Thread by @Biotech2k1

Taking a look at the #CRISPR landscape.

This is a sector that went from hot to not in a real hurry lately as the biotech sector has collapsed. Here I am going to go over the sector and where I think the opportunities are.

1/ I hear people talking about how far the CRISPR space has fallen. Some think its too far and some think its not far enough. What I do know is that this space has developed a lot since its bottom in 2020.

2/ We still have very strong data from $CRSP in SCD. Their data is second to none. This is a very huge indication where the only limit is capacity. We got very promising early data from $NTLA in in-vivo gene knockout.

3/ $BEAM has come a long way moving its first program into the clinic next year. They have singed multiple very meaningful partnerships with $VERV, $SANA, $APLS and Prime.

4/ The potential for the technology is just as large as it was when all the hype was driving these stocks to all time highs. There are still hundreds of genetic diseases that could be addressed with this technology.

5/ Just the amount of ex-vivo applications around cell therapies could be multiple billions in potential. They have all the CAR-T and CAR-NK applications. They have all the stem cell applications.

6/ I even think there are other cells from islet to heart to neurons that could have applications. Then there are the potential for Liver indications. There are several dozen that could be addressed with LNP delivery.

7/ Over time, they will develop new delivery methods which will gains new access to new tissues with CRISPR.

8/ Now that we looked at the potential, let us look a the quality of the individual companies. I think $BEAM is my top pick. There is a lot of risk around indels with first generation companies. Base editing and Prime editing address many of these issues.

9/ $BEAM also offers a platform in high demand for partnerships. What they don't build for themselves, they can license to others at very attractive royalties.

10/ When it comes to the first generation companies like $NTLA, $CRSP and $EDIT, the fear of indels has not emerged yet. If they turn out to be unwarranted, then these companies will have huge potential. I think $NTLA and $CRSP have strong fundamentals.

11/ I would urge much caution on $EDIT. That company has been a train wreck since it started. They have had one bad management after another and their data is still the weakest of the group.

12/ I am not a big fan of $VERV, but they are doing base editing. I think their space of cholesterol and triglycerides is very competitive and might no have a space for gene editing options. Its a much higher risk play.

13/ $GRPH has been the red headed step child of the CRISPR space. I have pointed out issues with their science around efficiency. There are other risks with attempting gene insertions using double stranded breaks even with HDR repair.

14/ The last company in this space is $CRBU. I think they have some exciting potential around iPSC cell engineering. They seem uninterested in other applications of CRISPR which seriously hinders their potential in my opinion. Why no in-vivo programs? Why no stem cell programs?

15/ I personally like $NTLA, $CRSP, and $BEAM. I think $CRBU is a cheaper and higher risk play, but you might like one of the other higher risk plays.

16/ I think the valuations have really come in for many of these companies. Most of them are flush with cash and have long runways. I think $BEAM at less than $5 billion with all their partners is very attractive.

17/ $CRSP is down right cheap here with $2.4 billion in cash and a market cap just over $5 billion. I think $NTLA is the most expensive of them all for a company with just a handful of patient data. It could get to $80 before its reasonably priced.

18/ All the rest are cheap, but I fear many of those will be value traps. They have real issues and real concerns.

Happy Shopping.