Discover and read the best of Twitter Threads about #GeneEditing
Most recents (24)
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/@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.
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
The Great Ormond Street Hospital for Children in London has preformed the world's first base editing treatment for a 13-year-old patient's once "incurable" T-cell leukemia to rid her body of cancer. #cancer #geneediting #baseediting #cancertreatment #Leukemia #CGTNAmerica
Invented six years ago, base editing uses the four bases of the building blocks of the human genetic code: adenine (A), cytosine (C), guanine (G) and thymine (T). #cancer #geneediting #baseediting #newcancertreatment #cancertreatment #Leukemia #CGTNAmerica
Scientists alter the structure of a base, converting it to another and changing the genetic instructions. The hospital used this technology to edit and create a new type of T-cell that finds and destroys T-cells, including leukemic T-cells. #geneediting #baseediting #Leukemia
🧵 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!
$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
#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.
It is one of the first few in-vivo #GeneTherapy being tested. Congrats to the $NTLA and $REGN teams.
"You have the feeling, when you ride, that this animal understands you and you understand this animal. You also have this feeling of mastering this big animal — it makes you feel powerful.” -- Ludovic Orlando @LudovicLorlando greeneracresvaluenetwork.wordpress.com/2022/05/26/dai…
Genetic roots of three mitochondrial diseases identified via new approach
phys.org/news/2022-05-g…
#MitochondrialProteins, #CRISPRCas9, #GeneEditing, #MitochondrialFunction
phys.org/news/2022-05-g…
#MitochondrialProteins, #CRISPRCas9, #GeneEditing, #MitochondrialFunction
In Partial, Grudging Defense Of The Hearing Voices Movement
astralcodexten.substack.com/p/in-partial-g…
#MentalHealth, #NonPharmaceuticalTreatment, #HearingVoicesNetwork, #PeerSupport
astralcodexten.substack.com/p/in-partial-g…
#MentalHealth, #NonPharmaceuticalTreatment, #HearingVoicesNetwork, #PeerSupport
"God gave you a voice. Use it. If you can see the worth in me, then you can see the worth in everyone you meet. We are all called to serve, as an everyday act of humility, as a habit of mind. To see the worth in every person we serve."-- Elizabeth Bonker greeneracresvaluenetwork.wordpress.com/2022/05/24/dai…
Gene Editing Can Change The Social Behavior of Animals in Unexpected Ways
sciencealert.com/crispr-gene-ed…
#GeneEditing, #VasopressinHormone, #AnimalBehavior, #UnexpectedOutcomes
sciencealert.com/crispr-gene-ed…
#GeneEditing, #VasopressinHormone, #AnimalBehavior, #UnexpectedOutcomes
Deep Visual Proteomics defines single-cell identity and heterogeneity | Nature Biotechnology
nature.com/articles/s4158…
#DeepVisualProteomics, #CellularPhenotypes, #ImageAnalysis, #ArtificialIntelligence
nature.com/articles/s4158…
#DeepVisualProteomics, #CellularPhenotypes, #ImageAnalysis, #ArtificialIntelligence
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👇
Daily Bookmarks to GAVNet 03/18/2022 greeneracresvaluenetwork.wordpress.com/2022/03/18/dai…
Protein tweak makes CRISPR gene editing 4,000 times less error-prone
newatlas.com/science/crispr…
#CRISPR, #GeneEditing, #Cas9Protein, #ErrorCorrection
newatlas.com/science/crispr…
#CRISPR, #GeneEditing, #Cas9Protein, #ErrorCorrection
You're doing it wrong: You need to compare apples to oranges
techxplore.com/news/2022-03-y…
#CognitiveTraining, #ComparativeStudy, #VirtualReality, #SociallyAssistiveRobot
techxplore.com/news/2022-03-y…
#CognitiveTraining, #ComparativeStudy, #VirtualReality, #SociallyAssistiveRobot
Who's ready for another jab in March? Roll up your sleeve (again) for #Pfizer 1.1:
CEO Albert Bourla, Jan 10, 2022: "And we know that the 3, the 2-dose[s] of the #vaccine, offer very limited protection, if any."
#4IR #BiotechEconomy #GeneEditing #mRNA #Crispr
Thread 👇
CEO Albert Bourla, Jan 10, 2022: "And we know that the 3, the 2-dose[s] of the #vaccine, offer very limited protection, if any."
#4IR #BiotechEconomy #GeneEditing #mRNA #Crispr
Thread 👇
Daily Bookmarks to GAVNet 01/02/2022 greeneracresvaluenetwork.wordpress.com/2022/01/02/dai…
Introduction to Complexity and Applied Complexity
appliedcomplexity.io/education
#JoeNorman, #AppliedComplexityScience, #OnlineClassEnrollment, #CourseSyllabus
appliedcomplexity.io/education
#JoeNorman, #AppliedComplexityScience, #OnlineClassEnrollment, #CourseSyllabus
1/10War on virus is about cognitive dissonance. Those of us who stood by silent as minority communities were gentrified through the war on drugs, or profited by it, do not imagine being the targets of an epic urban renewal through lockdown measures and vaccine mandates.
2/10War on virus is about destabilization. The bulk of wage workers as well as business owners who cater to the establishment must collide with those who have realized that their livelihoods are being destroyed or their businesses are being swallowed by large corporate entities.
3/10Communities are divided. Family members are divided over fear and uncertainty. We are being primed for further colonization of humanity and nature.
2/12/21: Newsweek: "Miraculous #mRNA #Vaccines are only the Beginning"
#mRNA vaccines offer "an early look at how the miraculous tools of the #genetics #revolution will transform our #healthcare & our world over the coming years."
#4IR #biotech
newsweek.com/miraculous-mrn…
#mRNA vaccines offer "an early look at how the miraculous tools of the #genetics #revolution will transform our #healthcare & our world over the coming years."
#4IR #biotech
newsweek.com/miraculous-mrn…
"...our Promethean technologies, our moment of triumph could set us on a path to disaster. Of all the species that have ever lived, our single group of hominins now has the capacity to remake all of #biology."
#GeneEditing #4IR #Biotech
#GeneEditing #4IR #Biotech
The new #vaccines are perfect early examples of this "godlike #technology." ... This approach will soon create a whole new platform for fighting cancers & other diseases, as well as for providing enhancements even more profound than #vaccination."
Human #Experiment w/ #mRNA
Human #Experiment w/ #mRNA
2/12/21: Newsweek: "Miraculous #mRNA #Vaccines are only the Beginning"
#mRNA vaccines offer "an early look at how the miraculous tools of the #genetics #revolution will transform our #healthcare & our world over the coming years."
#4IR #biotech
newsweek.com/miraculous-mrn…
#mRNA vaccines offer "an early look at how the miraculous tools of the #genetics #revolution will transform our #healthcare & our world over the coming years."
#4IR #biotech
newsweek.com/miraculous-mrn…
"...our Promethean technologies, our moment of triumph could set us on a path to disaster. Of all the species that have ever lived, our single group of hominins now has the capacity to remake all of #biology."
#GeneEditing #4IR #Biotech
#GeneEditing #4IR #Biotech
The new #vaccines are perfect early examples of this "godlike #technology." ... This approach will soon create a whole new platform for fighting cancers & other diseases, as well as for providing enhancements even more profound than #vaccination."
Human #Experiment w/ #mRNA
Human #Experiment w/ #mRNA
Daily Bookmarks to GAVNet 05/17/2021 greeneracresvaluenetwork.wordpress.com/2021/05/17/dai…
Battlestar Galactica Lessons from Ransomware to the Pandemic
theinsight.org/p/battlestar-g…
#ransomware #PandemicResponse #LessonsLearned
theinsight.org/p/battlestar-g…
#ransomware #PandemicResponse #LessonsLearned
Female northern elephant seals spend 18 hours a day foraging in deep sea
phys.org/news/2021-05-f…
#FemaleSeals #foraging #BehaviorTracking
phys.org/news/2021-05-f…
#FemaleSeals #foraging #BehaviorTracking
Daily Bookmarks to GAVNet 05/15/2021 greeneracresvaluenetwork.wordpress.com/2021/05/15/dai…
Non-hospitalized COVID-19 patients: Low-risk of serious long-term effects, but more visits to doctor
medicalxpress.com/news/2021-05-n…
#COVID19 #LongHaulers #risk
medicalxpress.com/news/2021-05-n…
#COVID19 #LongHaulers #risk
New Concept Weapons #NCW
Directed Energy #Weapons #DEW
Force multiplier
New sword
Invisible card
People’s Liberation Army #PLA
PLA Strategic Support Force #PLASSF
new #technology testing
Disruptive #technologies
#Space Engineering University
#Chinese academic and military
#China
Directed Energy #Weapons #DEW
Force multiplier
New sword
Invisible card
People’s Liberation Army #PLA
PLA Strategic Support Force #PLASSF
new #technology testing
Disruptive #technologies
#Space Engineering University
#Chinese academic and military
#China
New Sword
Kinetic energy weapons #KEW
#DEW:
#Laser
#microwave
#electromagnetic #pulse
particle beam
New #atomic weapons:
#neutron bomb
#antimatter
#Sonic weapons:
#noise
#infrasound
jamestown.org/program/new-co…
Kinetic energy weapons #KEW
#DEW:
#Laser
#microwave
#electromagnetic #pulse
particle beam
New #atomic weapons:
#neutron bomb
#antimatter
#Sonic weapons:
#noise
#infrasound
jamestown.org/program/new-co…
This is the third edition of my tweetstorms on #GeneTherapy #CellTherapy #GeneEditing. This one compares $SGMO to $CRSP and attempts to look at #GenomeMedicine from the perspectives laid out by C.Woods at ARK This is part one of the series. Pt 2 tomorrow AM 1/20
The charts of both are attached and useful context. $CRSP went public in 2016. The weekly chart is attached and notes the pct ownership of $ARKG $ARKK since CY18. Prior the stock was stagnant below the support/resistance levels show. 2/20
I'm including two charts for $SGMO. Wkly and Mthly. The weekly shows they were pulled along with the #geneediting group The monthly shows this rally/bust cycle is not unique. Compare the SGMO 2017-2018 to CRSP. Pretty similar. More later. 3/20
🌄Good evening everyone!
Welcome to In Conversation with 𝐃𝐫 𝐑 𝐂𝐡𝐢𝐝𝐚𝐦𝐛𝐚𝐫𝐚𝐦, Former Principal Scientific Adviser to Govt. of India, Chairman AEC & Secretary @DAEIndia👥
🗣️Please post your recommendations, comments with #ChatwithRChidambaram
Welcome to In Conversation with 𝐃𝐫 𝐑 𝐂𝐡𝐢𝐝𝐚𝐦𝐛𝐚𝐫𝐚𝐦, Former Principal Scientific Adviser to Govt. of India, Chairman AEC & Secretary @DAEIndia👥
🗣️Please post your recommendations, comments with #ChatwithRChidambaram
Good evening everyone! 🌇🕕
Welcome to In Conversation with Dr. Renu Swarup @RenuSwarup @DBTIndia
Please post your recommendations, comments with #ChatwithRenuSwarup.
Welcome to In Conversation with Dr. Renu Swarup @RenuSwarup @DBTIndia
Please post your recommendations, comments with #ChatwithRenuSwarup.
What's up with $SGMO valuation? This thread will try to show some history and the setup for what I still hope to be an outsized move. First worth noting that news will always trump technicals. First here is a month chart I've been using for the company 1/
This being a 10 year chart, it doesn't show how long $SGMO has been around but it does show virtually no trading volume and a 3 yr ceiling on the price as they were in early development stage while trying to get ill-chosen indications into pre/clinic 2/
The first wave $SGMO rally started in 2013 when the ceiling was broken. The company was talking up #GeneEditing #GeneRegulation #HIVcure and #diabetes None were ready but the volume picked up and the first big rally ensued going from around $9 to $20 3/
