1. Looking at the CRISPR landscape.

This has been one of the hottest spaces the past year. I was in this space when everyone hated CRISPR. It was all about mutations and risk. Now its all about changing the future. The risks are still there, but only the sentiment has changed.
2. I got into $CRSP back in the fall of 2018 when they published their preclinical data on SCD. The NHP data showed it was superior to the current companies everyone loved like $SGMO and $BLUE. I had even owned $SGMO at the time.
3. I opted to go with the science and bailed on the over loved $SGMO and dumped into $CRSP. The first generation CAS companies use double stranded breaks. There is a high level of risk that mutations will occur over time. This isn't something that pops up in a few months.
4. It can take years. The fear in the midst of the frantic euphoria for these companies it they are a ticking timebomb of cancer. It could take 3 to 5 years to relieve these fears. I have been playing this space with one foot out the door locking in profits frequently.
5. This includes $NTLA, $CRSP and $EDIT. No one is going to doubt the data. This editing technology works and it works powerfully. If there was no fear of mutagenesis, no one would have developed the base editors. The fact that base editors exist tell us they are really scared.
6. Then along came $BEAM with base editing. I got into this one right after the IPO. It was my new hot play as it was working to address all the issues of 1st generation editors. It was no perfect, but it was a great bridge to what could come next.
7. Base editing still has a lot of risk with off target edits of the guides, by-stander edits of the deaminase, and it can not do insertions any longer as a base editor. It eliminated the risk of mutations from indels, but created other risks.
8. After the $NTLA data in hATTR, there has been a massive craze around CRISPR. This has brought out a flurry of new companies and science to be published as everyone wants to capitalize on the craze.
9. That brought out $GRPH as an IPO. This company still uses Double stranded breaks, but inserts using a homologous DNA strand. I find a lot to worry about with this technology. It seems the market thinks so too based on its valuation.
10. Then came $VERV that is a $BEAM knock off. They licensed their tech from $BEAM and pretty much owe half of anything they develop to $BEAM. Yet it gets a $3 billion value. Go figure. Its might as well be a subsidiary of $BEAM.
11. $CRBU was the last to go public, but it was one of the original CRISPR companies. It just waited so long to go public. They are focused on cell therapies. That has not really appealed to the CRISPR fanatics which is just fine with me. Its the only CRISPR company I still own.
12. At some point the craze will break and these companies will get valued based on other similar companies in this space of biotech. That could be a very powerful correction.
13. I still like the science here, and I still follow the space. I just can't get behind the level of irrational exuberance in the space. That never ends well. Its been a very rewarding space. $BEAM is up over 500% and $CRSP is up over 600% and I moved to the sidelines.
14. There has been a big correction so far this year in this space, but I think its still trading at 3x the value of any other biotech at this same stage of development. Many of those other companies have as much or even more potential as CRISPR.
15. There will come a point in this correction to get back into the CRISPR names. It has to make the risk worth the reward. Right now the risks are high and the values are way to high for that risk.

• • •

Missing some Tweet in this thread? You can try to force a refresh
 

Keep Current with Biotech2k

Biotech2k Profile picture

Stay in touch and get notified when new unrolls are available from this author!

Read all threads

This Thread may be Removed Anytime!

PDF

Twitter may remove this content at anytime! Save it as PDF for later use!

Try unrolling a thread yourself!

how to unroll video
  1. Follow @ThreadReaderApp to mention us!

  2. From a Twitter thread mention us with a keyword "unroll"
@threadreaderapp unroll

Practice here first or read more on our help page!

More from @Biotech2k1

28 Sep
1. Looking at Cell Therapy Landscape.

I got into this space during the early days of Kite and Juno with CAR-T. The idea of taking an immune cell and using editing to reprogram it to fight cancer was about the coolest thing ever.
2. The data for these programs was amazing for the blood cancers where tumors were easy to reach and antigens were ideal. These companies got bought up as the dream was CAR-T would be a huge success for all cancer.
3. Then along came solid tumors and CAR-T failed to impress over and over again. The challenge was that CAR receptors are great for surface antigens like a CD19 or BCMA, but the majority of solid tumor antigens were inside the cells.
Read 19 tweets
27 Sep
1. Looking at the Protein Degrader landscape.

I guess I can claim I was in the original protein degrader company with being an early Celgene investor. The idea of protein degradation came about from the discovery of how Revlimid worked. For many years, no one knew how it worked.
2. The understanding of how it targeted the E3 ligase Cereblon to degrade several key transcription factors led to the idea that we could harness the Proteasome as a tool to develop drugs to destroy harmful or unwanted proteins.
3. For a very long time, I was not a huge fan of the CelMods that Celgene was developing based off this technology. The response rates in cancer were modest in the mid 30% range. Then newer companies began coming to market fully focused on this science.
Read 14 tweets
27 Sep
1. Looking at the Pathways landscape:

When I look across pathways, I see so much potential. There are so many receptor of tyrosine kinase drugs out there that work very well like EFGR, HER2, MET, RET, ALK and so many others.
2. These drugs are well developed, but there is a need for newer generations as resistances develops. The two companies I own that are mostly focused on receptor of tyrosine kinases are $BPMC and $TPTX.
3. $BPMC is working around PDGF, KIT and RET. They have some early stage drugs for 2nd and 3rd generation EFGR mutations. $TPTX is working on smaller molecule 2nd generation inhibitors around ROS1, NTRK, RET, MET and ALK.
Read 12 tweets
27 Sep
#Viral_Structure

A look at binding proteins and membranes.
1/ In the first 2 threads we looked at the viral genome and the protein capsid. There are 2 other major structural components of the virus with the binding proteins and a membrane.
2/ All viruses come with proteins that project from their surface. These are ligands that bind to specific receptors on cells to gain entry. These proteins give the virus its tropism.
Read 15 tweets
26 Sep
#Viral_Capsids

A look at viral capsids.
1/ The capsid is a strong protein structure that encloses and protects the viral genome. The most basic viruses will use a single protein produced many times to build the capsid. The more complex viruses will use multiple proteins to build their capsid structure.
2/ The basic structure of the viral capsid comes in 3 basic designs. They are Icosahedral, Helical and Complex.
Read 18 tweets

Did Thread Reader help you today?

Support us! We are indie developers!


This site is made by just two indie developers on a laptop doing marketing, support and development! Read more about the story.

Become a Premium Member ($3/month or $30/year) and get exclusive features!

Become Premium

Too expensive? Make a small donation by buying us coffee ($5) or help with server cost ($10)

Donate via Paypal Become our Patreon

Thank you for your support!

Follow Us on Twitter!

:(