1/ I am planning some threads on cell therapies. I want to start with a history of cell therapies with Autologous CAR-T with companies like Kite, Juno and $BLUE. Get into the limitations.
2/ Then cover the transition to Allogeneic with companies like $CRSP, $ALLO, $DTIL, $ATRA, $TCRR, $CRBU. Cover the limitations and benefits over Autologous.
3/ Then I want to cover TCR therapies and how they address the unique issue of intra vs extra cellular antigens with $ADAP and $IMTX.
4/ Then I want to get into the #iPSC technology and how #CRISPR took the iPSC technology to the next level.
5/ If you any company in this space you want me to cover, just let me know.
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This will look into the differences between cell therapies using TCR therapies and those using CAR-T. Both of these technologies have their advantages and disadvantages.
1/ A T cell receptor (TCR) therapy uses the original T cell receptor. Some of them will sort and select donor T cells looking for the correct ones that react to the antigen they desire. Other programs will edit the TCR to change its antigen of target to the one desired.
2/ All T cells work off of Human Leukocyte Antigens (HLA). This is the same antigens that govern the process of rejecting in organ transplants. When using these T cells, they have to be matched from donor to recipient.
Back in 2013 and 2014, the idea of CAR-T therapy exploded onto the scene in biotech with the public offerings of companies like $BLUE, KITE and JUNO.
1/ These companies extracted T cells from the patient, edited them to insert the CAR receptor, and put them back into the same patient. This was the first CAR-T cell therapy treatments for oncology.
2/ These companies were red hot and had massive valuations (remind you of anything else?). Then they had a few set backs with both JUNO and KITE experiencing some deaths in their trials. The stocks got wrecked.
I haven't been in this company very long, but I think Jason Kelly is doing a great job so far. He seems to understand the responsibility of making ever dollar count as an early stage biotech company.
2/ Science:
They are engineering cells for use as factories in manufacturing. Its all about engineering life with a purpose. They are broken down into 2 parts. The first it the codebase which is all the genetic information and the clinical results of the edits they observe.
I am going to break this down into 2 companies that do the sequencing and data collection and 2 companies that do the equipment for the sequencing.
1/ The first company that does the sequencing and data collection is $NVTA. I think they are building a world class company across all areas of sequencing from genetic testing to fertility and even oncology.
2/ They have taken on a very risky and aggressive strategy of huge spending to lay the foundation for the future of personalized medicine. There is a lot of risk in this strategy as they have accumulated a lot of debt and have a massive cash burn.
This has been a space that really needed new technology after decades of older technology failing to work. The need for precise edits without off target effects still exists.
1/ The top pick here is clearly $BEAM with their base editing technology. They are using a modified CAS9 that only has 1 cutting domain. This allows them to use a deaminase to modify a single base and cut the opposite strand for repair.
2/ This technology has limitations as it can only do ABE and CBE modifications. It can't do insertions. There is still a ton of potential across hundreds of genetic disorders for this technology. They could do billions in revenues if the science works.