My Top 10 Ideas
These are my top 10 ideas. They are just my picks. They should not be considered investment advice. You should always consult your financial advisor before making any investments. These are very speculative companies and the risk is very high.
#1 Beam Therapeutics
Beam Therapeutics is a Base Editing company working in the CRISPR space. They are the only company that controls all the technology behind base editing. They have several very big partnerships with Pfizer, Verve, Apellis and Sana. They have a program in early phase 1 for Sickle Cell disease. This is a very large indication with 90,000 in the US and 300,000 patients world wide. They have a few follow up in-vivo indications for genetic disease like AATD and GSDIa. They are working in an early T cell therapy using base editing. If base editing works out, there are thousands of genetic diseases that could be treated with this technology.
#2 Ginkgo Bioworks
Ginkgo Bioworks has 2 franchises and both of them have huge upside potential. The first is their Cell Engineering platform that does engineering in the synthetic biology space. This works very much like a pipeline where they spend years developing a product before it goes commercial and they earn royalties. They have over 60 programs in this pipeline with an expected 100 more to come in the next year. Each successful program adds more potential downstream royalties. The other platform is around biosecurity. This is all about detecting, tracking and containing pandemics. As technology makes the world smaller and smaller, the chances for another major pandemic grows. This is something that is preventable and can save lives.
#3 Relay Therapeutics
Relay Therapeutics is a TechBio company working on developing new drugs for cancer using Artificial Intelligence and Machine Learning. They model the behavior of proteins so they can figure out how to target them. They are taking on some of the hardest targets in cancer. They have some of the most impressive data ever for a FGFR2 drug with an impressive 88% response rate. If they can carry over that success into PI3Ka, they will have a huge blockbuster with over 100,000 patients for this indication. They are developing drugs for some of the largest indications in cancer like Breast Cancer.
#4 Sana Biotechnology
Sana Biotechnology is building 2 platforms which could both be very big. The first is their delivery program around Fusogen. This is their virus-like particle program for in-vivo delivery of genetic editing. This is still in early stages, and the company admits it needs some technology breakthroughs to make it work at commercial scale. If it does work out it adds a massive upside for Sana. The other program is iPSC cells. They are developing therapies with immune evading edits. They are developing several cells like islet cells, T cells and other cells. There is massive potential in cell therapies depending on how well the technology works out. Diabetes alone with islet cells could be worth billions.
#5 Kymera Therapeutics
Kymera Therapeutics is developing Targeted Protein Degraders. They have a target for inflammatory disorders with their IRAK4 drug in development. They are still in early stages, but the data shows some proof of efficacy in some inflammatory disorders. If this works out in some of the bigger inflammatory disorders, this can quickly become a very big drug. They are also working on another drug for Fibrotic Disease. They have several drugs targeting cancer.
#6 Intellia Therapeutics
Intellia is a first generation CRISPR company working on gene knockout in the liver. They have some very successful data in 2 indications now with hATTR and HAE. They are advancing other in-vivo programs into the pipeline. Gene knockout is one thing first generation CRISPR can do well. This gives them a lot of potential in these indications while they work toward building their own precision editor from their Rewrite buyout. I think aTTR alone could be worth a few billion in revenues if it works out well. It is a very competitive market so it is hard to know how much market share it can take from other therapies.
#7 Recursion
Recursion uses robotics to run millions of experiments every week. All the data is captured by their Supercomputer where it can be analyzed by their Artificial Intelligence and Machine Learning. This allows them to develop drugs from all of that data. They even license access to this data to other companies. They have 3 indications in phase 2 with others coming into the clinic. All of their indications have a very nice size market for them. There is an endless amount of potential this system could produce if it works as well as we all hope.
#8 Invitae
Invitae is a genetic testing company with a focus on Oncology, Women's Health and Genetic Disease. They use kits to collect samples which they process using Pacific Biosciences machines. They collect that data into their genetic database. The data is shared with patients and doctors. There is also a use for the technology to help scientists find the right patients for clinical trials. There is a level of population genetics information that is useful here for researchers. I think they have the potential to become the Google of genetics data if it all works out. This one has a higher level of risk as they have about $1.5 billion in debt and still burn a lot of cash. There is a high level of risk they go broke before they succeed.
#9 Revolution Medicines
Revolution works in the Kinase space. These are the many growth pathways that exist inside cells that drive cell growth. Many of these pathways mutate in cancer to drive cell growth. They are developing drugs around the key RAS kinase which is mutated in upward of 30% of all cancers. They first drug targets multiple mutations of this RAS kinase. This had some early safety and efficacy data. The patients all had stable disease or better in the early trial. The safety seems tolerable and manageable so far. If this drug works out, it could be a huge blockbuster of the like of Revlimid. They have several other drugs in development around specific mutants of KRAS along with several companion inhibitors. These are designed to work with RAS inhibitors to drive better effects.
#10 Century Therapeutics
They are an iPSC cell therapy company. They are building cell therapies from common progenitor cells for NK and Gamma/Delta T cells. They are working on using these cell therapies to avoid the immune system. They are focused on multiple cancer indications. Some of these can be pretty large. I really love their science, but they end up at number 10 because I am not sure they could be a really big company. Much of that will depend on which cancer targets and indications they pick. This is still early in phase 1 so we need to see the data first.