Discover and read the best of Twitter Threads about #crispr

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The most impressive science in biotech right now is:

1. #iPSC
2. #AI/#ML in biotech
3. #SynBio
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."… 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
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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:… 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
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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.
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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).
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
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.
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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…
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.

« Les chercheurs souhaitent maintenant utiliser cette technique pour lutter contre un large éventail de troubles psychiatriques. »…
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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.
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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’.… (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)
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1/ I have been spending a lot of time with the market selling off trying to share and teach about investing and how to build a portfolio to diversify across assets and risks.
2/ I think its good to take time when the market is down to focus on improving our game for the next time around. Since I am a biotech focused investor, I would like to share with you the trends within the biotech space I think could propel the next decade of innovation.
3/ If you know me already, these won't be very new to you. My first big concept is using tech in biotech with developing #AI, #ML and #Automation to drive down the costs and improve the success of clinical development.
Read 18 tweets
I went back and looked at last year. After the JPM conference in January, I probably used the word #bubble at least 200 times talking about the $XBI, #crypto and #EV. Today, I think biotech is at fire sales prices with an opportunity down 60%. The others still have a ways to go.
From the peak:
$XBI is down over 62%
$BTC is down over 57%
$TSLA is down almost 40%

I think biotech offers the best opportunity as #crypto has no real world applications. $TSLA is a very strong and profitable company, but still grossly overvalue by a good 20% or more.
Most biotechs are trading below cash levels. I haven't see valuations this cheap since the bottom of the 202 pandemic and the lows of the 2015 Hillary crash.
Read 5 tweets
Sarah Ferguson and Tony Robbins > Pegasus Group Holdings

#Epstein #CRISPR Image
Jay Bloom, Chairmen of Pegasus Group Holdings, LLC, is the original founder of the Las Vegas Mob Experience
2020:"Curren Price Jr., center, with LAFC owners, from left, Peter Guber, Magic Johnson, Will Ferrell and Tony Robbins at the groundbreaking for the soccer franchise’s Banc of California stadium

Managing partner Henry Nguyen is vice-chairman and owner"… Image
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2/ #AlphaFold can predict the folded protein shape, but not the complicated folding process itself. Often cells use protein complexes called chaperonins to help other proteins to fold. Here we purified a chaperonin called TRiC from cultured human cells.
3/ Purification was possible only by using #CRISPR genetic scissors. We introduced a purification handle in one of the 8 TRiC subunits. This allowed purification of the endogenous chaperonin as opposed to an engineered version of it. Quite a TRiCK! #samplerevolution
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Les bovins génétiquement modifiés autorisés pour la consommation humaine aux États-Unis.

Le bétail - modifié via #CRISPR pour mieux résister aux conditions d’élevage intensif - est le 3ème animal OGM autorisé aux US après le saumon et les porcs.…
Ce sont ces « nouveaux OGM » (des êtres vivants génétiquement modifiés par les ciseaux génétiques #CRISPR ou similaire) que la Commission européenne est en train de déréglementer en UE…
Avec le soutien des macronistes…
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1/ I am planning some threads on cell therapies. I want to start with a history of cell therapies with Autologous CAR-T with companies like Kite, Juno and $BLUE. Get into the limitations.
2/ Then cover the transition to Allogeneic with companies like $CRSP, $ALLO, $DTIL, $ATRA, $TCRR, $CRBU. Cover the limitations and benefits over Autologous.
3/ Then I want to cover TCR therapies and how they address the unique issue of intra vs extra cellular antigens with $ADAP and $IMTX.
Read 5 tweets
Taking a Look at #CRISPR gene editing:

This has been a space that really needed new technology after decades of older technology failing to work. The need for precise edits without off target effects still exists.
1/ The top pick here is clearly $BEAM with their base editing technology. They are using a modified CAS9 that only has 1 cutting domain. This allows them to use a deaminase to modify a single base and cut the opposite strand for repair.
2/ This technology has limitations as it can only do ABE and CBE modifications. It can't do insertions. There is still a ton of potential across hundreds of genetic disorders for this technology. They could do billions in revenues if the science works.
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Thread : La #guerreRussieUkraine nous montre que, plus que jamais, il faut rejeter fermement les idéologues #antiscience tels que #Melenchon2022 et #Jadot2022
En matière d’agriculture, ces deux #ecologiste.s (tenants de l’#idéologie #écologiste à ne pas confondre avec l’#écologie qui est une #science), dénoncent ce qu’ils appellent le « #productivisme »
Autrement dit, ils dénoncent le fait que la France soit l’un des rares (5 à 10 selon la méthode d’estimation) pays au monde à avoir son indépendance alimentaire. Car, scoop, si on produit beaucoup de nourriture, c’est parce qu’il y a beaucoup de monde à nourrir.
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While #science thought us that we can even cut and paste #genes with #CRISPR technology…even with that we only influence 3% to 5% of #chronicillness. The rest depends on #how you #live your #life!!! So how do we #SelfRegulate our #body?
1. Good #sleep is more important than most think! What happens during sleep? The fluctuation of consciousness…that we call the waking state. But in many wisdom traditions of the world, the waking state is merely a lucid dream that consciousness is having…
Read 41 tweets
Daily Bookmarks to GAVNet 03/18/2022…
Protein tweak makes CRISPR gene editing 4,000 times less error-prone…
#CRISPR, #GeneEditing, #Cas9Protein, #ErrorCorrection
Read 13 tweets
Really chuffed to share our latest work from Sanjana Lab out in @nature today (…).

We tested >12,000 genes to find positive regulators of T cell proliferation to be used for next-gen #immunotherapies.

A thread...
(2/28) T cell therapies have shown the potential to cure patients from #cancer – but even in B cell cancers, relapses outnumber cures… One of the problems is limited T cell persistence. #celltherapy #CARTcell
(3/28) We wanted to find new genes (including genes never expressed in T cells) that could improve T cell persistence. Now, the question was: should we use #crispr activation or directly deliver target genes on a #lentivirus?
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Very pleased to share our ✨New Paper✨ about an epigenetic conflict between a restriction–modification system and a methylation-dependent nuclease.

Want to know how that pans out? Continue reading here 🧵👇🏻 or head over to @NAR_Open for the full story:…
It all began when we started investigating the anti-#CRISPR repressor Aca2 from Pectobacterium #phage ZF40. Back then, we stumbled upon an RM system in Pecto that inhibited transformation and conjugation and completely blocked phage infection. 🧬🚫 [2/n]
Because this strain could only take up DNA with RM-compatible methylation, we knocked out the restriction endonuclease of the RM system, enabling us to pursue our anti-CRISPR regulation project:…

And that could have been the end of it... [3/n]
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