I have only been in Intellia for a very short time. I think the new CEO, John Leonard, is doing a fantastic job. They have really expanding their pipeline and jumped into developing their own base editing program.
2/ They seem to be very ambitious now toward advancing the pipeline. They are staying on the innovation side of the science and not falling behind.
3/ Science
They are one of the original CAS9 companies using CRISPR technology to do in-vivo editing in the liver and ex-vivo for cell therapies. They are focused on doing gene knockouts where mutations have little or no effects.
4/ This is a far safer place to start over insertions which are the most difficult. Their cell therapies for CAR-T and TCR are pretty much in line with all the other CRISPR companies. They are in the mix, but none of them are really leading the science.
5/ They are all easy, highly validated targets using the most basic cell edits. Their recent entry into trying to develop their own form of base editing with the purchase of Rewrite gives them the advantage over all the other first generation CRISPR companies.
6/ Potential
There is a lot of potential for them in the liver diseases which is where they are focused for now as they work on delivery to other tissues. The ATTR indication is about 50,000 patient depending on how much market share they can get.
7/ It could be worth $2 billion or more. Half of that would go to their partner Regeneron. The HAE indication is really small. That will be their second program. The TCR programs for AML and Lymphoma don't seem to be big winners.
8/ They are smaller indications that will be restricted by HLA type for AML and very competitive for CD30. I do think this company can continue to develop and grow over time.
9/ It has a lot of long term potential for all the indications assuming they can continue to move into new delivery for different organs beyond the liver. The liver has become an extremely competitive space along with cell therapies. Every company on earth is in these 2 spaces.
10/ Valuation
I think $5 billion dollars for a company with just phase 1 data is still massively over valued. Most companies in at this stage are only $3 billion or less.
11/ They have just over $1 billion in cash and burned about $270 million in 2021. That gives them well over 2 years of cash as they move more programs into the clinic.
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Sharing some strategy while the sector is still down.
1/ I pick science themes like the use of #AI or #ML in drug discovery. Then I go out and find every company I can in that space. I make myself a list and start doing my research. I slowly whittle down the crowd to 5 names that stand out.
2/ Then I go further into the companies with listening to webcasts so I get to know if the management knows their stuff. I dig into the science and data. I even read the 10K or S-1 and understand the balance sheet. Then I look at the indications and the potential.
I have only been in Century Therapeutics for a short time. I got a chance to listen to Lalo Flores present a few times. He does not come across as a great speaker.
2/ I don't think that is a deal breaker, but it does make me more cautious. It will take time to know how well he is doing with the business and clinical development.
$IPSC is building alloevasion NK an gamma/delta T cells. They will be moving their NK for CD19 into clinic this year. This will be one of the first alloevasion NK cells in the clinic with $SANA. They knock out the HLA-I, HLA-II and insert the HLA-E to block NK cells.
1/ This will give us the first look at the CD47 vs HLA-E in preventing NK cells and/or Macrophage from cell killing.
2/ Their CNTY-101 is their CAR-NK for CD19. This has the alloevasion and will be in the clinic later this year. The one this its missing is the enhanced CD16 so it can work powerfully with antibodies. That is the one disadvantage I think they have compared to $FATE.
1/ I prefix this as sequencing companies is not in my space of expertise so my numbers or expectations may be off. I do like the company as my play on sequencing in the Genomics space.
2/ Management
I have been in Pacific Biosciences for only a short time, but I think Christian Henry is doing good so far. It will take a while to really know how good he really is at running this company.
I have been in Sana since not long after the IPO. I think Steve Harr has done an outstanding job so far developing the science and business. He picked up a key license from Beam for CRISPR CAS12b for editing of the iPSC cells.
2/ Science
They have 2 very incredible platforms that complement each other with delivery using Fusogen and Cell engineering using iPSC and CRISPR technology.
I have only been in Invitae for a few months, but I think Sean George is doing a great job. He has spent a lot of money over the past few years to build up a world class company. It is very important to build a company that is competitive.
2/ They will focus on profitability going forward. They said they will continue 40% + growth for many years while focusing on margins and cash burn. They will only do acquisitions if they add strong value.