Discover and read the best of Twitter Threads about #CRISPR
Most recents (24)
Gene scissors found to cause chaos in genome of tomatoes. And as the effects can be easily overlooked without exact genomic analyses, it's not unlikely that they also occurred in plants obtained from gene editing that were already deregulated in the US testbiotech.org/en/news/crispr… 
Chromothripsis-like effects occurred after the application of #CRISPR/Cas. In chromothripsis often several hundred genetic changes occur simultaneously in a 'catastrophic' event. Many sections of the genetic material can be swapped, twisted, recombined or even lost if this occurs
Molecular geneticist Dr Michael Antoniou: "Yet again we see a phenomenon already observed in a human cell context—major DNA damage from gene editing—now appearing in plants. Due to the inadequate analysis generally done on gene-edited plants, #GMO developers will often miss this"
Lange erwartet, nun plötzlich geleakt:
@annette_bruhns berichtet über den Vorschlag der EU-Kommission zur Reform des #Gentechnik-Rechts
table.media/berlin/analyse…
#GiveGenesAChance
Demnach würden NGT-Pflanzen (#CRISPR & Co.) in zwei Kategorien eingeteilt: kurzer 🧵1/9
@annette_bruhns berichtet über den Vorschlag der EU-Kommission zur Reform des #Gentechnik-Rechts
table.media/berlin/analyse…
#GiveGenesAChance
Demnach würden NGT-Pflanzen (#CRISPR & Co.) in zwei Kategorien eingeteilt: kurzer 🧵1/9
Kat 1: Was auch natürlich oder durch klassische Züchtung entstehen könnte, wären demnach von der #Gentechnik-Regulierung ausgenommen.
Kat 2: Für Pflanzen mit völlig neuen Eigenschaften, soll eine neue, angepasste Regulierung unterhalb der klassischen Gentechnik eingeführt werden
Kat 2: Für Pflanzen mit völlig neuen Eigenschaften, soll eine neue, angepasste Regulierung unterhalb der klassischen Gentechnik eingeführt werden
Es soll eine öffentliche Datenbank für Kat1-Pflanzen geben. So bleibt gewährleistet, dass zum Bio-Betriebe solche Sorten nicht verwenden. Da es die Branche so wollte, bleiben NGTs bei Bio komplett verboten. 3/9
Lots of moving parts in this thread but I hope people will find it useful in understanding the #4IR, or Fourth Industrial Revolution.
Israel is at the nexus. Everything from #Covid, to the war in #Ukraine, to infrastructural sabotage like the #Nordstream act as delivery systems.
Israel is at the nexus. Everything from #Covid, to the war in #Ukraine, to infrastructural sabotage like the #Nordstream act as delivery systems.
Most people understand there is more going on than what the #Zionist controlled news tells us. Stemming from long before Nixon and Chabad puppet Heinz Kissinger took the US off the gold standard, but that was a pivotal moment in transforming the US into an Israeli vassal state.
Just as 9/11 was a false flag pretext to usher in a surveillance state under the Patriot Act, Covid-19 and insidious forms of irregular warfare are being used to facilitate the planned demolition of the United States. And yes, it is indeed (((them))).
1/ 🧪🔬 Ever wondered if scientific research can be predicted? Our latest paper "What's next? Forecasting scientific research trends" tackles this question. Check out the details here 👉 arXiv:2305.04133v1 [thread] 🧵
2/ 🌍 The world of science is constantly evolving. But what if we could predict these trends, especially in life sciences? That's what our work aims to do. Imagine the possibilities!
3/ 📚 We've mined historical publications, research/review articles, and patents. The result? Models that can foresee scientific trends 5 years in advance. Time travel, anyone? #AI #DataScience #timeseries #trends
More tweetorial! Let’s dig into the “proximity bias” we found @RecursionPharma confounding #CRISPR screens, what it means, and where it comes from.
You can always read along in the preprint here: biorxiv.org/content/10.110…
You can always read along in the preprint here: biorxiv.org/content/10.110…
If you missed the first tweetorial in the series, click here to understand what the red-and-blue heatmaps here mean, and how we use them to map the functions of genes at a genome-wide level:
To recap: if you knock out each gene in the genome, plot all their pairwise similarities, and sort by genomic position, a curious pattern emerges in which #CRISPR knockouts look more similar to KOs on the same chromosome arm than to KOs on other arms.
Tweetorial time! We @RecursionPharma mapped consequences of #CRISPR screening of >17K human genes, found a systematic bias confounding all CRISPR screens, traced its molecular cause, and propose a debiasing algorithm.
“But Imran,” you say, “I’d rather read your thrilling 41-page manuscript than read tweet threads!”
I can’t blame you, it’s great! (I may be a biased source.) Here ya go: biorxiv.org/content/10.110…
I can’t blame you, it’s great! (I may be a biased source.) Here ya go: biorxiv.org/content/10.110…
In this first tweetorial, I’ll share some of the foundations of the similarity-based “maps” we build @RecursionPharma as background for what we found out about CRISPR by building a map over the whole genome.
Formidable publication sur l'utilisation de l'édition génomique #CRISPR pour obtenir des variétés de manioc à base teneur en Cyanure.
Limitant ainsi le risque de maladie de Konzo chez les populations Africaines les plus pauvres.
👇
frontiersin.org/articles/10.33…
1⃣⤵️
Limitant ainsi le risque de maladie de Konzo chez les populations Africaines les plus pauvres.
👇
frontiersin.org/articles/10.33…
1⃣⤵️
Environ un milliard de personnes dans le monde dépendent du manioc comme source de calories, dont environ 40 % d'Africains.
Or le manioc a naturellement des fortes concentrations de cyanure qui disparaissent à la cuisson.
2⃣⤵️
Or le manioc a naturellement des fortes concentrations de cyanure qui disparaissent à la cuisson.
2⃣⤵️
Chez les populations les plus pauvres, la préparation (cuisson) du manioc n'est pas toujours optimale.
Le cyanure étant un puissant neurotoxique, la consommation de manioc mal préparé peut provoquer la maladie de Konzo (ex em-consulte.com/article/218962…) entrainant des paralysies
3⃣⤵️
Le cyanure étant un puissant neurotoxique, la consommation de manioc mal préparé peut provoquer la maladie de Konzo (ex em-consulte.com/article/218962…) entrainant des paralysies
3⃣⤵️
Die Bio- & "Ohne Gentechnik"-Branche lobbyiert gerade massiv gegen eine wissenschaftsbasierte Reform des EU-#Gentechnik-Rechts. Man hat mit der Angst vor Gentechnik in den letzten Jahren gut verdient und sorgt sich nun um das Geschäftsmodell.
Worum geht es? 🧵/13
Worum geht es? 🧵/13
Die @EU_Commission erarbeitet derzeit einen Gesetzentwurf für eine Reform der sog. Freisetzungsrichtlinie, die auf europ. Ebene den Anbau von genetisch veränderten Pflanzen regelt. Die aktuelle Richtlinie ist im Kern 30 Jahre alt und nicht mehr geeignet, eine Reihe von
"Neuen genomischen Techniken" (NGTs), die in den letzten 20 (!) Jahren entstanden sind, adäquat zu regulieren. Dazu zählt auch die mittlerweile recht populäre "Genschere" #CRISPR. Während Gentechnik bei Pflanzen früher bisher vor allem genutzt wurde,
1/As promised & after reading $CRSP latest Q4 2022 #financial #report here is my impression regarding @CRISPRTX latest corporate status. I have focused only on the main issues that I found to be the most interesting & relevant. #CRISPR #BioTech #FinTwit #Genomics #GeneEditing 🧵
2/IMO the most significant corporate event in Q4 was $CRSP announcement that regulatory Exa-cel submissions of both #SickleCell & #BetaThalassemia validated in the #EU & #UK & that the @US_FDA BLA submission is on track by the end of Q1 ‘23 - possibly reaching the #markets in ‘23
3/Exa-cel/CTX001 - the key program in $CRSP portfolio, is an #autologous Ex-Vivo #CRISPR/#Cas9 #GeneEditing therapy aimed for patients suffering from #TDT or severe #SCD. The latest readout for both programs was phenomenal with 42/44 TDT & 31/31(!) demonstrated remarkable results
1/@GraphiteBio announced that after reviewing its business, it decided to discontinue any further development of nulabeglogene autogedtemcel (nula-cel) for #SickleCell disease, to explore a range of strategic alternatives & to reduce 50% in its workforce! #CRISPR #BioTech $GRPH
2/@GraphiteBio is also exploring the potential to continue nula-cel development externally. $GRPH intends to continue research activities associated with its early-stage non- genotoxic conditioning program, with the goal of advancing toward potential development candidates.👇
3/In addition, @GraphiteBio’s Board of Directors has approved a corporate restructuring that would reduce the company’s workforce by approximately 50%, among other actions to reduce cash burn while the company explores strategic alternatives. $GRPH
1/@LineageCell announced it entered an exclusive option & license agreement with $ERNA for the development of novel B2M deficient induced pluripotent #stem #cell #iPSC by using Eterna’s #GeneEditing technology thus developing therapies for #CNS diseases. #BioTech #CRISPR $LCTX
2/The new cell lines - to be developed by $ERNA, will support the creation of additional product candidates at $LCTX and specifically for the treatment of central #nervoussystem #CNS disorders & other #neurology targets via Eterna’s IP #CRISPR collaboration with @FactorBio
3/This agreement will also allow @LineageCell to enter the #CRISPR field and will provide $LCTX an option to obtain an exclusive license to utilize & sublicense $ERNA novel #GeneEditing cell lines for #preclinical, #clinical, & commercial purposes in the field of CNS diseases.
Our story on inserting short sequences with prime editing is now online @NatureBiotech.
nature.com/articles/s4158…
So much has changed from the preprint including the discovery that 3'-flap nucleases can inhibit the insertion of long inserts. Lots to unpack 🧵
nature.com/articles/s4158…
So much has changed from the preprint including the discovery that 3'-flap nucleases can inhibit the insertion of long inserts. Lots to unpack 🧵
A quick recap: We wanted to understand what factors influence how well a given sequence can be inserted with prime editing. To experimentally test this, we integrated 3,604 pegRNAs into 4 target sites in three cell lines and measured their insertion rates
Our cell lines differed in their mismatch repair status and we recapitulate the well-known effects of MMR in inhibiting the insertion of short sequences with our libraries.
sciencedirect.com/science/articl…
nature.com/articles/s4146…
sciencedirect.com/science/articl…
nature.com/articles/s4146…
🧵You can own the Future of Tomorrow Today.
I have heard many people try to call this crash in biotech the end of the #Biotechnology sector. A sector that has been around for decades. One of the most necessary sectors in the entire market.
I have heard many people try to call this crash in biotech the end of the #Biotechnology sector. A sector that has been around for decades. One of the most necessary sectors in the entire market.
1/ Here I am going to lay out for you the opposite case. I have been investing in tech and biotech for 27 years. I have seen so much technology that excites me about the future, and I see it today.
2/ The biggest question I always get in biotech investing is when will we run out of things to discover or great new technologies to invent. I my response is we will never run out until we cure every disease.
History is written by the winners, which is why #Luddite is a slur meaning "technophobe" and not a badge of honor meaning, "Person who goes beyond asking what technology does, to asking who it does it *for* and who it does it *to*."
locusmag.com/2022/01/cory-d… 1/
locusmag.com/2022/01/cory-d… 1/
If you'd like an essay-formatted version of this thread to read or share, here's a link to it on pluralistic.net, my surveillance-free, ad-free, tracker-free blog:
pluralistic.net/2023/02/07/ful… 2/
pluralistic.net/2023/02/07/ful… 2/
🛑🛑Tras la repercusión del tuit de abajo sobre un artículo resumen de los ensayos clínicos más prometedores de 2023 publicado en @Nature y un sondeo positivo sobre resumir o no su contenido, me dispongo a ello.
pd. Aportaré info adicional y actualizando en estos días
Vamos🧵
pd. Aportaré info adicional y actualizando en estos días
Vamos🧵
1º varias notas aclaratorias:
-En la pieza de @Nature le preguntan a 11 investigadores sobre ensayos, de su área de estudio, prometedores o esperados en 2023. Yo resumiré lo que dicen y aportaré información adicional
-En la pieza de @Nature le preguntan a 11 investigadores sobre ensayos, de su área de estudio, prometedores o esperados en 2023. Yo resumiré lo que dicen y aportaré información adicional
- Clinicaltrials.gov es una web donde se registran los ensayos clínicos, se informa de todo lo relacionado con su diseño, objetivos, resultados que se esperan, equipos implicados, fármacos que se van a usar y en qué enfermedad y otras cosas más
🧵My Top Science Themes for Biotech:
1. #AI/#ML and #Automation in Drug Discovery
2. #CRISPR
3. #Genomics
4. #SyntheticBiology
5. #iPSC
6. #TargetedTherapies
1. #AI/#ML and #Automation in Drug Discovery
2. #CRISPR
3. #Genomics
4. #SyntheticBiology
5. #iPSC
6. #TargetedTherapies
Excited to share our new manuscript on a novel #neuropeptide family that regulates #feeding across #evolution. Kudos to @Pat_TFrancisco for tackling this challenging project and coming up with the name @marmite . #homeostasis biorxiv.org/content/10.110… 1/22
Also kudos to all our collaborators @TastekinIbrahim @EzraGili @fern1_ana @BartDeplancke @albinojorgemai @alisson_gontijo which allowed us to expand the scope of our study and contributed critical insights. It takes a village 2/22
🧵The greatest sign I see in this #biotech market is the deafening quiet. All those rock stars of the 2021 biotech bubble are now gone. Their podcasts are gone, their Youtube channels are gone, and their $400 subscription services are gone.
1/ The only people left talking about biotech are few and far between assuming they can even endure the myriads of scoffers and skeptics. See, real investors are not fair weather investors.
2/ They aren't pounding the table at the peaks and disappearing back to their day jobs in the crash. The real investors are hear every day. They are the ones picking up the pieces left behind.
1/@GraphiteBio presented preclinical results supporting the use of a single-#cell #RNA #sequencing method to assess #gene correction outcomes in #patients treated with nulabeglogene autogedtemcel (nula-cel) - GPH-101. #BioTech #CRISPR #GeneEditing #Genomics $GRPH #ASH22
2/GPH101 (nula-cel) is a #CRISPR #Cas9 #GeneEditing autologous #stem #cell-based #therapy in clinical development aimed to treat #SickleCellDisease. GPH101 is designed to directly correct the underlying mutation, thereby decreasing HbS production & restoring HbA expression #ASH22
3 $GRPH gene correction platform involves editing hematopoietic #stem cells found in the #bonemarrow that develop into various types of #blood #cells. Since red blood cells lose their genomic DNA during maturation-tracking #GeneEditing in mature cells via sequencing is impossible
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.
🧵 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!
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/$BLUE)
Cost: ~2m
27’: 500 patients= 1b
28’: 2000 patients= 4b
29’: 1b (ESCAPE looming)
30’: zero
31’: zero
Approval: 1H 2027
Wave 1 Market: 25k US/14k EU
Market in 4 years: ~27k total ($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!
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!
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👇🧵
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…
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.
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.
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.
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.
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.
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.
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.
