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CRISPR Therapeutics: A Review Of Its Clinical Pipeline And Progress

Tech Junkie profile picture
Tech Junkie


  • CRISPR Therapeutics is a gene-editing company focused on using CRISPR/Cas 9 in therapeutics areas such as hemoglobinopathies, immuno-oncology, regenerative medicine and in vivo applications.
  • In November 2019, the company reported positive interim data from the 2 patients treated in its lead program.
  • Despite the promise, I will be holding on starting a position in the company although I will be monitoring its progress closely.


CRISPR Therapeutics is a gene-editing company focused on the development of CRISPR (Clustered Regulatory Interspaced Short Palindromic Repeats)/CAS9(CRISPR-associated protein 9)-based therapeutics. The company is focused on translating revolutionary CRISPR/Cas9 technology into transformative therapies in therapeutics areas such as hemoglobinopathies, immuno-oncology, regenerative medicine and in vivo applications.

The Need for Gene Editing

Aberrant DNA sequences cause thousands of diseases that have not been treated by traditional small molecule and biologics as such treatments do not address the underlying genetics causes. Gene editing has the potential to provide curative therapies to many genetic diseases by precisely altering DNA sequences within the genomes of cells, which is done with the aid of enzymes cutting the DNA at specific locations. After a cut is made, natural cellular processes repair the DNA to either silence or correct undesirable sequences, potentially reversing their negative effects. As the genome itself is modified in this process, the change is permanent in the patient.

Gene editing also has other applications beyond treating genetically-defined diseases. It can also be applied to the engineering of genomes of cell therapies to make them more efficacious and safer. Cell therapies have been making a meaningful impact in certain therapeutics areas, such as oncology. An example of that is the approval of the CAR-Ts by Novartis (NVS) and Gilead (GILD).

The CRISPR/Cas9 Technology

As its name suggests, the company is utilizing CRISPR/Cas9 as its method of gene editing. Their technology is based on the work of their co-founder, Dr. Emmanuelle Charpentier, who is acknowledged as one of the key inventors of CRISPR-Cas9, and her collaborators.

Figure 1 Applications of CRISPR/Cas9 (Source)

The CRISPR/Cas9 technology is a versatile technology that can be used to disrupt, delete, correct or inset genes. It is used to make cuts in DNA at specific sites of targeted genes, and once the DNA

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Tech Junkie profile picture
5+ years of commercial experience in the biotech industry, with strong knowledge in biopharmaceuticals and cell/gene therapies. MBA educated with a B.Sc in Bioengineering.Invest in innovative technology and biotech companies. Covers both Tech and Biotech companies on Seeking Alpha.

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Comments (31)

From a pure numbers perspective this is over-priced. You have RNAi companies actually generating profit with comercialised products and they are valued at anywhere between 4-12B. CRISPR has no commercial product, sorry this is a nuts valuation, and when you consider that the RNAi cos have deep pipelines in PII, and PIII with big pharma partners. CRISPR will need to focus on quickly bringing best in class results for diseases with unmet needs and have minimal or no side effects. The risk is totally on the downside, only perfect execution with perfect clinical data will support this valuation. However I like the technology and the potential it has, waiting on the sidelines at the moment for more data.
Dan Strack profile picture
That’s how biotech stocks work. ALNY had a higher stock price 4 years ago, well before marketable products or P3 results.
Jean-Jacques Rousseau profile picture
There are very few articles being written about CRSP and most focus on short-term prognostications/ and technical predictions.

There are also some opinion pieces, like this one, that expand on the company pipeline slide and mention some not that relevant other names (ATRA...?).

No analysis of the true market opportunity in TDT/SCD, B-cell cancers, or MM.

There should be >10 patients that have been dosed by now in the lead CTX001 program, the next interim analysis and data presentation should come up soon and include preliminary data from newly infused patients as well as provide further support for best in class status if the TDT and the SCD pts remain free of transfusions and VOD respectively.

CRSP is the clear leader in CRSPR (clustered regularly interspaced short palindromic repeats) editing in contrast to EDIT and NTLA that have struggled to move into clinic. They all have a bright future when the technology is validated in the form of the first FDA approval.

The fact that there is so little coverage of CRSP (e.g. contrast that to TSLA) tells me that the vast majority of the investing public/institutions/speculators/traders have yet to awaken to this transformative revolution that is slowly boiling under our noses.

People who think CRSP is expensive at $3B will love it at >$30B.

PS I look forward to the author revisiting this company after the next interim analysis snapshot.
Jean-Jacques Rousseau profile picture
Institution and retail who don't see this revolution will miss out on incredible wealth creation.

Those who underestimate this opportunity might want to remember Alec Baldwin from the Glengarry Glen Ross film:

“Oh yeah, I used to be a salesman... it's a tough racket.”
Andrew86 profile picture
@Jean-Jacques Rousseau

TY for your post. Can you point to any research that will he;p others better understand the opportunity?

I gather you believe this company has a lock on this technology with patents?
Tech Junkie profile picture
IF you actually read the whole article, you'd actually find that most of your comments were addressed.

"that expand on the company pipeline slide and mention some not that relevant other names (ATRA...?)" ATRA is relevant in that CRSP is working on allogeneic CAR T and ATRA has a lead program, tab-cel for PTLD, that is on track to be filed for BLA in the second half of this year. While tab-cel is not a CAR-T, it is an allogeneic T cell program and likely would be the first allogeneic T cell immunotherapy to be approved and ATRA is also pivoting towards allogeneic CAR-T.

"CRSP is the clear leader in CRISPR (clustered regularly interspaced short palindromic repeats) editing in contrast to EDIT and NTLA that have struggled to move into the clinic. They all have a bright future when the technology is validated in the form of the first FDA approval." It was mentioned that CRSP was ahead of both companies.

However, Bluebird is ahead of CRSP in both TDT and SCD and Bluebird have gotten more patient dosed and showed more durability in their results to date, as compared to CRSP who has only given interim data for 2 patients. Bluebird already has product approved in TDT in Europe and further along in SCD, as well as close to having their autologous CAR-T approved for MM as well as Lenti-D for CALD.
Yet, they have had recent struggles with the pricing of the products and the complexity in manufacturing delaying approvals. What we can learn from Bluebird is that having a product approved is just the first step. Despite having dosed much more patients and having a more advanced pipeline, they are just "trading at 4b market cap". While I'm not saying that they can't be one day be trading at 30B market cap, it is very optimistic to be so sure of their technology given that they won't be the first in class in their therapeutic treatment and even if they get approvals, they'd have to do exceptional commercially and what've we seen from Bluebird and the autologous CAR-Ts are that there have definitely been struggles in this aspects.
Isn’t the women who had sickle cell a little bit more than just ‘interim data’?
Wasn’t she basically cured?
That would put CRSP light years ahead of any competitors, who are still just dosing lab rats , yes?
Jean-Jacques Rousseau profile picture
Tech Junkie profile picture
It's interim data in the sense that the follow-up time is still short, and they need further confirmation that the response is lasting and durable.

They are the furthest along among the companies using CRISPR, but Bluebird has more advanced programs in both TDT and SCD for gene editing. For CAR-Ts, there are several other players in both autologous and allogeneic.

In other words, they are definitely not the first-in-class and would need to be best-in-class.
Thank you for the article. I took the opposite view and bought a few shares of CRSP recently. For me there is a "Vertex Put" that will hold up the value. Also, once the technology proves successful in a few applications, development for future applications will accelerate and those with years of prior development (such as CRSP) will be at a major advantage.

Of course, as your article states, the technology is not yet proven. The combined market cap of all CRISPR/CAS-9 companies is extremely low considering the potential of being a replacement to many forms of drug development. Either their market will go to zero or it will take over a good portion of the existing pharmaceutical space. The current market cap almost hints at the former being more likely. For sure, investors with lower risk tolerances and investors who understand the need to preserve capital should stay away. Investors with higher risk tolerances and those who, like myself, are fools, are beginning to buy.
Tech Junkie profile picture
There were some rumors earlier on that Vertex may acquire CRSP but that seem to go away as recently Vertex actually reduced their positions in CRSP.
Andrew86 profile picture
@Tech Junkie @Dan Strack

Thanks for article. I'm new at looking at this stock so need some help.

I came across it due to Ark owning the stock. She seems to think it is a platform technology. So rather then looking at pipline alone, she believes that over the next few years the pipline could grow a lot.

Does the writer believe that at all? If gene editing is the future does this stock have the potential to get a substantial cut from those that use their technology? Assuming patents hold.

If Ark are right, wouldn't 3 Billion be cheap? Assuming they are one of the few companies that get a cut.

Dan Strack profile picture
By platform technology, I believe it relates to clinical cures rather than treating symptoms as has been done by the medical industry throughout history. Their platforms can grow organically and through partnerships.

I don’t consider the potential patent payments at all when looking at the stock. Unrelated market dips are a great time to buy.
Tech Junkie profile picture
Not sure if I get your point but CRISPR can be used in a variety of applications. In the case of this company, they are applying it in rare blood disorders (TDT/SCD), immunology-oncology (Allogenic CAR-T), in vivo and regenerative medicine. In the first 2 areas, CRSP is not the most advanced companies and they need to prove that their treatments are superior. In the later 2 areas, they are still only in preclinical development.

The company could technically license CRISPR CAS-9 out, although the origins of this technology are from an academic center and not sure how strong those patents are in the first place (see my other comment to your post). Also, there are 3 other companies using CRISPR and while they are less advanced in clinical development, they could technically also license out their platform.

Beyond that, there are other gene-editing techniques such as TALEN by Cellectis, ARCUS by Precision Bioscience and Zinc Finger by Sangamo Therapeutics. All of these companies have different approaches/partnerships with big pharma and different therapeutic focus.

So, to answer your question: Yes CRISPR CAS9 could be regarded as a platform for gene-editing and be used in several applications but CRISPR therapeutics is not the only company using this technique. There are also other companies using different gene-editing techniques.
Andrew86 profile picture
@Tech Junkie @Dan Strack @Jean-Jacques Rousseau @King Rat

Thanks for your answer.

I could be wrong but isn't gene editing going to be huge? Huge as in worth trillions to the winner/winners? Isn't all other medical research backward in comparison?

As investors shouldn't we be trying to work out who the winners will be?

Ark believe it will be three companies who control all the patents:

Wood argued that the public markets are vastly undervaluing the three companies that control the foundational patents for the most promising gene-editing technique, called CRISPR-CAS9: Intellia Therapeutics (NTLA), Crispr Therapeutics (CRSP), and Editas Medicine (EDIT)

Be interested in the views of others.
Agree. $ 3B market cap is not justified for the stage of pipeline development even it is a sexy technology
agree as well.
even if they succeed in getting a product, that indication is so crowded, they'll never make enough money to justify the current valuation
Dan Strack profile picture
1 or 2 products won’t justify their valuation (enterprise value is much lower at just over $2 billion), but rather proving this technology can cure diseases is the investment thesis. If CRSP is successful in sickle cell or beta thal., the current valuation is just a drop in the potential market cap bucket.
you would be surprised how few indications they can use their current platform for... either the technology evolves to be able to address more diseases or they slowly disappear...
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