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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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The royalty revenue for Spinraza is 12.5% or revenue. The generated $113 million in 2017 off of almost $900 million in sales. That had to be close to 50% of profit. Net income margin on these drugs typically runs around 25-30%. And, that $113 million is pure profit to IONS.

The AKCA strategy seems to be make it the commercialization arm and IONS keeps R&D.

The drug complications are under control. So, why no love? That is the question. Somewhere, I am wrong.
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Sorry, my post ended in wrong place.
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and I got all excited, too...


No one cares about my gene editing empire-to-be.

Dreamer <--- recipient of no caring.
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Here was our discussion from several months ago:

Something changed since then?
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Genomics has great promise.....treatment, diagnosis, personalization of medication.....and on and on.

So far, virtually none of it has been shown to be of benefit in humans....yet.

Actually, China may be at the forefront of this; but that's because of lack of oversight. Actually changing a persons genetic make-up is fraught with ethical and scientific challenges. The Chinese are already trying some therapies on humans.

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Something changed since then?


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's people!" - nothing to do with this article, just love that movie line.

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CRSP and NTLA seem like all hype. EDIT is slightly better I guess.

You may have some highs along the way, but if you told me these were all penny stocks I 5-10 years, I wouldn't be surprised. You can also see how the business model pans out with Spark therapeutics for these one and done treatments to see if sustainable log term.

FMI might be worth a flyer as a picks and shovels play.

My own unrequested opinion- only a very small percentage of your portfolio, only what you're willing to lose completely. You can always add more if/when any of them advance into trials/have positive trials.
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I owned all of them at one time as well as SGMO and BOLD, bought back around Feb 2017. I eventually sold out a few months ago after they all ran up. I am watching now, I actually wish they would just start raising funds at this high valuation as they have at least another 4 years to go before they are likely to get something close to market. Given that, I think there will be plenty of time to get in after 2020 though I may buy back into SGMO.
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