calling all doctors! EDIT, CRSP, NTLA?

I know there are some smart bio/dr types out there, given all the Nektar research lately.

Curious your thoughts on these 3 that I constantly see heralded as leaders in the nascent CRISPR gene-editing space?

They are all fairly small, about $1.5b-2.5b, but in the research phase vs making money phase right now. So I am having trouble valuing them and the upside. My thought was to do a basket of all 3 - but that assumes the TAM is real, the promise is real, and that I just don’t know which will be the victor. Or - is there room for multiple winners here? Unclear the patent fighting, if any, that exists in this space.

CRISPR Therapeutics is a leading gene-editing company focused on the development of transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. Our multi-disciplinary team of world-class researchers and drug developers is working to translate this technology into breakthrough human therapeutics in a number of serious diseases. Our lead program, CTX001, aims to treat Sickle cell disease and ß-thalassemia using an ex vivo approach. We are also pursuing oncology indications with our allogeneic CAR-T platform and liver, lung, and neuromuscular indications using in vivo approaches. In addition, we have established strategic collaborations with Bayer AG and Vertex Pharmaceuticals to develop CRISPR-based therapeutics in other diseases with high unmet need. We have licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from our scientific founder, Dr. Emmanuelle Charpentier, who co-invented the application of CRISPR/Cas9 for gene editing. We are headquartered in Zug, Switzerland with R&D operations in Cambridge, Massachusetts, USA and select business operations in London, United Kingdom.

At Editas Medicine, we believe we have entered a new era in genomic medicine as the growth of genomic information in recent years has significantly expanded our understanding of genetically defined diseases. Furthermore, a new technology known as CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) has the potential to achieve accurate, directed changes in DNA and fulfill the promise that started with the sequencing of the human genome – the potential to treat diseases at their source, at the DNA level.

At Intellia, we push the boundaries. We are focused on positively disrupting health care treatment of patients with genomic diseases. Conventional medicines treat the symptoms instead of the genetic cause of severe and life-threatening diseases, meaning that patients must take these therapies throughout their lives.

Intellia is revolutionizing medicine by harnessing the power of genome editing to develop potential cures. We bring new hope for people living with conditions including cancer, genetic disorders, viral infections, inflammatory disorders and many more.

As one, we are:

Unleashing the power of genome editing to transform medicine by correcting the underlying genetic cause of disease
Developing genome editing solutions for personalized and curative treatments
Broadening the potential application with a simplified manufacturing process
Harnessing DNA’s natural pathways to safely and effectively treat, and even cure, patients
Intellia is at the center of a scientific revolution – in a unique position to use its unmatched, powerful and precise technology to correct the genetic causes of disease.

Intellia can unlock the promise of genome editing using CRISPR/Cas9 to create a new class of therapeutic products.


Any thoughts from our smart medical Fools?
Can you point me in any good directions for newsletters/blogs that objective look at CRISPR and all 3 of these companies?
Any previous posts of note that helped summarize the pros/cons of each of the 3 stocks in this space?

Appreciate any feedback. Happy to research more on my own, but didn’t want to reinvent the wheel if this has been drilled down extensively in the recent past. Thanks!


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There has been some discussion of these companies in the past on Saul’s board,on NPI and by TMF writers.

Here are a couple of links. I can search more later when have more time.……


I see your TMF article expressing doom on CRISPR and raise you this TMF article saying that study turned out to be flawed and was retracted. :slight_smile:…

After further review…
As it turned out, the scientists who wrote the article were wrong, and the scientists at the biotechs focusing on CRISPR were right. The original team that wrote the Nature Methods article posted a corrigendum (an error correction) last week on bioRxiv (pronounced “bioarchive”), a website that provides access to preprint versions of biology papers. The original online version of the initial article on Nature Methods was also subsequently retracted.

It was basically like the NFL’s review of a play. And after further review, the original play called on the field was reversed. The scientists who initially claimed that hundreds of unintended mutations could occur with CRISPR now stated that “CRISPR-Cas9 editing can precisely edit the genome at the organismal level and may not introduce numerous, unintended, off-target mutations.” Oops.


I actually have no opinion on these companies the TMF analyst, or CRISPR in general, I was just trying to provide you with some useful links for your review. Glad you found the more recent article.


all good David…just wanted to reply with the rebuttal link for those coming across CRISPR for the first time, so it didn’t appear already dead in the water.

There hasn’t been much interest, so I will leave this topic alone unless others bring it up. Will research it more and if I find something compelling/new, will bring it forward.

In the meantime, here is what I shared on NPI board:

Since Dec, when Tinker & Duma first briefly looked at these 3:
CRSP looks like it just about tripled.
EDIT up maybe 50%, NTLA appears flat.

Heard a very interesting podcast over the Winter with CRISP expert:

MIT article in Dec:…

Science mag in Dec:…

Since then - more recent stuff

March - making mosquitos malaria-resistant:…

CRISPR can cut RNA too (not just DNA):…
The ability to target RNA instead of DNA expands how Cas9 scissors can be used. Potential uses range from controlling which genes are turned off or on to combatting human viruses that are made of RNA to rapidly detecting infectious agents.

CRISPR-edited crops:…
In a statement released on March 28 the USDA stated that they will not regulate plants that have been modified through genome editing as long as the same genotype could have been produced through traditional breeding methods. This announcement has the potential to shave tens of millions of dollars off the cost of crop develop.

apparently a study was done that sent CRISPR stocks plummeting. And then it was retracted.
A sign of FUD hitting the market could possibly be (ironically) an indicator that there is something real about the TAM of the market.…
It was an overreaction, as so many market spikes and dips turn out to be; the company CEOs were quick to push back, and scientists and journalists soon pointed out flaws in the paper that misconstrued cause and effect. After nearly a year of continued criticisms, and failures to reproduce the results, the authors conceded that the skeptics might be right. Last week, the journal that published the paper, Nature Methods, finally retracted it.

Some ex SpaceX employees starting up their own biotech to leverage CRISPR:…

and it looks like human trials starting:…
“Soylent green is people…it’s people!” - nothing to do with this article, just love that movie line.


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I briefly looked into it. It seemed way early in the space for me to figure out how it’s going to play out. My biggest issue is the immediate disruption that would be caused by a superior alternative. No matter how good Crisper looks, it’s still way short of what is needed. Fuma would know more about it as I think he’s looked into it more heavily, I think, but he seemed pretty critical of the tech from what I recall.

From what I read, targeting RNA for editing was the real breakthrough but still the “editing” thus far has left a lot to be desired. Most of the “successes” are just deleting a faulty gene but not replacing it. Kind of like declaring victory at halftime IMO.

When I did my initial research I came upon this article back in November:… so I bought NKTR instead.



A serious issue for CRISPR -

Shares of companies developing CRISPR-based therapies slid Monday after STAT News reported two new studies showed edited cells might cause cancer.

Gene editing tool CRISPR-Cas9 has been hailed as a breakthrough that could allow scientists to treat and possibly even cure genetic diseases. In two studies published Monday in the journal Nature Medicine, researchers found editing cells’ genomes with the technique might increase the risk of cancer. Those edited cells are those ones that are intended to treat diseases. :…