CRISPR Therapeutics
A cutting edge biotech company that could change an industry
If you have been following Celeritas Capital for a while you might remember a post where I introduced the world of biotech trading. If you have not read this article it is linked below. I recommend you read although it is not necessary to gain information from this article.
The Lovely World of Biotech Catalysts
In this article we plan on trying something new. Let us know if you found this helpful or informative. Introduction: A brief introduction to the biotech industry. Biotech companies start like most other companies, as a small operation with a few employees and little to no revenue for many years. This problem is only exacerbated by the fact biotech compa…
Table of Contents:
CRISPR the Science
CRSP the Company
Some financials
Forecasting Possible Sales for Exa-cel
My opinions & closing
CRISPR the Science:
I am sure most of our subscribers have their own personal views on gene editing. This is of course perfectly fine, with that being said we hope you do not disregard this entire article due to personal beliefs. Now onto the science, CRISPR-Cas9 is a type of gene editing process that uses the CAS9 enzyme to do gene editing. The process is extremely complex, but I will try and simplify it for Exa-Cel. (CRISPR Therapeutics lead treatment)
Exa-cel is a gene therapy being developed by CRISPR Therapeutics and Vertex Pharmaceuticals for the treatment of sickle cell disease (SCD) and beta-thalassemia. It works by extracting a patient's stem cells and editing them to flip the genetic switch back to making fetal hemoglobin (HbF). HbF is a form of hemoglobin that is naturally present in fetuses but is switched off after birth. To prepare for treatment with Exa-cel, patients are given a course of chemotherapy with busulfan to kill off their old stem cells. They are then given the edited stem cells, which can then produce HbF. The treatment is a long and extensive process, but it has shown very promising results in clinical trials. In patients with SCD, Exa-cel has been shown to produce high levels of HbF and eliminate vaso-occlusive crises (VOCs), which are the most serious complications of the disease. The FDA has granted Priority Review to Exa-cel for SCD with a target action date of December 8, 2023, and Standard Review for beta thalassemia with a target action date of March 30, 2024. If approved, Exa-cel could be a major breakthrough for the treatment of SCD and beta-thalassemia improving thousands of patients’ standard of living. It will also be the first CRISPR treatment to go to market if approved.
CRSP the Company:
CRISPR Therapeutics ($CRSP) is a pioneering company in the CRISPR-Cas9 domain, utilizing the original CAS9 enzyme for gene editing applications. In light of potential concerns surrounding the use of double-stranded breaks within the human genome, CRISPR Therapeutics has directed its efforts towards employing this technology for editing cells external to the human body, such as stem cells and T cells utilized in therapies. This is a very important fact CRSP 0.00%↑ does not (currently) edit any genes in living humans. The company stands at the forefront of advancements in the CRISPR field and is a leader in the gene editing field in general.
CRSP 0.00%↑’s leading candidate, CTX-001 or Exa-cel as the company has named it. Exa-cel is designed for the treatment of Sickle Cell Disease (SCD) and β-thalassemia which is a blood disorder. Exa-cel has entered the stages of commercial approval submission in both Europe and the United States. This is very exciting for investors however, it is important to remember that there are still a few steps before Exa-cel can go to market. (See my last article on biotech catalysts for more info) The company has also embarked on a comprehensive pipeline of CAR-T cell therapies utilizing CRISPR gene editing technology. With a strong leading candidate in Exa-cel and a robust pipeline of other treatments CRSP 0.00%↑ from a treatment viewpoint looks very promising. (Figure 1) Next, we will take a look at CRSP’s financial situation.
CRSP’s current assets to liabilities look healthy and should give the company a solid runway. This is a very important metric for biotech companies. I will expand more on CRSP’s financials in my model section below.
Exa-cel sales projection & CRSP Model
These are models I made and are some of the first financial models I have created. As with all financial models they should be taken with a grain of salt. I think they are okay models but I would love any feedback, as modeling biotech companies is rather difficult. Now onto the models first which is a projection for Exa-cel sales if it is approved. An important fact to remember is that Exa-cel is a collaboration with Vertex, with CRSP 0.00%↑ being entitled to 40% of the revenues created by Exa-cel.
To explain the assumptions made in the model, I predict the costs for Exa-cel to be $2.5 million dollars per dose. With an average of 594 doses a year. So after the financial year of (FY) 2030 ends my model shows Exa-cel will create $4.1 billion in revenue for CRSP 0.00%↑. (Figure 3) This is a new type of financial modeling for me, so any feedback would be greatly appreciated!
Figure 4 above, is my model for CRSP 0.00%↑ and its implied share price. My view is CRSP 0.00%↑ is undervalued by 48.2% with a possible share price of $71.60. This is my view based on the model I created using www.stratosphere.io which is a great tool for a wide array of investors, I highly recommend it.
If you are interested in learning more about www.stratosphere.io check out an amazing article by
which is linked below. (Also make sure you subscribe to if you are not already!)
My opinions & closing
If you do not follow biotech stocks or don’t read every new scientific article on gene editing this was probably a lot to take in. I hope if you take anything from this article it is biotech, specifically CRISPR is in a very exciting place.
In closing, I am bullish on CRSP 0.00%↑ and believe that their PDUFA reviews in December and March 2024. It is going to be a huge event in the biotech space. Always do your own due diligence and research before making any investment. This article is something a little bit different than my usual posts but this piece was extremely interesting to write. I think I will be publishing longer-form content for the foreseeable future. Thank you to all of my subscribers! I truly appreciate everyone who takes the time to read Celeritas Capital.
DISCLAIMER: We are not Financial Advisors, and all information presented is for educational purposes ONLY. Financial markets can be highly volatile, so good risk management is a must. The writer of this article also plans on purchasing CRISPR Therapeutics shares.
great coverage, Crispr is quite a name and to be watched for sure ... thank you!