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May 13 • 5 tweets • 1 min read
I think $RXRX is the most advanced company working in the Tech in Biotech space. They are using #robots to run millions of experiments per week to feed all the data into their super computer.
They then use that data to find Known Chemical Entities (KCE) and New Chemical Entities (NCE). Their first 3 drugs are all going into phase 2 this year. They are KCE drugs that were in-licensed for CCM, NF-2 and FAP.
They also have a C-dif drug working on its IND. They have 3 really interesting NCE drugs in preclinic for CDK12, SKT11/KRAS and Myc. All of them have good promise.
They have many partnerships with over $13 billion in potential milestones. Assuming they have good phase 2 data, this company could quickly be worth 10x to 20x this value in just a few years.
They have over $590 with less than a $1 billion market cap.

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More from @Biotech2k1

Biotech2k Profile picture
May 14
I am going to mention a few more #CRISPR companies that didn't meet my expectations to be in my portfolio. My next pick would actually be $VERV. I have some reservations on how much market share they could really capture in an extremely competitive space of Cholesterol.
$VERV has impressed me on the management front. There is noting I would bet on more then top notch management. A great CEO will make magic out of nothing. I think $VERV with base editing and a great CEO will be successful, I just don't know how successful.
Then comes $CRSP which use to be my first and only #CRISPR company. I completely changes on this company. They have a great SCD program what will be successful, but it ends there.
Read 5 tweets
Biotech2k Profile picture
May 14
My 3rd #CRISPR company has to be $CRBU, even though, I count them in my cell therapies space. They are not focused on any in-vivo editing at this time. They are completely focused on cell therapies around CAR-T and NK cells.
They are using the CAS9 and CAS12 enzymes along with their own unique guide called chRDNA. This uses a combination of RNA and DNA in the guide to increase binding affinity and better editing.
Their early data this week for CAR-T was impressive. It is still very early and clearly PD-1 will not be the "be all to end all". There are a lot of challenges to allogeneic CAR-T that need to be solved.
Read 6 tweets
Biotech2k Profile picture
May 14
My second pick for #CRISPR is $NTLA. I know they are on the losing side of the patent dispute. We still don't know what if any impact that will have. They do have a great management team that is building a great company.
They are using first generation CAS9 editing in-vivo in the liver and for ex-vivo for cell therapies. They are also developing their own base editing program. They are still ambitious and chasing innovation. That is what gets my vote.
They have best in class data in their early ATTR program in phase 1. Its got a long way to go, but could be a huge success if this data continues. They have about $1 billion in cash to get them through this bear market in biotech.
Read 4 tweets
Biotech2k Profile picture
May 14
$BEAM is my top pick in the #CRISPR space. They have base editing which solves many of the issues with the first generation editors. They still have the CAS12 if they need it to do any cell editing in their cell therapies.
All their tech licenses comes from the winning side of the patent dispute which makes them safe. They have a great management team. They also have $1.2 billion in cash which will last them a long time in this bear market.
They are just entering the clinic this year so it will all come down to the data. Their preclinical data seemed superior to that of $CRSP at the same stage of development.
Read 4 tweets
Biotech2k Profile picture
May 13
1/ I looked at $RLAY with its data so far and all the technology going on. They are still my top pick in #AI/#ML in drug discovery.
2/ They use so many technologies to understand how proteins are encoded and move while they function. They use #ML to sort through DNA encoded library screens.
3/ They are taking on some of the most toxic targets in oncology and developing game changing drugs. They have 3 drugs for FGFR2, PI3Ka and SHP2. The PI3Ka space could be as high at 150,000 patients.
Read 4 tweets
Biotech2k Profile picture
May 12
1/ I have been spending a lot of time with the market selling off trying to share and teach about investing and how to build a portfolio to diversify across assets and risks.
2/ I think its good to take time when the market is down to focus on improving our game for the next time around. Since I am a biotech focused investor, I would like to share with you the trends within the biotech space I think could propel the next decade of innovation.
3/ If you know me already, these won't be very new to you. My first big concept is using tech in biotech with developing #AI, #ML and #Automation to drive down the costs and improve the success of clinical development.
Read 18 tweets

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