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CRISPR Basket Sale
Sold my basket of CRISPR stocks ($BEAM, $CRBU, $CRSP, $NTLA) and investing all proceeds into $GNOM. With a newborn on hand now, time is more valuable and trying to keep up with the gene-editing stocks isn't worth the time since they're far from having any treatments on the market. Would rather let an ETF run.
Thanks for sharing. Have to maximize your time when you can! Glad you were able to realize these decisions needed to be made to keep the balance in your life!
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On Bill Gate's 5 Favorite Books of the Year: The Code Breaker, Walter Isaacson's Book on Jennifer Doudna and CRISPR
If you're into Crispr stocks, you might be wondering why $CRSP has done poorly while $NTLA has done great.

One thought would be because of Jennifer Doudna's involvement. Doudna was invited to join Crispr Therapeutics in 2014 but she declined. Instead she founded Intellia Therapeutics.

If you're into this space, The Code Breaker is a must read.
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Rob T's avatar
$114.7m follower assets
What mRNA is good for and what it maybe isn't
Over on Science, Derek Lowe's latest is a must read: link

My notes:

Big players like Moderna and BioNTech (Pfizer partner) dominate, while CureVac failed on now Sanofi wants to get in on the action (vaccine failed and was only 47% effective)

Moderna was originally going to be a therapy company (pump people up with mRNA to treat them) and not as a vaccine company. Vaccines are great as you leverage the immune system to have a memory and fight off viruses after the injection. Ongoing therapeutics are tough - how often do you keep injecting people with mRNA? You're using mRNA to make proteins - which the immune system may try to fight! And what if the reaction is severe - in a quest to solve one ailment you cause another!

Targeting is difficult - liquid nanoparticles that deliver mRNA "pile up in the liver" - so low hanging fruit is to work on the retina (things stay there and don't go to the liver) or target liver diseases.

CRISPR dominates in treating certain genetic diseases - because using mRNA to target people whose genes make bad proteins in order to deliver good proteins means you still have the bad proteins lying around, but if you CRISPR, you fix the problem at the source - and the body no longer makes bad proteins and only good ones. CRISPR for the win!

What else could mRNA fix? Alzheimer's forget it - we don't know what causes it.

What about monocolonal antibodies?

Apparently that's being looked at:

CAR-T chimeric antigen receptor T-cell therapy is promising.

As you can tell from the name, that’s not an antibody-driven approach at all, but rather uses T cells on the attack, and instead of a vaccination to rouse them (which hasn’t really worked), it’s a much more laborious procedure that engineers a patient’s own T cells to deliberately recognize some particular surface antigen on the targeted cancer cells. It works for some kinds of leukemia (and not always then), and the successes are due partly to leukemia cells being individually accessible out in the bloodstream, and very much because some key antigens have been identified that lets these cells be specifically attacked. Even then, your leukemia cure likely comes at the cost of permanently impairing the population of the leucocytes involved. That’s a real concern, but if you’re to the point of doing CAR-T your alternative is basically death within months, so it’s a worthwhile trade. Naturally, people are working on other specific ways to mobilize T cells as well.

Check out the 1 yr chart of MRNA

Disclosure: No position in $MRNA or $PFE, but own $BEAM, $NTLA and $CRSP.
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CRISPR is cool tech, but doesn’t seem to have a major advantage over other approaches in the indications they’ve selected. Feel like you’re paying a huge premium for the hype and optionality behind the tech.

Just going through the pipeline - SCD and Beta Thalessemia is a good target imo, but they’re sharing more than half the economics w/ Vertex. Vertex has commercialization rights. So basically CRSP will develop the drug. vertex funds a lot of the R&D and pays CRSP for milestones. Then if the drug sells, CRSP gets royalty payments on the sales

Their CAR-T assets also aren’t super impressive either imo.

  1. There’s like a dozen different anti CD-19 or anti BCMA CAR-T in the clinic.

  1. Don’t see any major advantage w/ using CRSPR to transduce CAR gene vs lenti. It’s all ex vivo.

  1. For CAR-T to be these days, you need more than just an antigen binding CAR. Gotta have some kind of other advantage. Think AUTL (no grade 3 CRS), TCRR (keeps entire TCR complex), ALLO (allogeneic).

Rest of in vivo gene tx pipeline has a lot of competition imo. With DMD, you have SRPT, PFE, and SLDB with full blown gene therapy. GBIO will likely enter the space too. All of them are ahead of CRSP.

You also have DYN and RNA w/ ASO/RNAi drugs for DMD and DM1. For Cystic Fibrosis, VRTX already owns 90% of the market w/ an oral drug.

Only pts left are those w/ non 508del mutations, and you have KRYS and FDMT going after them.

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@robtsai is a must-follow for anyone interested in biotech & stocks with asymmetric payoffs
In just the past few days I've learned quite a bit about:

This is a space I've wanted to get smarter on for a while, but most resources out there have been pretty inaccessible. Thanks @robtsai for making these complicated topics comprehensible!
Hey this is really cool! Is there any chance that there is a transcript for the video though? No worries if not!
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