Thread by @Biotech2k1

@HassanAesthetic Profile for $CRSP

1/ They are the other CRISPR company using the original CAS9 enzyme for editing. This enzyme has all the same risks as for $NTLA with potential off target edits and mutations. It still has all the versatility too. That is one of its great benefits. It can insert and delete.

2/ One of the nice things about $CRSP is they have been focused on all indications outside of the body. Their lead programs are in stem cells, T cells and regenerative medicines.

3/ This is a great place to start as the cells can be verified for quality before they are given to the patients.

4/ They are the most advanced with their Sickle Cell Disease stem cell therapy. They extract stem cells, edit them, verify their quality and insert them back into the patients. This is a long and complex procedure.

5/ The data has been outstanding and the closest thing to a cure I have ever seen. The patient population is huge for SCD with 90,000 each year in US and 300,000 world wide.

6/ The big constraint will be capacity. Sam already said he hoped they would reach 1,000 doses per year. I suspect they can ramp production over time. If you give them a price of $1.5 million, that would get to $1.5 billion sales per year.

7/ I think they could ramp that to nearly $3 billion a year, but I am not certain. They share this program 50/50 with $VRTX. That would attribute about $1.5 billion each year to $CRSP. This program is worth $15 billion market cap if you put 10x sales on it.

8/ It has the capacity to go higher, but it takes a long time to build and train that capacity. They will need to train sites for doing these procedures.

9/ Their second set of programs are around T cell editing for CAR-T. They have 3 basic CAR-T assets in development for CD19, BCMA and CD70. This is where I got disappointed with $CRSP and sold out.

10/ None of these CAR-T therapies are cutting edge for any of these companies. They are all just copying what the leaders are doing. That means, by the time they get to commercial, they will be already obsolete.

11/ I don't have a lot of big hope for these programs around CAR-T to earn a lot of money. They are not putting in the necessary time and research to lead the space. Maybe they get a few hundred million sales at best for "me too" products.

12/ The data so far in allogeneic CAR-T has been very underwhelming with less then 6 months of response.

13/ Their last program is regenerative medicine and it was my favorite long term. They are working toward using CRISPR editing and stem cells to guide cells toward new applications like islet cells for insulin.

14/ Their first program is for diabetes. They create islet cells and implant them within a Maxcyte device to produce insulin. This could be a big deal, but I saw a ton of data showing these devices produce a lot of long term side effects with immune reactions.

15/ I think my grand dream of using CRISPR + stem cells to grow entire organs for transplants is still decades away. I was a huge fan of $CRSP, but its lost its luster as they are failing to continue to innovate.

16/ Other than their SCD, they haven't advanced much of anything for years. I would say they are falling behind as other newer innovations comes to market.

17/ I think the valuation is attractive, but probably justified as they are not nearly as exciting anymore compared to other editing programs going on. I think the SCD program is worth $3 billion at least and maybe up to $5 billion value.

18/ If you throw in the cash on the books, I think this could be worth $7 billion on a valuation. That puts it at about $91 for valuation. That makes it not too crazy at only $9 billion valuation.

19/ Their innovation might change once they get SCD to commercial, but they have a ton of catch up to do. They could use that cash from SCD to buy the innovation eventually.

That is an error. Its Viacyte not Maxcyte. I got my companies confused lol.