Clustered Regularly Interspaced Short Palindromic Repeats

[Gregmal]

AKA CRISPR

 

I haven't seen anything here on this so I thought I'd do everyone the favor of floating what is described by scientists as a "once in a generation breakthrough". Yes I know this idea is not for everyone, but thats OK, it can only be for those that want to make money or have the ability to see where the puck is going...If you require a single digit PE and low P/B in order to make an investment you can stop reading now. If you are open minded and wish to continue, I introduce you to the breakthrough technology...

 

That is CRISPR and the variants Cas9/12/cpf1/3.

 

What CRISPR does is effectively target specific genes within an organism by activating proteins naturally occurring within the immune system and directing via RNA an attack on a specified areas. In layman's terms think of going to the dermatologist and having a mole removed. CRISPR more or less removes a malignant mole from your genome. It is that simple and this is why it is going to change modern medicine. Up until now, the majority of treatments for any sort of ailment involved putting together a concoction of, lets call them, "things" and then seeing how they reacted with the body. CRISPR entirely removes this aspect of risk. It is as simple taking a taking a scissor to an undesired or harmful trait.

 

A good piece is found here, https://www.technologyreview.com/s/604126/edible-crispr-could-replace-antibiotics/

 

So far, CRISPR has been effective on every complex organism it has been attempted on. It is able to target just about any disease, and on a scientific level, is so easy to apply high school students are messing around with it in AP classes.

 

CRISPR itself is not really that controversial, however the aspect that is controversial stems from the optionality. When programming the RNA so to speak, you have two options. One is to cut out the genome, and the other is to cut out and replace. Cutting out and replacing is where you can take an undesired aspect and flip it into a desirable one.

 

There is a fortune that will be made from this and there are a bunch of different avenues where one can look to invest. Rather than follow specific companies I have found it helpful to follow specific doctors whom have developed and patented this "technology". These names are Jennifer Doudna, Feng Zhang, Emmanuelle Charpentier and George Church.

 

The various fundraising rounds have been loaded with the biggest names out there. Bill Gates specifically has described this as "one of the most powerful technologies of the 21st century".

 

There are a handful of public companies with holds on the patents, as well as a good few private companies with shares occasionally available which offer an opportunity to play this space. The names I think are worth following are Caribou Biosciences, Editas Medicine, Crispr Therapeutics, Synthego, and Beam Therapeutics.

 

I have found Nature.com, Pubmed, and Researchgate to be sites with ample resources on this and database access to current events. Curious if others have studied this, and/or made investments or have ways to play this.

 

Be forewarned that yes, these companies will not likely to make money for years and that yes, this whole theme is not even in the first inning yet; it's just batting practice, maybe pregame warm ups. But the opportunity is so massive and the patents even today IMO are severely undervalued mainly because people fail to fully understand or appreciate the potential and have all sorts of misnomers floating around because of stigmas attached to boogeyman words and phrases like "GMO" and "playing god".

[Cigarbutt]

You seem to be mixing RNA for DNA but let's not get wrapped up in unimportant details. Right?

The topic was touched in reply#25 in the HTL thread.

The race is on.

https://www.vox.com/science-and-health/2018/11/30/18119589/crispr-gene-editing-he-jiankui

[Gregmal]

Yes we touched on it in that thread. I had familiarity with it then and further began looking into this over the following months and after doing some work, and hearing people in the field rave about this, began looking for ways to express this optimism through an investment. I have yet to find a specific one.

 

And FWIW CRISPR can target both RNA and DNA. Its primary delivery mechanism however is a guide RNA with an endonuclease that then splices the DNA. The mole removal analogy to me is easier than doing a biology walkthrough just to make this coherent to the average investor.

 

https://phys.org/news/2018-02-crispr-cas9-tool-rna-dna-precisely.html

 

The idiot Jiankul not only committed a highly unethical violation, but potentially set things back in terms of public acceptance. One of the big things Jennifer Doudna(more or less the one who discovered this) has been a major advocate of responsible introduction of CRISPR to the public. This way, it is understood rather than feared like GMO's.

[Parsad]

Broad subjects like this belong in the "General Discussion" section, where I will move it to.  It should not clutter the "Investment Ideas" board, where individual stocks are categorized by their ticker symbol.  Cheers!

[LC]

https://www.usatoday.com/story/news/health/2020/03/04/crispr-gene-editing-tool-used-inside-patient-blindness-portland/4950458002/

 

Scientists say they have used the gene editing tool CRISPR inside someone's body for the first time, a new frontier for efforts to operate on DNA, the chemical code of life, to treat diseases.

 

A patient recently had it done at the Casey Eye Institute at Oregon Health & Science University in Portland for an inherited form of blindness, the companies that make the treatment announced Wednesday. They would not give details on the patient or when the surgery occurred.

 

It may take up to a month to see if it worked to restore vision. If the first few attempts seem safe, doctors plan to test it on 18 children and adults.

 

[Gregmal]

https://www.usatoday.com/story/news/health/2020/03/04/crispr-gene-editing-tool-used-inside-patient-blindness-portland/4950458002/

 

Scientists say they have used the gene editing tool CRISPR inside someone's body for the first time, a new frontier for efforts to operate on DNA, the chemical code of life, to treat diseases.

 

A patient recently had it done at the Casey Eye Institute at Oregon Health & Science University in Portland for an inherited form of blindness, the companies that make the treatment announced Wednesday. They would not give details on the patient or when the surgery occurred.

 

It may take up to a month to see if it worked to restore vision. If the first few attempts seem safe, doctors plan to test it on 18 children and adults.

 

The potential of CRISPR is breathtaking. And most people, dont have the slightest clue.

 

The investment angle is a little tougher, but playing the CRSP, EDIT, NTLA, BEAM trading sardine strategy will definitely be profitable. I'm still really hoping to see a Caribou IPO. To me, these are positions everybody should have a low single digit collective basket of. Upside "could" be enormous.

[Guest cherzeca]

so, I started looking at crispr maybe 5 years ago because George Church was a classmate and I was curious.  george by the way is a brilliant scientist and a good guy.  what I found was that while the science was real, the pathway to investment profit is very unsure. patent fights. the typical chasms between science lab and commercialization.  I confess I haven't relooked at it in awhile. I would encourage this thread and I would look forward to other contributors' posts.

[Gregmal]

so, I started looking at crispr maybe 5 years ago because George Church was a classmate and I was curious.  george by the way is a brilliant scientist and a good guy.  what I found was that while the science was real, the pathway to investment profit is very unsure. patent fights. the typical chasms between science lab and commercialization.  I confess I haven't relooked at it in awhile. I would encourage this thread and I would look forward to other contributors' posts.

 

Yea its definitely a bit challenging figuring out the eventual dollar flow to specific companies. The best Ive been able to do with certainly is probably come back to a company like Illumina. But thats not to say these companies won't benefit. The honest answer is its hard to tell. Its encouraging to see some of the backers of these companies, ranging from multi billion dollar pharmas like Vertex and Bayer, to Google Ventures and the Gates Foundation.

 

Ive found, scientists are very smart dudes and I dont discredit what they say(same with all the medical experts speaking about corona) but they are much more academics than they are financially savvy individuals. They get wound up in theory and exercise but often remain as clueless to the investment ramifications of things as Joe the Plumber. That said, all of the people in the field Ive ever encountered, talk about this as if they're Columbus encountering the Americas. Endless potential. So its something to be excited about and the securities will act as a mechanism for those that follow to make some money. "Trading" may make some uncomfortable, but its a good hedge to the risks we've already discussed on the subject.

[Guest cherzeca]

this is a good background read, written by church:  https://www.amazon.com/Regenesis-Synthetic-Biology-Reinvent-Ourselves/dp/0465075703

 

this space is definitely worth keeping an eye on

[Gregmal]

https://www.timesofisrael.com/with-new-elegant-chemo-israeli-scientists-edit-genome-to-destroy-cancer-dna/

 

https://advances.sciencemag.org/content/6/47/eabc9450

[Spekulatius]

I don’t have much of a clue about CRISPR technology and I am not sure one has to. I have become convinced that in most cases, platform companies are not the way to invest in Biotech. Rather invest in smart management team that are platform agnostic, but have deep knowledge in therapeutic areas and clinical development and let them pick the technology that they think works best.

 

I don’t have many positions in Biotech, but I do have a smallish position in BMRN because I have followed them for a decade or more, when I could find stories in the local newspaper. Now looking at them, they have proven that they know what they are doing and brought several products to market and are cash flow positive now. That’s more like a bet I feel making, but everyone’s view is different.

[Gregmal]

https://www.bloomberg.com/news/articles/2020-12-05/crispr-gene-editing-shows-promise-in-blood-disease-study-updates

[Gregmal]

From one of my headline feeds.....

 

Oppenheimer analysts ups CRSP price target to $155 from $105, maintains OUTPERFORM rating.

 

Current share price? $159...

 

 

LOL We should put all the analysts together in a room with all the scientists and academics and see what comes out....

[Gregmal]

https://seekingalpha.com/news/3644173-crispr-therapeutics-secures-grant-for-gene-therapies-for-hiv

[Gregmal]

https://www.usnews.com/news/top-news/articles/2021-01-19/chinese-scientists-develop-gene-therapy-which-could-delay-ageing

[Parsad]

https://www.cnn.com/2023/11/16/health/uk-casgevy-approval-crispr-gene-editing-sickle-cell-scn/index.html

 

Cheers!

[Sweet]

It’s going to be a lot harder to apply CRISPR to other types of tissue type.  With blood you can wipe out the entire bone marrow and slowly replace it.  It’s going to be difficult to do this at whole body level.  Great technology but long road ahead.

[Saluki]

The Code Breaker book about the two women who got the Nobel prize for it is great.  There are a lot of articles written about attempts by male scientists to claim credit for it as was shamefully done to Rosalind Franklin by Watson and Crick.  It's ironic that in cementing the legacy of two women in the CRISPR discovery, the Latino scientist, who discovered, named it, and figured out it's role in microbial defense, is never mentioned.  Francisco Mojica: https://en.wikipedia.org/wiki/Francisco_Mojica

 

Although if you want to channel peak wokeness, you should refer to him as a LatinX scientist . 

[james22]

I keep hoping this thread is about a new Stereolab album.

[Sweet]

1 hour ago, Saluki said:

The Code Breaker book about the two women who got the Nobel prize for it is great.  There are a lot of articles written about attempts by male scientists to claim credit for it as was shamefully done to Rosalind Franklin by Watson and Crick.  It's ironic that in cementing the legacy of two women in the CRISPR discovery, the Latino scientist, who discovered, named it, and figured out it's role in microbial defense, is never mentioned.  Francisco Mojica: https://en.wikipedia.org/wiki/Francisco_Mojica

 

Although if you want to channel peak wokeness, you should refer to him as a LatinX scientist . 

https://www.nature.com/scitable/topicpage/discovery-of-dna-structure-and-function-watson-397/

[patience_and_focus]

This is one of those things that will never be settled given tea leaf reading of intentions and lack of precise information on why something didn't happen. We should move on while acknowledging that scientific work is cut throat competition like many (all?) other fields and jostling for the ultimate prize.

 

https://sciencehistory.org/education/scientific-biographies/james-watson-francis-crick-maurice-wilkins-and-rosalind-franklin/

 

"

At King’s College London, Rosalind Franklin obtained images of DNA using X-ray crystallography, an idea first broached by Maurice Wilkins. Franklin’s images allowed James Watson and Francis Crick to create their famous two-strand, or double-helix, model.

 

In 1962 Watson (b. 1928), Crick (1916–2004), and Wilkins (1916–2004) jointly received the Nobel Prize in Physiology or Medicine for their 1953 determination of the structure of deoxyribonucleic acid (DNA). Wilkins’s colleague Franklin (1920–1958), who died from cancer at the age of 37, was not so honored. The reasons for her exclusion have been debated and are still unclear. There is a Nobel Prize stipulation that states “in no case may a prize amount be divided between more than three persons.” The fact she died before the prize was awarded may also have been a factor, although the stipulation against posthumous awards was not instated until 1974."