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Biotech2k

@Biotech2k1

28 Sep

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.

Read 15 tweets

Biotech2k

@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

Biotech2k

@Biotech2k1

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

Biotech2k

@Biotech2k1

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

Biotech2k

@Biotech2k1

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

Biotech2k

@Biotech2k1

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

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