I have been in this company for about 8 months now. I think the management is good, but way too early to tell. They are my small cap high risk play.
1/ They are using CAAR-T to target autoimmune disorders. They use a CAAR receptor that encodes a self antigen that drives auto immune disease. The concept is the self reactive B cells should target binding to the CAAR receptor and the T cell would kill them.
2/ Based on the really low level of memory B cells in the body, it could be very difficult to clear them out. Its a high risk play, but if it works, it could be a huge opportunity.
This is a newer company that just went off its lock up period. I don't know the management that well, but it can't be any worse than the disastrous $IOVA. That company is completely broken.
1/ They are working on Tumor Infiltrating Lymphocytes (TIL). This is the most successful use of TCR therapies in solid tumors. This starts with removing a met or doing a biopsy of the tumor. It takes that tumor and breaks it down to get all the antigens for that specific patient.
2/ Those tumor antigens are introduced to the patient's own T cells in the lab to activate them toward those antigens. Those T cells are then put back into the patient with PD1 to target and kill all tumor cells and mets.
This is a new IPO and they are still within the lock up period until late January. I have followed them as a private company at the ARM conferences as part of my overall CRISPR research for years.
1/ I can safely say I feel this is a good management team at this early stage of the game. They are the original CRISPR company holding all the original patents. They also pioneered new technology with chRDNA.
2/ I classify them as cell therapies because that is where their focus is. They have 3 allogeneic CAR-T programs in development while building an iPSC manufacturing for iNK and iT cells.
This is a newer company that recently IPO'd. They are still in the lock up period until mid December. I don't know anything about their management yet. They are working on iPSC cell manufacturing using CRISPR MAD7 editing.
1/ The MAD7 resembles the Cfp1 CAS enzyme. They are focused on developing 2 NK cell programs as their lead programs. They have CNTY-101 which is a standard CD19 and they have CNTY-103 which has CD133.
2/ They are targeting GBM with CNTY-103 which makes this a completely unique program compared to what some other companies are doing. They have some other early CAR-NK and CAR-T programs in development using iPSC.
I have only been in $SANA for about 6 months, but I really like their 2 franchises. I also like their management team, although, I don't know them that well.
1/ Their first franchise is the Fusogen programs. This is all about delivering any genetic payload to cells. Their fusogen platform uses virus like particles. If we treated this as a stand alone biotech, I would value it around $1 billion to $1.5 billion.
2/ Their second platform is working to build iPSC manufacturing around hypoimmune cells. This will be right along the lines of what $FATE has built. I would also value this platform around $1 billion to $1.5 billion.
I have been in and out of $FATE for over 3 years now. I first bought it back in 2018. I kept the company all the way up till $100 where it just seemed to expensive sold I sold it and waited. I recently started buying a little back.
1/ They are the pioneer of the iPSC platform. I remember when they started with allogenic NK cells which since then they dropped. They also had ProTmune which was a stem cell product for transplants. They also discontinued that program.
2/ If we are really counting the track record, they had a lot of wrong answers until they had a right one. What they learned from engineering stem cells and NK cells led to the programs they have today.
I just into $GLUE not long ago. The company is still with in the lock up period till around Christmas. I don't know the management, but I find the pipeline to be very exciting.
1/ $GLUE is completely focused on molecular glues. They have a very exciting pipeline around some unique targets. Their lead indication is for Myc driven cancers. This accounts for 10% of NSCLC and 50% of SCLC. That makes it a very nice sized indication.
2/ Depending on the data, it could be well worth over $1 billion sales if the data works out. They are planning a preclinical data read out at AACR next weekend. That could move the stock.
I just recently started investing in this company. I don't know very much about the management yet. They do have a broad pipeline of targets around oncology using both heterobifunctional and molecular glues.
1/ Their lead indication is a molecular glue that binds to the CRBN E3 ligase and target IKZF1 and IKZF3 transcription factors. This is in development for Multiple Myeloma. This is a nice size indication where Revlimid earns about $2 billion per year in this space.
2/ Their second drug is a heterobifunctional molecule targeting the BRD4 protein. This is an protein that plays a role in DNA packaging and gene expression. The indications for this drug seem very small.
I just started investing in this company recently. I don't know the management well yet, but I love their science programs. They are using heterobifunctional degraders like with $ARVN.
1/ Their first indication is IRAK4 in inflammation. This is known to play a big role in inflammation and oncology. They are targeting multiple indications for inflammation.
2/ So far there is only biomarker data, but that is encouraging. The potential of IRAK4 in inflammation could be worth a billion per indication. I think $3 to $5 billion is not unrealistic should it work out as well as hoped.
I just started investing in $ARVN a few months ago. I was waiting for more of the data to validate the whole protein degrader thesis before I got in. Too much science fails to not wait for some data.
1/ Their lead program is for Estrogen Receptor + cancer. There is a huge amount of competition in this space. 75% of all breast caner is ER+ so its a big indication. They needed to do a partnership to drive market share for this drug.
2/ Their second drug is for Androgen Receptor + CRPC. This is another very competitive space. They will probably need a partner here or they will do poorly. Their drug is not as impressive as the ER indication. Probably why they have 2 follow on AR drugs in development.
My last pathway company to profile for today. They are unique as they are focused on the DNA damage repair pathways. This is a new frontier of discovery. There are not a lot of companies in this space yet.
1/ They are my first and only pure Synthetic Lethality play. Their first drug is around the ATM/ATR pathway that regulates double stranded breaks. This concept looks for pathways that cancer depends on to drive growth. It targets them to kill the cancer cells.
2/ The ATM/ATR pathway has been attempted before. It has concerns with toxicity. There is a level of concern and caution about trying new things in this same area that failed before.
It took me a long time to warm up to $SDGR. For a long time, I just dismissed it as a tech company. I could care less about a tech company. Then I found out they were developing their own pipeline.
1/ Then again, I dismissed them as a tech company pretending to be a biotech. What would a tech company know about developing drugs? I listened to a few of their presentations from their science guru. That is when I thought they had potential.
2/ They have a lot of partnerships for companies using their software developing drugs. I am not going to cover any of those as they all have different terms and levels of profit sharing. They do offer potential should they work out.
I got into this company about 6 months ago. They are an early stage company with no data yet. They have a big risk/reward profile with using AI to attempt to take on some of the most toxic targets in pathways.
1/ I spoke before about how the SPH2 and PI3K kinases are at the top of the MAPK and mTOR pathways. Being at the top of the pathway gives them strong suppression effects, but a lot of toxicity. $RLAY is taking on these targets using its AI platform to develop inhibitors.
2/ Their fist program is around SHP2 which they already partnered out. This helps mitigate any failure if the technology doesn't help improve this target more then other companies. We have no data yet on this program. This has been turned over to the partner so data is waiting.
This is a new IPO, but I jumped on it because of its management. This management was from the old Ignyta which was very successful for me in the past. I go into Ignyta early and held it until the day it was bought out. I know this is a winning management team.
1/ They remind me very much like $RVMD with a big focus on the MAPK pathway. They have no data yet, but they did dose their first patient. I would expect some early data in the Spring of next year.
2/ Their first targets are all about MAPK and using combinations of target to lock down this pathway in cancer. Many drugs will suppress the pathway, but it rebounds over time.
I got into this company early this year. I love the pathways they are targeting, but they have little data or success yet to show they have great science yet.
1/ Their management is still new to me as they haven't had much data yet. Their first program was for SHP2 which had lack luster data. At least they were wise enough to partner that away for some cash. They are highly focused on the internal cell growth pathways.
2/ These are the MAPK and mTOR pathways. These are some of the hottest pathways in all oncology. The problem is the closer to the receptor you go for the pathways, the broader the suppression and the higher the toxicity.
I got interested in $MRTX about 3 years ago while I was an Array Bio investor. They licensed some of the KRAS technology from Array. That got me interested.
1/ $MRTX just replaced their CEO. Chuck moved up to Chairman and they hired David Meek to groom into the new CEO role. He has a history with commercial experience. That is good for taking the company to the next level with approval and sales.
2/ Their original drug is Sitravatinib which hits multiple kinases. This is designed to alter the tumor microenvironment to shift things back toward cellular killing. It had some decent data for patients who developed resistance to PD-1 inhibitors.
I have been in this company just over a year now. I am pretty sure the management is strong, but it hasn't had the chance to navigate the entire process yet. They still need to show they can get a drug approved and execute on selling it.
1/ This company is working on the growth receptors pathways. They are a 2nd generation of kinase company. They are developing much smaller inhibitors that can get in there and still inhibit the kinase even with mutation. That is their big tech development.
2/ Their drugs are designed to get around and overcome resistance. Will they develop resistances of their own? My money would be on yes as cancer always mutates to find a way around most drugs.
I have been in this company for over 3 years now. Its been a fantastic company. They have top quality management.
1/ They have 4 commercial indications already. These 4 are relatively small, but they validate the pipeline and the ability of management to develop and commercialize drugs. This is one of the most trusted managements I have found since my early days with Celgene.
2/ Their fist indications are Ayvakit for D842V mutations of PDGFRa in GIST. This is a very small indication accounting for about 5% of GIST. I am sure with its data, it gets used off label for other GIST patients. I think this wont account for more than $50 million sales.
Considering a radical bubbl-ectomy of my portfolio do to the massive bubbles in CRISPR and Protein Degraders. I am looking at doing this into the next big pop for the $XBI. Some of these valuation make me feel stupid just looking at them.
1/ My initial plan was to buy back into CRISPR names as the bubble imploded, but too much money chasing that bubble. I think I would better be served making money elsewhere. There is only money to be lost there.
2/ The protein degraders are getting dangerous at these values for all but poor $GLUE. I might cut them all loose on the next big pop in the $XBI.
This has been one of the hottest spaces the past year. I was in this space when everyone hated CRISPR. It was all about mutations and risk. Now its all about changing the future. The risks are still there, but only the sentiment has changed.
2. I got into $CRSP back in the fall of 2018 when they published their preclinical data on SCD. The NHP data showed it was superior to the current companies everyone loved like $SGMO and $BLUE. I had even owned $SGMO at the time.
3. I opted to go with the science and bailed on the over loved $SGMO and dumped into $CRSP. The first generation CAS companies use double stranded breaks. There is a high level of risk that mutations will occur over time. This isn't something that pops up in a few months.