. The former deals with modifying a gene inside the body, while the latter deals with the ability to edit the gene outside of the body. The reason for this? Well, in order to target additional indications. If this company only had in vivo gene editing then it would be limited on the target indications it could go after. With having the ability to do both, then it can go after a lot more targets. It has even started to develop cellular therapies that go after various types of cancers both autologous (taking cells from patients’ body to modify them) and then allogeneic (donor cells taking from healthy donors not of the cancer patient themselves). It’s incorporating its cell therapy program with a technology known as SLEEK. Not only that, but it was a clear winner of the patent battle between Broad and CVC over patents for CRISPR technology. Although, this is not the only win it will need as it will likely drag out in courts for years to come.

Rare Eye Disease Treatment With EDIT-101

Editas Medicine is currently exploring the use of its in-vivo Crispr/Cas9 genome editing treatment known as EDIT-101 for the treatment of patients with Leber congenital amaurosis 10. LCA10 is a retinal disorder with the mutation of the CEP290 gene. What makes this a good target for the company is that the mutation of the CEP290 gene is found in up to between 20%-30% of all LCA patients. This is an inherited retinal disorder that leads to impaired vision or blindness. What also makes this a good target is the fact that there are no approved treatments for it. This means if Editas can somehow get through all of the clinical trials using EDIT-101, then it can have the ability to target the whole market all by itself. There are several symptoms that are associated with this disease such as:

• Decreased visual acuity
• • Photophobia (eye discomfort in bright light)
  • Nystagmus (involuntary side to side movement of the eyes)

• High hyperopia (Farsightedness)

The use of EDIT-101 is being explored in the phase 1/2 BRILLIANCE study. There will be a total of 18 patients with LCA10 who are to be given this Crispr/Cas9 treatment. They range between the ages of 3 to 17 with various baseline visual acuity assessments. Each patient will receive a single administration of EDIT-101 via subretinal injection in one eye. Patients will be looked at every three months for the first year, and then less often for an additional two years. The latest update for this study is that the biotech is enrolling patients in the mid-dose pediatric cohort. There’s a catalyst opportunity for investors to look forward to as well, because it’s expected that it will report results from this study in the second half of 2022.

Things are looking good for this program so far, however, it was mainly on the safety front with a bit of preliminary efficacy thrown in. That’s because Editas had released initial preliminary safety and efficacy data from the phase 1/2 BRILLIANCE study. This includes data from the first two cohorts only, which were as follows:

• Adult low-dose cohort 6×10¹¹ vg/ml
• Adult mid-dose cohort 1.1×1012 vg/ml

In terms of safety data, there were two patients from the low dose cohort that were evaluated and then about four patients from the mid-dose cohort that were evaluated. The adverse events that these patients experienced were only mild and mainly dealt with the actual surgical procedure itself and subretinal injection of the Crispr treatment with EDIT-101. The most important thing is that there were no dose limiting toxicities observed, which as I have always said up to now is very important. That’s because if no safety issues are found at this stage, then a company can push forward toward treating adult patients with a higher dose. Not only that, but in this case, it allowed Editas Medicine to push forward by moving toward its pediatric patient population as well. As far as efficacy goes two adults in the low dose cohort and three adults in the mid-dose cohort who had at least three months worth of treatment with EDIT-101 were evaluated upon a follow up visit. It was noted that these patients saw improvements in BCVA, FST, Visual function Navigation and other visual improvements. I’m not going to go out on a limb and say that these measures were statistically significant. This only proves that EDIT-101 is somewhat active, but the newer data to be released in the second half of 2022 is going to be very important in determining the true strength of CRISPR. That’s because it will let Editas know for sure if it can continue to advance this particular program or not. I would deem this EDIT-101 CRISPR program as being the most important in the pipeline, because it’s the most advanced program. As such, this new data to be released in the latter part of this year deals with results from specific cohorts which were activated by the Independent Data Monitoring Committee (IDMC) once the safety results from the phase 1/2 BRILLIANCE study were released. The specific cohorts you should expect are the adult mid-dose cohort and then the adult high-dose cohort. This is with respect to at least 12 months of data from this specific study. Again, this is a longer treatment period and it will give investors and the company a good idea on whether or not it is worth pursuing EDIT-101 for this indication.

Financials

According to the 10-Q SEC Filing, Editas had cash, cash equivalents and marketable securities of $566.4 million as of March 31, 2022. A big reason for the large influx of cash is because of an underwritten public offering of 3,500,000 shares of common stock at a public offering price of $66 per share. It even offered the underwriters a 30-day option to purchase up to an additional 525,000 shares of common stock at the very same price. Total gross proceeds from this company from this offering was around $231 million. It believes that this is enough cash on hand to fund its operations into early 2024. I think Editas has enough cash for now, but my feeling is that it will likely might raise cash at least by the end of 2022. It’s possible it could do so earlier if it reports some positive news development beforehand. Specifically, if the stock climbs on the back of positive results from the phase 1/2 BRILLIANCE study.

Risks To Business

The biggest risk first and foremost would be the ongoing patent dispute battle that is ongoing. The U.S. Patent and Trademark Office ruled in favor of the Broad Institute of MIT and Harvard for the patents surrounding CRISPR gene editing technology. Editas has licensed rights from Broad to use such patents, therefore I believe it’s better off compared to some other Crispr biotechs. Both Charpentier and Nobel Prize laureate Jennifer Doudna Ph.D. believe they were the first to prove this method and should take ownership of the patents. Unfortunately, the PTAB ruled in favor for Broad. There will likely be an appeal by CVC to the Federal Appeals court if it’s ultimately decided to go that route. Regardless, CVC state that it owns 40 other patents that were not a part of this ruling. This specific ruling only covered 13 patents which will be disputed. This is a major risk to be aware of because it’s hard to say ultimately how the legal battle will end with respect to specific patents. As such, the pipeline and or programs could be affected. In the meantime, it’s a huge win for Editas which gives a good scope of using both technologies of Crispr, which are Crispr/Cas9 and Crispr/Cas12a. Both of these Crispr types give the company the ability to go after a lot more indications than either one alone. A second risk would be the data readout from the phase 1/2 BRILLIANCE study with EDIT-101 in patients with LCA10 expected in the 2nd half of 2022. While preliminary safety and efficacy data was pretty good, it remains to be seen how well the next set of data will turn out. Initial data touched up on safety and some efficacy markers like BCVA, FST, Visual function Navigation and other visual improvements. The main thing to see is if these measures end up being statistically significant.

Conclusion

The final conclusion is that Editas Medicine is a great speculative biotech play to keep an eye on. While it’s still risky, it does have various options it can go after. For instance, it can make use of either Crispr/Cas9 or Crispr/Cas12a to build its pipeline from. Even then, it doesn’t only have EDIT-101 for treating patients with LCA. It has EDIT-301 in development for transfusion dependent beta thalassemia and sickle-cell disease – SCD. EDIT-301 is part of the ex-vivo CRISPR program, whereas EDIT-101 deals with the in vivo CRISPR gene editing technique. Lastly, it’s looking into advance cell therapies in the oncology arena. How will it accomplish this? It unveiled a new gene editing technology it has named “SLEEK,” which stands for SeLection by Essential- gene Exon Knock-in. This is important because it improves upon cell therapies in a few ways. For instance, it improves upon knock-in of transgene cargos. In other words, ability to far more efficiently artificially introduce a gene into the genome of another organism. A second improvement would be high heterogeneity (meaning genetic variation) which is not safe or efficient. The SLEEK technology looks to improve upon these weaknesses of current cell therapies. As such, it’s already advancing a product using this technology known as EDIT-202. It’s the company’s first iPSC (induced pluripotent stem cells) -derived Natural Killer (NK) cell oncology program in preclinical testing. With two Crispr technologies, plus its iPSC NK cell oncology program with SLEEK, I believe it has multiple shots on goal for its pipeline.