Biotech2k Profile picture
Jun 17 20 tweets 8 min read
The most impressive science in biotech right now is:

1. #iPSC
2. #AI/#ML in biotech
3. #SynBio
4. #CRISPR
5. #TPD
#iPSC has to be doing some of the most amazing science in the entire sector right now. The funny part is they don't get all the hype as the other hot science spaces. They are completely engineering cells that have many new functions and remove many old functions.
They are making all kinds of cells from stem cells into eye, heart, liver, pancreas and immune cells. They don't get much love because of the bad data of the allogeneic CAR-T programs, but some of these companies are completely disruptive.
$IPSC is making a common progenitor cells for iNK and iT cells that have upward of 12 edits to them to remove their immunogeniticy, enhance their performance, and eliminate the toxic need for lymphodepletion.
$SANA is crating iPSC cells into heart, eye and pancreatic along with immune cells like CAR-T. They have a hypoimmune platform that protects these cells from immunogenicity. They are also working on in-vivo cell engineering with their Fusogen delivery.
$FATE is the OG of the iPSC world. They have developed many of the edits the other companies are now deploying. They are further along in the development, but they are still limited to just immune cells with CAR-NK and CAR-T.
#AI/#ML in biotech is one of the hottest new spaces and it has many critics as many people are resistant to learning new tricks. This can reduce the costs and time of discovery and drug development significantly.
$RLAY is one of my favorites as they have a platform of licensed and built technology that allows them to design drugs to target proteins. Its about combining CAD design, #AI and #ML to predict how proteins move and how to best target them.
$EXAI has 2 really awesome platforms. The first is their single cell biopsy that uses genetic information to pick the best treatments for cancer patients. The other platform uses all the collective knowledge of scientific data to use #AL and #ML to pick new drug targets.
$RXRX is a very unique company that uses robots to run millions of experiments a week and feed all that data into their supercomputer. It can use #AI and #ML to find and discovery new possible drug targets.
#SynBio is all about engineering microbes to reprogram them to make materials for manufacturing like fragrances or flavors. It has the potential to go so much further by engineering new life.
$DNA is my top #SynBio company. They are focused solely on engineering microbes to produce anything that a company might need. They produce the Heme for the fake meat to make it takes like real meat. There is unlimited potential in this space.
$TWST is my other #SynBio play. They are all about DNA. They do DNA synthesis and sequencing. They work with companies to develop drugs with their library of DNA data. They are even developing a DNA based storage system for data storage.
#CRISPR is all about doing gene editing. There are a few different editors out there now and more being developed all the time. Its fast, cheap and very easy to use. Along with its accuracy and safety to date, make it the best editing technology.
$BEAM is the king of the base editors. They have many partners which will pay them royalties to use their technology. They also have an ambitious pipeline of drugs for both ex-vivo and in-vivo gene editing. They are focused on genetic diseases and oncology.
$NTLA is one of the 3 original CAS9 companies. They have an ambitious pipeline around in-vivo liver editing. They are also working in the ex-vivo cell engineering of cells for oncology. They are working on their own version of DNA writing with their Rewrite buy out.
$VERV is other base editing company. They license it from $BEAM, but use it for different indications around cholesterol. I think they have a great management team which would propel them to great success.
#TPD is using targeted protein degradation to target proteins that were previously thought as undrugable. They are also going back and targeting proteins that were hit before with other technologies, but they show far less toxicity.
$KYMR is my favorite here as they are working on an IRAK4 and STAT3 degrader. They are focused on inflammation, fibrosis and oncology for their targets. They have huge potential if they show these targets lead to benefit in many inflammation or fibrosis diseases.
$GLUE is my other higher risk spec in this space. They are using #AI to develop molecular glues which have a different profile from the bigger bi-DACs like used by other companies. There is huge potential for them in inflammation and oncology if they targets work out.

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More from @Biotech2k1

Jun 17
I just got to go over the $SANA data, and I have to say I am a huge fan of this platform. They now have data in both mice and NHP for Hypoimmune (HIP) cells. They created islent, retinal, and cardiomyocytes. None of the iPSC HIP cells were destroyed by immune competent animals.
This is game changing as they look to have a real cure for Diabetes that won't require the complicated capsule that $CRSP is using. They could dominate this market. This is just the start of the many programs they have going on.
That program alone could be worth billions. Then they have the other cell which includes CAR-T both in-vivo and ex-vivo. This is amazing data. I think if I had to do my iPSC ranking today, I would put them at:
Read 4 tweets
Jun 17
Looking at $MRTX value:

An updated valuation of Mirati.
1/ Their first drug is Sitravtinib which has been in a very long Overall Survival phase 3 for NSCLC patients who are resistant to PD-1. The early data showed about 20% of patients treated with Sit returned to a response with PD-1.
2/ I think this would cover about 10,000 patients. My conservative number is around $500 million sales. If I put a 2x multiple on that since its phase 3, it give about $1 billion valuation to Mirati.
Read 7 tweets
Jun 17
Looking at $BPMC value.

An updated look at the value of Blueprint.
1/ The first thing with this company is it is already commercial with 2 drugs which are Ayvakit and Gavreto. These are huge indications. My estimates say they are peaking around $200 million sales. They do help fund the rest of the pipeline which is very powerful.
2/ I would put a 3x multiple on those sales as they are already plateauing. That would make the commercial sales of the business worth around $600 million value.
Read 8 tweets
Jun 16
I have heard a lot about the $COGT data for Advanced SM. When you look at the data on the surface it looks competitive. When you look at the comparable data, it doesn't look so good.
The first this is most (9 out of 11) of the $COGT patients were treatment naive. They had no other prior therapies. These are the easiest patients to target as they are not beat up by prior therapies.
The other thing is they claimed superior safety over "other KIT inhibitors" but some of the side effects they listed were not even related to treatment from Ayvakit like the intracranial hemorrhage. That patient has an accident.
Read 5 tweets
May 31
Cell Therapies 2.0 Part 8:

The final chapter for TIL therapies.
1/ Tumor Infiltrating Lymphocytes
2/ The Tumor Infiltrating Lymphocytes (TIL) approach is a TCR therapy, but it works differently. They excise the primary tumor from the patient. They isolate the T cell from inside the tumor. Then they genetically identify all the unique antigens for that specific patient.
Read 8 tweets
May 31
Update on my strategy portfolio:

I picked my 7 companies for the growth side of things. Since I already have a full biotech portfolio, I focused on Tech for this portfolio. I went with a 70% tech and 30% income ratio.
Here are my 7 tech picks:
#Fintech
1. $SQ
2. $AFRM
#Automation
3. $PATH
4. $PLTR
5. $U
#EV
6. $LCID
7. $RIVN
I was going to go with small cap growth stocks, but figured I should probably go with large caps for the stability. I picked my old favorite $CM and $VZ as 2 of them. I will find a 3rd soon for the income side of this portfolio.
Read 4 tweets

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