Discover and read the best of Twitter Threads about #CRISPR

Most recents (24)

If you are looking for cheap biotech ideas, I suggest looking to these key themes. I call them my technology of the future themes. I will even throw in some names. Just be sure to do your own DD as this is spec biotech which is risky.
#TechBio
$RLAY
$RXRX
$EXAI
$ABCL
$SDGR
#CRISPR
$BEAM
$NTLA
$CRSP
$CRBU
Read 6 tweets
🧵 My “One Thread to Rule Them All” for $BEAM

Attempting to estimate revenues ‘til 2031

⚠️ Warning: This is VERY long

Figures are “Blue Sky Scenario”

At the end I’ll throw in more conservative estimates

🚨 If you love BEAM or #GeneEditing + #CRISPR be sure to LIKE & RETWEET!
$BEAM-101

Approval: 1H 2027

Wave 1 Market: 25k US/14k EU

Market in 4 years: ~27k total ($CRSP/$BLUE)

Cost: ~2m

27’: 500 patients= 1b
28’: 2000 patients= 4b
29’: 1b (ESCAPE looming)
30’: zero
31’: zero
$BEAM-201

IND: Approved Today! 😊

Approval: mid 2027

Price: $375k (Wedbush)

Market is small, so little effect on revenue.

Success IMO will unlock hidden value. I see multiplex editing as a big weapon vs. cancer in 2030 and beyond!
Read 26 tweets
New🔥TCR #CRISPR✂️ cancer #immunotherapy paper just out in @Nature by PACT Pharma & UCLA.

Why all the 📸headlines & press coverage?

This small proof-of-concept study can give us a glimpse into the future of cancer therapy.

Let’s unpack the details & relevance of this study👇🧵
First things first:

Here’s the link to the paper, which has been made public by @Nature in an unedited form (before official publication), due to its perceived immediate relevance to the research & clinical cancer communities.

nature.com/articles/s4158…
A. The context ✍️ of this work:

Most cancerous lesions have mutations in the 🧬of their cells.

Cancer cells (literally) display small pieces of cellular mutated proteins (called peptides) on their surface.

These peptides, called neoantigens, are important therapeutic targets.
Read 21 tweets
What is #TechBio

Everyone has heard of Biotech. These are companies that develop technology around develop drugs. Its a very biology driven endeavor. Its about looking at the science, coming up with ideas and then testing them in the lab to find ones that work.
1/ Where Biotech is driven by Biology, #TechBio is driven by technology. Its about using tools like Computer Aided Design, Computer Modeling, Artificial Intelligence #AI, and Machine Learning #ML to design drugs. Its a technology first type of drug design.
2/ I have heard of it referred to as the Bionic Scientist. I love that concept as it truly represents what TechBio is all about. Its about using tools of technology to enable Scientists to better develop drugs. Lets look at the statistics of drug development.
Read 18 tweets
The Private Company #CRISPR Report

I am going to build this thread going through the private CRISPR companies. This includes the few that I do know and like. I know there are more out there. I will add them as I discover them and take time to look over their science.
1/ Mammoth Biosciences

This is my top pick of the private companies should they go public. I think they are gearing up for an IPO. The success of the $PRME IPO might bring them to the market quicker.
2/ They have 2 platforms based off CRISPR technology. The first uses CAS enzymes for DNA or RNA as a Diagnostic tool. They take the guide RNA for the sequence they wish to find. They include a reporter strand that matches the DNA sequence they want to find.
Read 28 tweets
The #CRISPR Report:

I am going to build this thread all day as I go through all the public CRISPR companies. Make sure you keep up with it as I add each company as the day goes along.
1/ CRISPR Therapeutics $CRSP

This is a Swiss-American company based in Switzerland and R&D in Cambridge US. They are the most developed company with their first therapy exa-cel submitted for FDA approval. This is for Sickle Cell disease.
2/ The company trades at about $4 billion valuation. They have just under $2 billion cash. The burn rate is over $700 million per year. That is an excessive amount of cash burn. I don't see how they sustain that even with commercial sales starting later next year.
Read 64 tweets
TRANSFORM-HF

What do we really know about efficacy of furosemide vs torsemide?

I am sure @robmentz and team have something to say about this 👊🏾

pubmed.ncbi.nlm.nih.gov/33714745/

#AHA #LBCT @AHAScience @noshreza
Chlorthalidone versus HCTZ - pragmatic emr study by @Areefishani et al. After #SPRINTTrial I use chlorthalidone. What’s the evidence?

pubmed.ncbi.nlm.nih.gov/35390512/

#AHA #LBCT @AHAScience @noshreza
PPAR-alpha (e.g., fenofibrate, permafibrate) - Are triglycerides as a target out for CV event reduction, or just this mechanism?

pubmed.ncbi.nlm.nih.gov/30342298/ @APradhanBrigham to present 👊🏾

#AHA #LBCT @AHAScience @noshreza
Read 8 tweets
So glad to finally be able to share nanofiber micelleplexes with the world! 😀 pubs.acs.org/doi/10.1021/ja…. It turns out #tinysticks are good at delivering #DNA into cells! I just wanted to celebrate with a short 🧵 on how this work arose. 1/13:
Back in 2017 whilst finishing my PhD on anti-cancer #Gquadruplex ligands with @MCGalan_Bristol, I became very interested in nucleic acid therapeutics and technologies such as #CRISPR. Approaches like cancer immunotherapy seemed to me an elegant way of treating disease. 2/13
However, many of the problems facing these exciting areas of research lay in biology. As a synthetic chemist I was unsure of what I could do to help. 3/13
Read 14 tweets
The last mile for #T1D cure:
'I believe that cell therapy can be the T1D cure. We've had landmark results in the last 2y.
-L. Piemonti

#ISPAD #ISPAD2022 #dedoc°voices #NothingAboutUsWithoutUs
#diabetesadvocacy #patientvoice #payitforward @dedocORG @ispad_org 1/n
The last mile for #T1D cure:
'We know now that #BetaCell replacement can cure T1D. This is the #BG profile of someone who used to live with #diabetes 1y 1/2 ago.'
-L. Piemonti

#ISPAD #ISPAD2022 #dedoc°voices #NothingAboutUsWithoutUs #payitforward @dedocORG @ispad_org 2/n
The last mile for #T1D cure:
'We started #BetaCell in Milan replacement in 1989. Had to wait for 30y for this procedure to be recognised.'
-L. Piemonti

Can you imagine living without the mental load & physical burden of #diabetes?

#ISPAD2022 #dedoc° @dedocORG @ispad_org 3/n
Read 13 tweets
$NTLA and $REGN report initial data from ph1 study of #ATTR for NTLA-2001. All patients across the two doses showed 92-94% TTR reduction at day 28.
#crispr #genomics #genetherapy #biology #biotech
At both dose levels, $NTLA-2001 was generally well tolerated. 2 patients reported transient infusion reactions, which was the only observed treatment-related adverse event.
$NTLA-2001 is an in-vivo administered #genetherapy.
It is one of the first few in-vivo #GeneTherapy being tested. Congrats to the $NTLA and $REGN teams.
Read 4 tweets
Looking at a little Strategy: 🧵

I hope putting out my game plan will help other get ideas on how to do a strategy for investing to maximize returns.
1/ I am finally getting comfortable with my strategy. I have a set amount now that I am contributing to each of my portfolios for #biotech, #tech and #crypto. My plan is to use the any rallies to fade some of the companies I want get rid of that disappointed me.
2/ Then I want to use that cash to accumulate more of the companies I like. The max tax loss I can take for a year about $3,000. That would reduce my taxes and get me back about 25% or $750 in tax savings. It will work out like getting some cash back on those losers.
Read 14 tweets
Let us skip the obvious bubbles in the #CRISPR stocks into this biotech rally and focus on where there is still real value you can buy into any down days.
I love the #AI/#ML in biotech space. All of $RLAY, $EXAI, $SDGR, $ABCL and $RXRX are super cheap. I think they could all be accumulated into any selling.
I love the #CellEnigneering space. I think $SANA and $IPSC are super cheap still. I would throw in $TWST and $FATE as cheap.
Read 5 tweets
The most impressive science in biotech right now is:

1. #iPSC
2. #AI/#ML in biotech
3. #SynBio
4. #CRISPR
5. #TPD
#iPSC has to be doing some of the most amazing science in the entire sector right now. The funny part is they don't get all the hype as the other hot science spaces. They are completely engineering cells that have many new functions and remove many old functions.
They are making all kinds of cells from stem cells into eye, heart, liver, pancreas and immune cells. They don't get much love because of the bad data of the allogeneic CAR-T programs, but some of these companies are completely disruptive.
Read 20 tweets
The UK Govt says that before moving to #GMO deregulation it consulted with the "independent experts" on its Advisory Committee on Releases to the Environment (ACRE), whose view is that gene-edited organisms pose no greater risk than traditionally bred foods. But guess what👇
Meet Jim Dunwell, chair of the UK's "independent" #GMO advisory body ACRE. Jim used to work for the GM firm Zeneca. He gets a Syngenta pension. And he was a founding member of a GM industry-funded group that lobbies "to make the case for GM crops & foods." gmwatch.org/en/myth-makers… Image
If someone with a GM industry background & Syngenta pension who co-founded a pro-GMO lobby group doesn't sound like an "independent expert" on #GMOs, then you need to know he's far from alone, eg he's just 1 of 3 members of ACRE who all connect to Syngenta
Read 5 tweets
A team of marine biologists led by Scripps Oceanography's Amro Hamdoun (@Hamdounlab) has created lines of sea urchins — the marine equivalent of lab rats — that can be used as genetic models using the gene editing technology known as #CRISPR.

Read more: scripps.ucsd.edu/news/genetics-… A smiling, bearded, and bespectacled man holds a bin of sea
Described in a new paper out today in @Dev_journal, the genetic makeup of the #SeaUrchins is fully mapped and can be edited to study human disease genes. The creation of these new research model organisms will accelerate the pace of marine #biomedical research. A postdoctoral researcher processes a sample under the micro
Having this new “genetically enabled” sea urchin could dramatically enhance the efficiency, reproducibility, and utility of studies of the developmental origins of diseases. A staff technician works with sea urchins in an experimental
Read 4 tweets
Building my Strategy Portfolio:

I funded it today and it will be just like I am starting out from scratch. I got $350 in it and plan to contribute about $200 to $250 each month. I went through much of my portfolio design which I will share so it might be educational.
1/ This portfolio will not be a buy and hold portfolio. It will shift and rotate based on where I think the Macro economy sits and where the best deals are in the market. That is the first thing I want to establish.
2/ I set a few rules so I buy 1 share at a time since there are zero fees. I plan to not pay up for stocks, but not sure if that will work once the bear market ends so its a guide not rule at the moment.
Read 14 tweets
Camino de BCN en AVE, para asistir a la XV reunión anual del @CIBERER Me acompañan @afernandezl28 y Gema Garrido, como miembros de nuestra unidad @CIBER_ISCIII en el @CNB_CSIC @CSIC
Tras dos años pandémicos hoy nos veremos las caras (con mascarillas).
reunionanual.ciberer.es
Dos días en un hotel en Castelldefels para debatir y compartir nuestras investigaciones en #enfermedadesraras entre los diferentes grupos del @CIBERER con la presencia y diálogo con los representantes de las asociaciones de pacientes @FEDER_ONG @julianig
reunionanual.ciberer.es
Empezamos! Pablo Lapunzina, director científico del @CIBERER da la bienvenida a la primera mesa, con Isabel Campos representante de @FEDER_ONG Margarita Blázquez, gerente del @CIBER_ISCIII @SaludISCIII y Josep Torrent del SAB del Ciberer.
#RACIBERER2022
reunionanual.ciberer.es/programa
Read 6 tweets
Des chercheurs ont utilisé #CRISPR-Cas9 (une technologie utilisée pour créer des OGM) sur des hamsters pour réduire leur agressivité et modifier leur comportement.

C’est le contraire qui s’est passé : les hamsters sont devenus plus agressifs entre eux iflscience.com/plants-and-ani…
Ces résultats suggèrent que nous ne comprenons pas encore bien les effets des NGT (nouvelles techniques génomiques), même lorsque les effets attendus sont faibles, et que les chercheurs en génétique ont une compréhension faible et biaisée du cerveau et du comportement social.
Pourtant…

« Les chercheurs souhaitent maintenant utiliser cette technique pour lutter contre un large éventail de troubles psychiatriques. » iflscience.com/plants-and-ani…
Read 12 tweets
1/As promised & after reading $NTLA latest Q1 2022 financial report here is my analysis regarding @intelliatx latest corporate status. I have focused only on the main issues that I found to be the most interesting & relevant. #CRISPR #GeneEditing #BioTech #FinTwit #Genomics👇
2/NTLA-2001 is the most advanced In-Vivo #CRISPR/#Cas9 #GeneEditing program in $NTLA portfolio & is aimed to treat Transthyretin Amyloidosis - (ATTR). NTLA-2001 could potentially halt & reverse the disease in a “one time” treatment by knocking out the TTR gene with a single dose
3/In February $NTLA has presented an updated clinical data from its Phase 1 study of NTLA-2001 which included 15 patients with ATTR amyloidosis. IMO NTLA-2001 readout looks promising & here is a summary that I wrote shortly after @intelliatx’s presentation of NTLA-2001👇
Read 14 tweets
I am going to mention a few more #CRISPR companies that didn't meet my expectations to be in my portfolio. My next pick would actually be $VERV. I have some reservations on how much market share they could really capture in an extremely competitive space of Cholesterol.
$VERV has impressed me on the management front. There is noting I would bet on more then top notch management. A great CEO will make magic out of nothing. I think $VERV with base editing and a great CEO will be successful, I just don't know how successful.
Then comes $CRSP which use to be my first and only #CRISPR company. I completely changes on this company. They have a great SCD program what will be successful, but it ends there.
Read 5 tweets
My 3rd #CRISPR company has to be $CRBU, even though, I count them in my cell therapies space. They are not focused on any in-vivo editing at this time. They are completely focused on cell therapies around CAR-T and NK cells.
They are using the CAS9 and CAS12 enzymes along with their own unique guide called chRDNA. This uses a combination of RNA and DNA in the guide to increase binding affinity and better editing.
Their early data this week for CAR-T was impressive. It is still very early and clearly PD-1 will not be the "be all to end all". There are a lot of challenges to allogeneic CAR-T that need to be solved.
Read 6 tweets
My second pick for #CRISPR is $NTLA. I know they are on the losing side of the patent dispute. We still don't know what if any impact that will have. They do have a great management team that is building a great company.
They are using first generation CAS9 editing in-vivo in the liver and for ex-vivo for cell therapies. They are also developing their own base editing program. They are still ambitious and chasing innovation. That is what gets my vote.
They have best in class data in their early ATTR program in phase 1. Its got a long way to go, but could be a huge success if this data continues. They have about $1 billion in cash to get them through this bear market in biotech.
Read 4 tweets
$BEAM is my top pick in the #CRISPR space. They have base editing which solves many of the issues with the first generation editors. They still have the CAS12 if they need it to do any cell editing in their cell therapies.
All their tech licenses comes from the winning side of the patent dispute which makes them safe. They have a great management team. They also have $1.2 billion in cash which will last them a long time in this bear market.
They are just entering the clinic this year so it will all come down to the data. Their preclinical data seemed superior to that of $CRSP at the same stage of development.
Read 4 tweets
Hey #CRISPR friends! ✂️🧬 Joy Wang, myself and Jennifer @doudna_lab wrote a @NatureRevMicro review on the ‘Structural biology of CRISPR-Cas immunity and genome editing enzymes’. nature.com/articles/s4157… (1/4)
We capture a snapshot 📸 of this incredibly fast progressing field, focussing on the mechanism of CRISPR mediated DNA-capture (adaptation), -destruction (interference) and non-canonical -transposition. (2/4)
Joy, Jennifer and I hope that the manuscript will provide the ✂️-community and newcomers with an easy to access overview of the current state of the field. (3/4)
Read 4 tweets

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