Thread by @Biotech2k1

Looking at $SANA:

This company gets no recognition as its not owned by some of the funds that lead to cult like status for some companies. They are probably one of the most disruptive companies in biotech.

1/ I think $SANA could be taking on all the things the CRISPR companies are doing ex-vivo and doing it in-vivo. They are combining 3 of the most powerful technologies in the market today with Fusogen, CRISPR and iPSC.

2/ The fact that this company is only at $3 billion market cap when they are at $5 to $10 billion is a gift for the retail investors. They could be bigger then all the CRISPR companies combined.

3/ Let us take a look at the pipeline, the science and the potential. I have to reign in my excitement a bit because I could lay out a case for this being the one company that hits $1 trillion market cap in biotech.

4/ They have 2 parts to their pipeline. The first half is using the Fusogen delivery technology to do in-vivo cell editing in T cells, liver and stem cells. The other half is about creating hypoimmune iPSC cells.

5/ Fusogen is a virus like particle. It takes the best of all things we know about viral delivery and combines it with LNP. This is a particle structure based of paramyxovirus which is in the 13kb to 19kb size. Big enough to carry any genetic payload.

6/ It combines that with a cell targeting ligand and a fusion protein that allows cell entry. This can target liver cells, T cells and even stem cells. They could take and edit stem cells inside the patient to fix SCD instead of the complex ex-vivo process of other companies.

7/ They are working first on doing in-vivo CAR-T therapies. This would be faster and safer for patients by using the patients own T cells in a safe and easy IV.

8/ Now this process does have its limits as they can only do the insertion of the CAR and not a bunch of different edits. This is autologous in-vivo CAR-T therapy which is revolutionary and validates the technology.

9/ This Fusogen vector can be used to delivery any gene editor or therapy into the liver or stem cells. With the right editing technology they could do everything every CRISPR company could do and do it in-vivo like fix stem cells for SCD.

10/ Just with these fusogen programs around CAR-T, Liver and Stem cells, it could be worth over $5 billion in long term revenues. There are hundreds of diseases from cancer to stem cells they could take on.

11/ The other half of their technology is about creating cell therapies that are completely invisible to the immune system. They have had tons of success transplanting these cells into NHP with fantastic results.

12/ The hypoimmune system takes the iPSC and knocks out the HLA-I, HLA-2 and upregulated CD47. This overcomes the T cells, NK cells and Macrophages.

13/ They can take those hypoimmune iPSC cells and then take them into anything they desire from T cells to islet cells to heart cells and to glial cells in the brain.

14/ They already have some very impressive mouse data for these cells in diabetes and repairing myelin in MS mice. The potential of these cells is in the tens of billions of dollars. They could cure MS with these kinds of programs.

15/ I know $CRSP is using a capsule from Viacyte to insert islet cells into the body. This system avoids the immune system completely with hypoimmune islet cells. The mouse data is really good for this program.

16/ While everyone else is getting all excited about genomics and cute buzzwords, $SANA is really disrupting the biotech world. The fact that those people haven't discovered it yet, is a bonus to the rest of us.

17/ Obviously, I got to temper the enthusiasm since this is still in mouse and NHP studies. They should be moving into the clinic in 2022 for the in-vivo CAR-T. This won't be about having the best CAR-T program. It will be about proving they can edit in-vivo with fusogen delivery