Fyodor Urnov @UrnovFyodor
Gene editor + Professor of Molecular Therapeutics @berkeleyMCB + Director of @igisci @danahercorp Beacon for CRISPR Cures. innovativegenomics.org Berkeley, CA Joined November 2018-
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It is Saturday and my daughter and I decided to make the national breakfast dish * of #Egypt: ful mudammas. Question: are the jalapeños mission-critical? * I read this statement in this lovely book and apologize if the above is incorrect. themediterraneandish.com/cookbooks/
Cleese's work for Python ("this is an ex-parrot!") and Fawlty Towers ("please try to understand before one of us DIES") is sublime. What a deep shame to see him advocate for snake oil. Ubi nihil vales, ibi nihil velis, Mr Cleese. H/t @pknoepfler theguardian.com/science/2024/a…
Pfizer is _commercializing_ this therapy. It was _developed_ by Spark Therapeutics under the leadership of Dr Katherine High @PennMedicine. She also built Luxturna - the first approved AAV medicine. Dr High is a hero and inspiration to all working in our field. #WomenInSTEM
Pfizer is _commercializing_ this therapy. It was _developed_ by Spark Therapeutics under the leadership of Dr Katherine High @PennMedicine. She also built Luxturna - the first approved AAV medicine. Dr High is a hero and inspiration to all working in our field. #WomenInSTEM
Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) nej.md/3UvzPDT Editorial: Extending Gene Medicines to All in Need nej.md/3QjCLAS #Hematology
Dr Booth is leading the way for all genetic medicines in “rare disease” with her approach to have her hospital gain approval for the gene therapy she trialed. 👏🏻👏🏻👏🏻
Dr Booth is leading the way for all genetic medicines in “rare disease” with her approach to have her hospital gain approval for the gene therapy she trialed. 👏🏻👏🏻👏🏻
I admire @Regeneron for its commitment to gene-edited therapies. Earlier, bringing in 270 and Philip Gregory, a superstar, to run edited CAR-T programs - now, bringing in Team @mammothbiosci - quite a constellation of shining stars themselves! 1+2=7 synergy there.
I admire @Regeneron for its commitment to gene-edited therapies. Earlier, bringing in 270 and Philip Gregory, a superstar, to run edited CAR-T programs - now, bringing in Team @mammothbiosci - quite a constellation of shining stars themselves! 1+2=7 synergy there.
Super excited to see the exa-cel (Casgevy) #SickleCellDisease results out today in NEJM. These are the data that led to FDA approval Dec23 (during #ash2023). Terrific study steering committee. More data to come in younger pts. Now to work on access! @VertexPharma @CHOPCancerCntr
Super excited to see the exa-cel (Casgevy) #SickleCellDisease results out today in NEJM. These are the data that led to FDA approval Dec23 (during #ash2023). Terrific study steering committee. More data to come in younger pts. Now to work on access! @VertexPharma @CHOPCancerCntr
The first approved gene editing therapy - the clinical trial data! Bravo, clinical team, bravo Vertex CMC, thank you patients on the trial! $CoI: I am a paid advisor to Vertex on the exa-cel program.
The first approved gene editing therapy - the clinical trial data! Bravo, clinical team, bravo Vertex CMC, thank you patients on the trial! $CoI: I am a paid advisor to Vertex on the exa-cel program.
Friends! Romans (and those who wish to visit Rome)! Countrymen (and folks from every other country)! I come to bring CRISPR to life, not to praise it (it needs no praise)! Enough Shakespeare - this meeting is PHENOMENAL, come join us.
Friends! Romans (and those who wish to visit Rome)! Countrymen (and folks from every other country)! I come to bring CRISPR to life, not to praise it (it needs no praise)! Enough Shakespeare - this meeting is PHENOMENAL, come join us.
The ambitious idea of using CRISPR to cure genetic diseases before birth is one step closer to reality. trib.al/j7ub6yf
This is essential analysis for design of editing strategies to treat Mendelian disease. Grateful to the authors and the journal for providing it.
This is essential analysis for design of editing strategies to treat Mendelian disease. Grateful to the authors and the journal for providing it.
An essential step on CRISPR’s journey to becoming a platform for addressing the public health challenge of genetic disease. 👏🏻 @PennMedicine team. 🙏 @MeganMolteni for reporting on bona fide impactful advances in our field.
An essential step on CRISPR’s journey to becoming a platform for addressing the public health challenge of genetic disease. 👏🏻 @PennMedicine team. 🙏 @MeganMolteni for reporting on bona fide impactful advances in our field.
Two important firsts in genome editing 1. Fetal non-human primate base editing delivers to multiple organs statnews.com/2024/04/22/cri… by @MeganMolteni @statnews @WPeranteau @kiranmusunuru 2. #AI-generated gene editors success in human cells nytimes.com/2024/04/22/tec… @CadeMetz…
What actually happened: AI produced CRISPR-inspired genome editors that have modest on-target activity in human cancer cells. Impact on actual clinical editing in next 3 years: none. Long term - I sure hope so. I think X needs better AI to write these "Explore" blurbs. :-)
AI's latest: start with natural #CRISPR and engineer new editors. Clinical efforts have plenty of editors already (issues are cost + regulatory). Looking ahead I hope this bring us closer to "perfect editor for a patient's mutation in weeks." @CadeMetz nytimes.com/2024/04/22/tec…
SCOTUS will determine whether jailing folks experiencing homelessness is a good idea. The justices should reread this timeless classic by O'Henry, supreme master of narrating tragedy - and compassion for it - with a Chekhovian light touch. americanenglish.state.gov/files/ae/resou…
I've been teaching the immortal Sturtevant paper since 1997 (it's on Classical Papers in Genetics) and the first thing I say to my students is the point his grandson makes below. #yesyoucan - but, I say, you don't HAVE to. Just experience joy from the work. H/t @OmicsOmicsBlog
I've been teaching the immortal Sturtevant paper since 1997 (it's on Classical Papers in Genetics) and the first thing I say to my students is the point his grandson makes below. #yesyoucan - but, I say, you don't HAVE to. Just experience joy from the work. H/t @OmicsOmicsBlog
As my 8 yo daughter celebrates @CaitlinClark22 - she found a woman athlete who dominates her sport the way Messi and Steph dominate theirs - effortlessly and beautifully: the climber Janja Garnbret. Watch her flash a boulder problem and faint with awe.
David R. Liu @davidrliu
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Adam Feuerstein ✡�.. @adamfeuerstein
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Innovative Genomics I.. @igisci
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Niko McCarty 🧫 @NikoMcCarty
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ysxicudest1989 @ysxicudest28034
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TRUMP2024🇺🇸⛪�.. @ABNewwine
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Carlos Santamaría Ma.. @cjsanmar
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Chaitanya K. Joshi @chaitjo
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law astro @law_astro
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Petar (he/him) @pvtodorov
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Apex Tradez @apex_tradez
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Manjari Narayan | Sub.. @NeuroStats
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nedilize1982 @nedilize1986079
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|| AOL Tarun || @tarundsnaol
1K Followers 6K Following कर्मण्येवाधिकारस्ते मा फलेषु कदाचन । मा कर्मफलहेतुर्भुर्मा ते संगोऽस्त्वकर्मणि ॥ https://t.co/ibBIXJ8bEe
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Doudna Lab @doudna_lab
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Patrick Hsu @pdhsu
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Nature Biotechnology @NatureBiotech
302K Followers 2K Following Publishing the very best of biotech science and business. Part of @SpringerNature and @NaturePortfolio. On Facebook (natbiotechnol) & IG (nature.biotechnology)
Nicole M. Gaudelli @NicoleGaudelli
5K Followers 269 Following EIR @GVteam, dreamer, inventor of ABE. Interested in genomic medicine and next-generation application of gene editing tools. Leonardo’s & Isabella’s Mom.
Adam Feuerstein ✡�.. @adamfeuerstein
114K Followers 750 Following Reporter @statnews. The Harry Kane of biotech. Dog ❤️er. Polk Award winner. #COYS. Said one analyst: The likes of Adam Feuerstein attack viciously.
John Evans @john_evans3
7K Followers 335 Following CEO, Beam Therapeutics. We are working on creating precision genetic medicines using base editing. (tweets are my own)
Innovative Genomics I.. @igisci
16K Followers 938 Following Using genome engineering to solve humanity’s greatest problems in health, climate & sustainable agriculture. Partnership between @UCBerkeley and @UCSF. #CRISPR
Jacob Corn @jcornlab
5K Followers 508 Following Genome editing, functional genomics, and cells figuring out how to eat themselves without dying. Professor of Genome Biology at ETH Zürich.
Omar Abudayyeh @omarabudayyeh
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Ben Kleinstiver @BKleinstiver
4K Followers 669 Following Associate Professor @CGM_MGH at @MGH_RI & @HarvardMed Genome editing / Protein eng. / Molecular medicine 🇨🇦🧬 Kayden-Lambert MGH Research Scholar '23-28
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Cal Basketball @CalWBBall
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Vertex Pharmaceutical.. @VertexPharma
72K Followers 89 Following The official channel for Vertex Pharmaceuticals. https://t.co/RRKBue0r0B
Arena BioWorks @arenabioworks
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Ryan Cross the Scienc.. @RLCscienceboss
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Ryan Leenay @RTLeenay
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Margaret Sullivan @Sulliview
138K Followers 4K Following Columnizing @GuardianUS. Thinking/teaching @columbiajourn … Knicks fan and #billsmafia. … Pro-democracy
Deirdre Kelly @Deirdre_Kelly
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Rupsa Chakraborty @RupsaChak
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Chris Nee (She/Her)�.. @chrisdocnee
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I. Emilie Steinmark @IEmSteinmark
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Jeff Vierstra @jeffvierstra
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Ophir Shalem @ophirshalem
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Jessica Chastain @jes_chastain
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Bree Iskandar @breezyscience
271 Followers 256 Following PhD @UNCPharmacy @BowersRangers | ‘23 @AAASMassMedia Fellow @statnews | Previously Content Editor @UNC_SWAC
Stephen Kelman @stephen_kelman
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Haydar Frangoul @hfrangoul
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Jonathan Saltzman @saltzmanglobe
430 Followers 19 Following Report at The Boston Globe; covers biotech; former Spotlight Team member; Bob Dylan fan; swimmer
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879 Followers 2K Following #science editor @srf, #Dozentin @MAZ. RT/T zu allem, was ich halbwegs einschätzen kann & relevant finde; #faktenzählen, https://t.co/oeGlDeVXZL
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Eric Vallabh Minikel @cureffi
5K Followers 893 Following My mission in life is to prevent prion disease.
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307 Followers 272 Following 1st-year PhD student @berkeleyMCB | @dartmouth ‘23
Caroline Seydel @CarolineSeydel
760 Followers 1K Following Science writer covering genetics, cancer, biotech. "Truth is a matter of the imagination. The soundest fact may fail or prevail in the style of its telling."
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6K Followers 2K Following CEO Stealth HealthTech Co. Formerly: CEO at @GenomicsEngland; COO at @quantumblack; Partner @mckinsey; UK Foreign Service; @bbc. Views are personal.
@realSarahPolley (she.. @realsarahpolley
65K Followers 1K Following My Mom was one of the 30 Helens on Kids In The Hall. She/Her
Natasha Loder 🐋 @natashaloder
22K Followers 4K Following Stories that matter. Health Editor, The Economist 💉 Writer ✍️ Broadcaster🎤 Cartoonist 💬 Views own.
Gerald Schwank @schwanklab
948 Followers 160 Following Associate Professor @UZH_en. We develop genome editing approaches for personalised treatment of rare diseases and cancer.
Stephanie Nolen @snolen
39K Followers 4K Following Global health reporter @nytimes. Former correspondent in S. Asia, Africa, Latin America & MidEast. Author, 28 Stories of AIDS in Africa.
Gunnar Esiason @G17Esiason
21K Followers 560 Following Saved by science 🧬 Patient advocate 🫁 Dad 👶🏻🐶 Biotech | Healthtech | Hockey coach 🦠🔬🏒 @BostonCollege @TuckSchool @Dartmouth 🦅🌲
Nuño Domínguez @Nunodomin
11K Followers 2K Following Periodista en Materia (sección de ciencia de El País). Estoy en [email protected]
Erin Doherty @Ee_Doherty
899 Followers 988 Following Postdoc @Doudna_Lab || @UCDChem Beal lab alum RNA Chemical Biology || she/her CIRM Postdoctoral Fellow || Former NIH F31 & NIH T32
Metagenomi @metagenomi
716 Followers 76 Following Using natural microbial communities to discover and develop next-generation therapeutics.
Mammoth Biosciences @mammothbiosci
6K Followers 2K Following Improving lives by reading and writing the code of life.
@UrnovFyodor @pknoepfler The charitable interpretation is that this is performance art and part of an elaborate skit… “What have double blind randomized control trials ever done for us?” 😬
An international consortium led by Dr. Stephan Grupp published final results of a key clinical trial on gene therapy CASGEVY for sickle cell disease treatment. The findings, published in @NEJM, led to FDA approval of CASGEVY™ in December 2023. More: ms.spr.ly/6011YJEX3.
Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) nej.md/3UvzPDT Editorial: Extending Gene Medicines to All in Need nej.md/3QjCLAS #Hematology
Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cells, 29 were free from vaso-occlusive crises for at least 12 consecutive months. Read the full CLIMB SCD-121 study results: nej.md/3UvzPDT
It’s stunning what Berkeley is able to achieve at the scale that it achieves it.
Our collaboration brings together pioneers in next-gen CRISPR gene editing and antibody-retargeted AAV technologies, with a shared commitment of translating high science into ground-breaking medicines for patients. Read our press release to learn more: mammoth.bio/regeneron-and-…
We look forward to working with @Regeneron to enable all-in-one AAV delivery and unlock the true potential of in vivo gene editing. Read our press release to learn more: mammoth.bio/regeneron-and-… @doudna_lab @UCBerkeley
Super excited to see the exa-cel (Casgevy) #SickleCellDisease results out today in NEJM. These are the data that led to FDA approval Dec23 (during #ash2023). Terrific study steering committee. More data to come in younger pts. Now to work on access! @VertexPharma @CHOPCancerCntr
Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cells, 29 were free from vaso-occlusive crises for at least 12 consecutive months. Read the full CLIMB SCD-121 study results: nej.md/3UvzPDT
Also great to see the thalassemia data for exa-cel.
Of 35 patients with transfusion-dependent β-thalassemia treated with gene-edited autologous hematopoietic stem and progenitor cells, 32 had hemoglobin levels maintained without red-cell transfusions for at least 12 consecutive months. Full study results: nej.md/3xYXrI1
The ambitious idea of using CRISPR to cure genetic diseases before birth is one step closer to reality. trib.al/j7ub6yf
Two important firsts in genome editing 1. Fetal non-human primate base editing delivers to multiple organs statnews.com/2024/04/22/cri… by @MeganMolteni @statnews @WPeranteau @kiranmusunuru 2. #AI-generated gene editors success in human cells nytimes.com/2024/04/22/tec… @CadeMetz…
The ambitious idea of using CRISPR to cure genetic diseases in the womb is one step closer to reality, new research in fetal monkeys shows. statnews.com/2024/04/22/cri… via @statnews
@UrnovFyodor @matthewherper @ADeAngelis_bio I agree with your vision completely — we should be sequencing everyone and use naturally occurring genetic modifiers to illuminate the therapeutic pathway. My comment was more that a company with a business model in ancestry is going to have a very hard a pivoting to this path.
@UrnovFyodor @ADeAngelis_bio I mean, I don't think buying a telehealth practice was an example of a canalized worldview! But I also think that at this point they are likely to be open to ideas. If I were advising them I'd tell them to find a way to fund the development or sale of their existing drug…
@matthewherper @UrnovFyodor @ADeAngelis_bio They had partnership revenue. A huge exclusive deal with GSK. They spent a bunch of resources trying to develop targets in house and failed. To me the 23andMe story is a that of failed leadership and bad decisions.
@matthewherper @UrnovFyodor @ADeAngelis_bio Also, they can’t deal with rare variation, precisely where the most informative genetics occurs.
@jeffvierstra @UrnovFyodor @ADeAngelis_bio Not sure I’d even see the companies as doing the same thing. Decode had a head start on 23andMe and seems to be a key part of Amgen’s discovery efforts…
@matthewherper @UrnovFyodor @ADeAngelis_bio In any case I chimed in because my family has a deep multigenerational fatal monogenetic disorder with examples gene carriers but not afflicted (though should be, dominant variant). I have sequenced my whole family pedigree and not easy problem and not a scalable one.
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