May 2024 PMID 38661449

Exagamglogene Autotemcel for Severe Sickle Cell Disease.

Haydar Frangoul, Franco Locatelli, Akshay Sharma, Monica Bhatia, Markus Mapara, Lyndsay Molinari, Donna Wall, Robert I Liem, Paul Telfer, Ami J Shah, Marina Cavazzana, Selim Corbacioglu, Damiano Rondelli, Roland Meisel, Laurence Dedeken

Mentions

177
Peak: 87 in Apr 2024
across 6 sources since Jun 2023

Extended reach

2,668,401
potential audience

Total likes

276

Video views

947

Mentions per month

Jun 2023 – Aug 2025

Where it's discussed

  • X / Twitter140 posts
  • News24 articles
  • Blogs6 articles
  • YouTube3 videos
  • Facebook3 posts
  • Bluesky1 posts

AI synopsis

What people are saying

The discussions emphasize the groundbreaking results of the research, citing that 97% of patients were free from vaso-occlusive crises for at least 12 months, which many refer to as ‘game changing science.’ Phrases like 'major breakthrough’ and comments about future potentials of gene therapy underscore the optimism and urgency surrounding this treatment's impact. Overall, the enthusiastic tone and use of specific results contribute greatly to the positive sentiment expressed across the board.

AgreementStrong agreement

Almost all discussions express strong support for the findings and approach presented in the publication.

InterestHigh interest

The discussions indicate a very high level of curiosity and engagement with the topic of gene therapy for sickle cell disease.

EngagementHigh engagement

Many posts dive into specific aspects of the research, including methodologies and implications, reflecting deep engagement with the material.

ImpactHigh impact

The consensus suggests that this research is perceived as having a significant and transformative impact on the treatment of sickle cell disease.

Abstract in brief

  • Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy utilizing CRISPR-Cas9 gene editing to increase fetal hemoglobin production in patients with sickle cell disease.
  • A phase 3 study involved 44 patients aged 12 to 35 with a history of severe vaso-occlusive crises; patients received edited stem cells after myeloablative conditioning.
  • Results showed 97% of patients were free from vaso-occlusive crises and 100% avoided hospitalization for over 12 months, with a safety profile similar to standard treatments.

Top posts

Most liked on X and Bluesky
Brianna
@_alltimebrianna · X

Seems like a great time to share the publication around this therapy. If you’re curious about the treatment, my work at St. Jude, or what gene therapy looks like, here it is: https://t.co/seRM9Uzz5L Also would like to direct your attention to the 97%!!! of patients with no pain. https://t.co/zNDIFi33he

Apr 26, 2024 · 78 likes · 7 shares · 6,375 followers View post →
NEJM
@NEJM · X

Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Extending Gene Medicines to All in Need https://t.co/JbDFObRWR0 #Hematology https://t.co/4FVV1rw79w

Apr 25, 2024 · 53 likes · 16 shares · 935,685 followers View post →
Muzaffar Qazilbash
@Transplant_Doc · X

Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso-occlusive crises at 12 month #bmtsm @BMT_updates @GruppSteve @AkshaySharmaMD https://t.co/7MBKoRrSCP

May 9, 2024 · 32 likes · 11 shares · 7,399 followers View post →
Chimeric Antigen Receptor T-Cell (CAR-T) & Gene Rx
@CARTTherapy · X

Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/X1oFd3e8Be

Apr 25, 2024 · 9 likes · 5 shares · 4,456 followers View post →
NEJM
@NEJM · X

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia https://t.co/LiTmJPo0pS Exagamglogene Autotemcel for Severe Sickle Cell Disease https://t.co/l5varni2by Editorial: Extending Gene Medicines to All in Need https://t.co/JbDFObRp1s https://t.co/kTmzjMyBjO

May 8, 2024 · 8 likes · 3 shares · 935,685 followers View post →
Salveo Therapeutics
@MySickleCare · X

Exagamglogene Autotemcel for Severe Sickle Cell Disease https://t.co/SxsdazA9qn? @akshatdoctor @TheSickleDoc @acweyand @DrPowerHays @DrAmandaBrandow @subarnac @drzsicklecell @nfayanders @seethal_jacobmd @sicklecelldoc @DrTitiFasipe @JuliaXuMD @DrScottHadland

Apr 24, 2024 · 8 likes · 3 shares · 297 followers View post →

All mentions

Dr. Gennadi Glinsky, MD, Ph.D.
@gglinskii · X

Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/y4YVpHtz7H

Aug 19, 2025 · 2,064 followers View post →
Toby
@JinyuanWang7 · X

@john_evans3 Hey John. Is this study different from "Exagamglogene Autotemcel for Severe Sickle Cell Disease" one?

Feb 22, 2025 · 419 followers View post →
Jauro XVII
@Danjauro_ · X

RT @EL_KUT: @Danjauro_ @muasaidu Very good data. Main issues are TRM from MAC and possible off target. Otherwise very good science https://…

Feb 3, 2025 · 1 share · 25,215 followers View post →
#FreeDadiyata
@EL_KUT · X

@Danjauro_ @muasaidu Very good data. Main issues are TRM from MAC and possible off target. Otherwise very good science https://t.co/Yy4QvAAhJo

Feb 2, 2025 · 1 like · 1 share · 3,357 followers View post →
Sanchit Turaga
@srturaga · X

Interesting phase 3 trials too: https://t.co/0DyUf07Yor https://t.co/kK6utbYIlf BBC article: https://t.co/CpJLyGRYRH

Jan 31, 2025 · 1 like · 2,607 followers View post →
This is me being reasonable
@dontwannagetfat · X

Gene therapy เช่น ยาที่ FDA approve ใน severe Sickle cell (Exagamglogene Autotemcel) ใช้ CRISPR/Cas9 ในการ guide ว่าจะปรับ DNA ตรงไหน ซึ่งนางไปปรับยีน BCL11A ตรงตำแหน่ง enhancer ให้สร้าง HbF มากขึ้น ฟังดูดี แต่เท่าที่อ่านงานวิจัย adverse events สยองใช้ได้ https://t.co/ltxZBBOwyZ https://t.co/nxBnCHclyr

Jan 2, 2025 · 4,912 followers View post →
Julio Rodríguez, PhD | Genética Clínica
@bitacorabeagle · X

10. Avances en terapia génica -Trastornos sanguíneos (anemia de células falciformes). -Restaurar la visión (amaurosis congénita de Leber). -Restaurar la audición (sordera autosómica recesiva tipo 9) https://t.co/T5yg9THPRT https://t.co/n99kCVqAgq https://t.co/M14DlRWJdu

Dec 30, 2024 · 5 likes · 29,056 followers View post →
drfranciscomd.bsky.social
@drfranciscomd.bsky.social · Bluesky

At #5 in our countdown: ‘Exagamglogene Autotemcel for Severe Sickle Cell Disease.’ This groundbreaking study demonstrates that 97% of patients treated with exa-cel were free from vaso-occlusive crises for at least 12 months, marking a significant advancement in gene therapy for sickle cell disease.

Dec 22, 2024 · 4 likes · 1 share View post →
Eman Hassan
@Eman__Hassan__ · X

RT @thelimbichaemUK: WATCH: https://t.co/jjp4SjI9WH @richardbuka and Dr Phillip Nicholson host a live version of their podcast “Don’t Just…

Sep 23, 2024 · 1 share · 151 followers View post →
the limbus haematology UK
@limbushaemUK · X

WATCH: https://t.co/jjp4SjI9WH @richardbuka and Dr Phillip Nicholson host a live version of their podcast “Don’t Just Read the Abstract”, deep diving into the recently-published ‘Exagamglogene Autotemcel for Severe Sickle Cell Disease’ (Frangoul et al., NEJM 2024). @HaemSTAR_UK https://t.co/it93DpkZR5

Sep 23, 2024 · 2 likes · 1 share · 417 followers View post →
Richard Buka
@richardbuka · X

We'll be discussing Frangoul et al. Exagamglogene autotemcel for severe sickle cell disease. https://t.co/8Ch4bvoOzr

Sep 13, 2024 · 1,419 followers View post →
the limbus haematology UK
@limbushaemUK · X

REGISTER: https://t.co/B73htxOWS7 Join @richardbuka & Dr Phillip Nicolson as they record a live version of their critical appraisal podcast “Don’t Just Read the Abstract”, diving into the recently-published ‘Exagamglogene Autotemcel for Severe Sickle Cell Disease’. @HaemSTAR_UK https://t.co/o0QMU6nHM1

Sep 6, 2024 · 2 likes · 417 followers View post →
Brianna
@_alltimebrianna · X

Attaching the link to the paper here for anyone who may want to read more: https://t.co/cNtR7AKh1N Also if anyone wants more info I could discuss this with a literal wall I love it so much.

Jun 26, 2024 · 5 likes · 6,375 followers View post →
china Victims of Sinovac COVID-19 Vaccine
@lxub84387 · X

RT @clairepomeroy: Reactivation of fetal hemoglobin synthesis by gene editing with exa-cel eliminated vaso-occlusive crises in 97% of #Sick…

Jun 11, 2024 · 1 share · 515 followers View post →
Dr. Claire Pomeroy
@clairepomeroy · X

Reactivation of fetal hemoglobin synthesis by gene editing with exa-cel eliminated vaso-occlusive crises in 97% of #SickleCellDisease patients, & gave transfusion independence to 91% of transfusion-dependent ß-thalassemia patients. https://t.co/eE6r464HNC https://t.co/GEHUja6aAG https://t.co/baTM3y76RZ

Jun 11, 2024 · 1 like · 1 share · 921 followers View post →
gilberto lopes
@GlopesMd · X

RT @SarahCannonDocs: SCRI Research Leader @hfrangoul helped pioneer exciting research published today in @NEJM showing results of CRISPR/Ca…

May 30, 2024 · 4 shares · 37,592 followers View post →
Vivek Subbiah, MD
@VivekSubbiah · X

RT @SarahCannonDocs: SCRI Research Leader @hfrangoul helped pioneer exciting research published today in @NEJM showing results of CRISPR/Ca…

May 30, 2024 · 4 shares · 38,808 followers View post →
Sickle Cell Disease Foundation
@SCDFoundation · X

A recent study revealed the now-approved gene editing therapy, Casgevy, significantly reduced rates of vaso-occlusive crises for 12 months among sickle cell patients in a trial. Read more of the study here. https://t.co/jt7885hi6S

May 23, 2024 · 2 likes · 1 share · 2,061 followers View post →
David Curtiss
@WhatsLeftOfTheD · X

Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/lNXN9mycKV

May 15, 2024 · 564 followers View post →
Young Geris (irony intended)
@YoungGeris · X

An exciting time for medicine - will be very interesting to see what the future holds for these gene editing therapies https://t.co/joH2A9RDwx

May 13, 2024 · 5,168 followers View post →
Heinrich-Heine-Universität Düsseldorf
@HHU_de · X

RT @MedHHU: Wegweisender Behandlungserfolg bei den schwerwiegenden Erbkrankheiten Beta-Thalassämie und Sichelzellkrankheit durch zielgerich…

May 13, 2024 · 1 share · 8,370 followers View post →
HHU_Medizin
@MedHHU · X

Wegweisender Behandlungserfolg bei den schwerwiegenden Erbkrankheiten Beta-Thalassämie und Sichelzellkrankheit durch zielgerichtete Geneditierungstherapie: https://t.co/banQiFLl2G + https://t.co/FQf17KCAZl

May 13, 2024 · 2 likes · 1 share · 430 followers View post →
Tom Hsiung
@TomHsiung · X

Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/WWk8vvHnGy

May 12, 2024 · 462 followers View post →
Prof Derek Connolly
@DrDerekConnolly · X

Is this the cure for sickle cell disease ? Treatment with exa-cel eliminated vaso-occlusive crises in 97% of patients Wow ! Game changing science

May 11, 2024 · 2 likes · 3,931 followers View post →
Cyrus Khandanpour
@c_khandanpour · X

RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…

May 11, 2024 · 11 shares · 408 followers View post →
旅人 tabito
@tabibito250 · X

Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/3LnDbVbr9b

May 10, 2024 · 1 like · 1,071 followers View post →
Mostafa Faisal
@MostafaFaisal14 · X

RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…

May 10, 2024 · 11 shares · 1,051 followers View post →
Hematologia ICO Hospitalet
@HematICO_Hosp · X

RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…

May 10, 2024 · 11 shares · 1,766 followers View post →
Stephan Grupp MD PhD
@GruppSteve · X

RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…

May 10, 2024 · 11 shares · 4,052 followers View post →
Alfredo I. Sagredo
@ASagredo3 · X

RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…

May 10, 2024 · 11 shares · 327 followers View post →
Chelsea C Pinnix MD PhD
@ChelseaPinnix · X

RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…

May 10, 2024 · 11 shares · 2,130 followers View post →
KOL Pulse AI
@KolPulseAI · X

RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…

May 10, 2024 · 11 shares · 951 followers View post →
Faheema Hasan
@faheema_hasan · X

RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…

May 10, 2024 · 11 shares · 5,325 followers View post →
Krishna Komanduri, MD, FASTCT
@drkomanduri · X

RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…

May 10, 2024 · 11 shares · 12,985 followers View post →
Robert Z. Orlowski
@Myeloma_Doc · X

RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…

May 10, 2024 · 11 shares · 16,306 followers View post →
Ruah Alyamany | روى اليماني
@Ruah_Mahmoud · X

RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…

May 10, 2024 · 11 shares · 1,266 followers View post →
Muzaffar Qazilbash
@Transplant_Doc · X

Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso-occlusive crises at 12 month #bmtsm @BMT_updates @GruppSteve @AkshaySharmaMD https://t.co/7MBKoRrSCP

May 9, 2024 · 32 likes · 11 shares · 7,399 followers View post →
Filippo Montemurro
@FilippoMontemu1 · X

Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine

May 9, 2024 · 2,136 followers View post →
Conrad Fischer
@SeeFisch · X

STOP WORRYING ABOUT COST. WE HAVE ENOUGH MONEY FOR CRUISE MISSILES AT ONE MILLION DOLLARS EACH Boom! 100% free from sickle hospitalizations after two years Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/WcLJBBZLEk

May 8, 2024 · 3 likes · 101,596 followers View post →
Pablo Mansfield
@mansfield_pablo · X

Major breakthrough for improvement in the lives of SCD patients who can be pain free and lead more productive & happier lives. Bravo! Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/YFCCfZ7uwt

May 8, 2024 · 339 followers View post →
Alimullah Bukhari (Qitmeer)
@alimbukhari11 · X

RT @NEJM: Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia https://t.co/LiTmJPo0pS Exagamglogene Autotemcel for Severe Si…

May 8, 2024 · 3 shares · 164 followers View post →
カミーユ=りく
@1978rikut · X

RT @NEJM: Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia https://t.co/LiTmJPo0pS Exagamglogene Autotemcel for Severe Si…

May 8, 2024 · 3 shares · 1,273 followers View post →
NEJM
@NEJM · X

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia https://t.co/LiTmJPo0pS Exagamglogene Autotemcel for Severe Sickle Cell Disease https://t.co/l5varni2by Editorial: Extending Gene Medicines to All in Need https://t.co/JbDFObRp1s https://t.co/kTmzjMyBjO

May 8, 2024 · 8 likes · 3 shares · 935,685 followers View post →
Vincent Cascone, PharmD, BCOP
@vinzumab · X

Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/RUicUbuCEz Eliminated vaso-occlusive crises in 97% of patients!

May 6, 2024 · 122 followers View post →
AlexJ EndoGenetica
@endogenetica · X

A era da terapia gênica cada vez mais próxima. No estudo silenciaram o gene BCL11A em pacientes com anemia falciforme para elevar a HbF e reduzir as crises oclusivas Exagamglogene Autotemcel for Severe Sickle Cell Disease | NEJM https://t.co/WCecINXJpV

May 5, 2024 · 2 likes · 318 followers View post →
Javier García Castro
@JavierGC280 · X

RT @LAB_BIOC: Patients with severe sickle cell disease, treated with CRISPR editing, appeared to stop severe vaso-occlusive crises, for at…

May 5, 2024 · 1 share · 235 followers View post →
Cellular Biotechnology Lab
@LAB_BIOC · X

Patients with severe sickle cell disease, treated with CRISPR editing, appeared to stop severe vaso-occlusive crises, for at least 12 months, in 29 out of 30 patients Exagamglogene autotemcel is a nonviral gene editing of BCL11A in autologous CD34+ HSPC https://t.co/QYaCms1Z1C

May 5, 2024 · 4 likes · 1 share · 143 followers View post →
旅人 tabito
@tabibito250 · X

Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/3LnDbVbYYJ

May 1, 2024 · 3 likes · 1,071 followers View post →
Salud juntos
@alfonso_casi · X

Exagamglogene Autotemcel para la anemia falciforme grave: El tratamiento con exa-cel eliminó las crisis vasooclusivas en el 97% de los pacientes con anemia falciforme durante un período de 12 meses o más. https://t.co/pDRPPMHTJf

May 1, 2024 · 8,578 followers View post →
Znger
@Znger4 · X

RT @EricTopol: A Welcome to the Era of Genome Editing today @NEJM Overview @G_Q_Daley https://t.co/pvVI26qjJR Casgevy for Sickle-Cell Dis…

Apr 30, 2024 · 124 shares · 494 followers View post →
Diane Dewhirst
@NJdoc · X

RT @EricTopol: A Welcome to the Era of Genome Editing today @NEJM Overview @G_Q_Daley https://t.co/pvVI26qjJR Casgevy for Sickle-Cell Dis…

Apr 29, 2024 · 124 shares · 20,640 followers View post →
Dr Ramdas Pananthattil
@PananthattilR · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 28, 2024 · 152 shares · 795 followers View post →
Dr Ramdas Pananthattil
@PananthattilR · X

RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…

Apr 28, 2024 · 16 shares · 795 followers View post →
Darrell Kotton
@Kottond · X

second trial here: Exagamglogene Autotemcel for Severe Sickle Cell Disease #itsthecells! #helpisontheway! DOI: 10.1056/NEJMoa2309676 | April 24, 2024 https://t.co/BkicYMZJPU

Apr 27, 2024 · 1 like · 1,862 followers View post →
BMJ Oncology
@BMJOncology · X

A CRISPR-based gene editing technology eliminated vaso-occlusive crises in 97% of patients with sickle cell disease in a landmark trial published in @NEJM. https://t.co/phRUwNHXsz https://t.co/0NeOySUHgH

Apr 27, 2024 · 4 likes · 1 share · 2,532 followers View post →
FollowMeImFaded
@FollowMeImFaded · X

RT @_alltimebrianna: Seems like a great time to share the publication around this therapy. If you’re curious about the treatment, my work a…

Apr 27, 2024 · 7 shares · 38 followers View post →
ARYAN®
@gladeolie · X

https://t.co/v4j6DNWO0N

Apr 27, 2024 · 1,492 followers View post →
桶屋 猫之介
@diccogiale21 · X

RT @norionakatsuji: 元論文→ Reference: Frangoul Haydar, Locatelli Franco, Sharma Akshay, et al. Exagamglogene autotemcel for severe sickle cel…

Apr 27, 2024 · 1 share · 358 followers View post →
Arvind Canchi (Conjeevaram)
@arvindcanchi · X

RT @DrDanMO: Exciting time:“Of the 30 patients who had sufficient follow-up…29 (97%; 95% CI, 83 to 100) were free from vaso-occlusive crise…

Apr 27, 2024 · 1 share · 10,879 followers View post →
Daniel Coyne
@DrDanMO · X

Exciting time:“Of the 30 patients who had sufficient follow-up…29 (97%; 95% CI, 83 to 100) were free from vaso-occlusive crises for at least 12 consecutive months” Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/1INdAgx1PP

Apr 26, 2024 · 3 likes · 1 share · 3,450 followers View post →
CRISP_SCIENCE
@ScienceCrisp · X

Casgevy/CTX001/exa-cel "Exagamglogene Autotemcel for Severe Sickle Cell Disease" N Engl J Med. 2024-04-24 https://t.co/vqILUdaWKI "Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia" N Engl J Med. 2024-04-24 https://t.co/v7XKqujwjB

Apr 26, 2024 · 3 likes · 1,917 followers View post →
Amanda Hill
@AmandaJillHill · X

RT @_alltimebrianna: Seems like a great time to share the publication around this therapy. If you’re curious about the treatment, my work a…

Apr 26, 2024 · 7 shares · 2,323 followers View post →
Norio Nakatsuji
@norionakatsuji · X

元論文→ Reference: Frangoul Haydar, Locatelli Franco, Sharma Akshay, et al. Exagamglogene autotemcel for severe sickle cell disease. NEJM. 2024. doi: 10.1056/NEJMoa2309676 https://t.co/HQW3qKS1yB

Apr 26, 2024 · 1 like · 1 share · 15,698 followers View post →
SYZ3RGY
@KyleHen9 · X

RT @_alltimebrianna: Seems like a great time to share the publication around this therapy. If you’re curious about the treatment, my work a…

Apr 26, 2024 · 7 shares · 316 followers View post →
Kelarax
@Kelarax_ · X

RT @_alltimebrianna: Seems like a great time to share the publication around this therapy. If you’re curious about the treatment, my work a…

Apr 26, 2024 · 7 shares · 805 followers View post →
Rory Howington
@zygrot · X

RT @_alltimebrianna: Seems like a great time to share the publication around this therapy. If you’re curious about the treatment, my work a…

Apr 26, 2024 · 7 shares · 108 followers View post →
FLIP
@FLIPtheGIANT · X

RT @_alltimebrianna: Seems like a great time to share the publication around this therapy. If you’re curious about the treatment, my work a…

Apr 26, 2024 · 7 shares · 538 followers View post →
Nailled_
@Nailed94 · X

RT @_alltimebrianna: Seems like a great time to share the publication around this therapy. If you’re curious about the treatment, my work a…

Apr 26, 2024 · 7 shares · 100 followers View post →
Brianna
@_alltimebrianna · X

Seems like a great time to share the publication around this therapy. If you’re curious about the treatment, my work at St. Jude, or what gene therapy looks like, here it is: https://t.co/seRM9Uzz5L Also would like to direct your attention to the 97%!!! of patients with no pain. https://t.co/zNDIFi33he

Apr 26, 2024 · 78 likes · 7 shares · 6,375 followers View post →
Jeannette Dabanch
@Jdabanchpena · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 26, 2024 · 152 shares · 10,344 followers View post →
stefano volpi
@stefanovolpi01 · X

RT @EricTopol: A Welcome to the Era of Genome Editing today @NEJM Overview @G_Q_Daley https://t.co/pvVI26qjJR Casgevy for Sickle-Cell Dis…

Apr 26, 2024 · 124 shares · 6 followers View post →
The Nutritional Party Book
@NutritionalBook · X

RT @EricTopol: A Welcome to the Era of Genome Editing today @NEJM Overview @G_Q_Daley https://t.co/pvVI26qjJR Casgevy for Sickle-Cell Dis…

Apr 26, 2024 · 124 shares · 421 followers View post →
Translational Exercise Biomedicine
@TEBjournal · X

RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…

Apr 26, 2024 · 16 shares · 217 followers View post →
Michal Gdula #StandWithUkraine
@michal_gdula · X

RT @EricTopol: A Welcome to the Era of Genome Editing today @NEJM Overview @G_Q_Daley https://t.co/pvVI26qjJR Casgevy for Sickle-Cell Dis…

Apr 26, 2024 · 124 shares · 1,039 followers View post →
Manoj Mayogi Mishra
@mayogisense · X

RT @CARTTherapy: Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/X1oFd3e8Be

Apr 26, 2024 · 5 shares · 572 followers View post →
Masa
@msysasa · X

RT @EricTopol: A Welcome to the Era of Genome Editing today @NEJM Overview @G_Q_Daley https://t.co/pvVI26qjJR Casgevy for Sickle-Cell Dis…

Apr 26, 2024 · 124 shares · 453 followers View post →
Silvia Monsalvo
@SilviaMonsalvo · X

RT @salvadorpayan: ¡Se resistían! Publicados en @NEJM los resultados de exa-cel (terapia CRISPR) en #talasemia y #drepanocitosis.

Apr 26, 2024 · 11 shares · 424 followers View post →
Josu Iraola Truchuelo
@JosuIraola1 · X

RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…

Apr 26, 2024 · 16 shares · 558 followers View post →
Mata Vorri MD, MPhil
@MataVorri · X

RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…

Apr 26, 2024 · 16 shares · 244 followers View post →
tonyjzhang
@TonyJZhang · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 26, 2024 · 152 shares · 24 followers View post →
John De Vos
@_jdevos_ · X

https://t.co/J14vFCcYOl

Apr 26, 2024 · 1,411 followers View post →
Martin Correa
@martinrafco · X

RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…

Apr 26, 2024 · 16 shares · 233 followers View post →
Dr. Nazifa Kadery, PharmD, RPh
@dose_of_naz · X

Treatment with exa-cel eliminated vaso-occlusive crises in 97% of patients with SCD for a period of 12 months or more #SickleCellDisease #ExagamglogeneAutotemcel #CRISPR https://t.co/lEt1ue44Br

Apr 26, 2024 · 254 followers View post →
Aghiles Hamroun
@AghilesHamroun · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 26, 2024 · 152 shares · 719 followers View post →
La-teez
@_lateez · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 26, 2024 · 152 shares · 13 followers View post →
Fahreddin Palaz, MD
@CRISPRvs · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 26, 2024 · 152 shares · 390 followers View post →
John Kopsidas
@jkopsidas · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 26, 2024 · 152 shares · 362 followers View post →
Valentin Garcia
@jvalentingg · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 26, 2024 · 152 shares · 1,188 followers View post →
Hematology Media Center (HMC)
@US_HMC · X

RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…

Apr 26, 2024 · 16 shares · 270 followers View post →
Hematology Media Center (HMC)
@US_HMC · X

RT @CARTTherapy: Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/X1oFd3e8Be

Apr 26, 2024 · 5 shares · 270 followers View post →
CARMEN_pr
@carmen_pr7 · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 26, 2024 · 152 shares · 67 followers View post →
Stefano Ciardullo
@StefanoCiardul2 · X

RT @EricTopol: A Welcome to the Era of Genome Editing today @NEJM Overview @G_Q_Daley https://t.co/pvVI26qjJR Casgevy for Sickle-Cell Dis…

Apr 26, 2024 · 124 shares · 121 followers View post →
Nazli Dizman
@NazliDizman · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 26, 2024 · 152 shares · 10,283 followers View post →
Sumanta K. Pal, MD, FASCO
@montypal · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 26, 2024 · 152 shares · 21,873 followers View post →
Santhosh Ambika
@RenoHemonc · X

RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…

Apr 26, 2024 · 16 shares · 4,006 followers View post →
Ash Alizadeh, MD/PhD
@AshAlizadeh · X

RT @EricTopol: A Welcome to the Era of Genome Editing today @NEJM Overview @G_Q_Daley https://t.co/pvVI26qjJR Casgevy for Sickle-Cell Dis…

Apr 26, 2024 · 124 shares · 3,948 followers View post →
이름뭐하지
@nameEO · X

RT @Alisvolatprop12: 중증 낫 모양 적혈구병에서 유전자 편집 (치료) - NEJM - 엑사셀(exa-cel)로 불리는 유전자 편집을 통한 세포 치료법을 12-35세 환자군에서 임상 시험한 결과 30명 중 29명이 1년 이상 혈관 막…

Apr 26, 2024 · 1 share · 2,229 followers View post →
Jernej Kovac
@JerKoNej · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 26, 2024 · 152 shares · 653 followers View post →
Yingbo Huang
@YingboHuang · X

RT @EricTopol: A Welcome to the Era of Genome Editing today @NEJM Overview @G_Q_Daley https://t.co/pvVI26qjJR Casgevy for Sickle-Cell Dis…

Apr 25, 2024 · 124 shares · 232 followers View post →
antoniofagundesjr
@fagundes_anton · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 25, 2024 · 152 shares · 237 followers View post →
Cornerstone Medical Education
@Cornerstone_CME · X

RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…

Apr 25, 2024 · 16 shares · 144 followers View post →
Jian Xu
@JianXuLab · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 25, 2024 · 152 shares · 2,727 followers View post →
Jian Xu
@JianXuLab · X

RT @EricTopol: A Welcome to the Era of Genome Editing today @NEJM Overview @G_Q_Daley https://t.co/pvVI26qjJR Casgevy for Sickle-Cell Dis…

Apr 25, 2024 · 124 shares · 2,727 followers View post →
EvelioClaros_hematologo
@hematologomd · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 25, 2024 · 152 shares · 225 followers View post →
EvelioClaros_hematologo
@hematologomd · X

RT @CARTTherapy: Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/X1oFd3e8Be

Apr 25, 2024 · 5 shares · 225 followers View post →
Franco Cavezzali
@FranCave2001 · X

RT @EricTopol: A Welcome to the Era of Genome Editing today @NEJM Overview @G_Q_Daley https://t.co/pvVI26qjJR Casgevy for Sickle-Cell Dis…

Apr 25, 2024 · 124 shares · 128 followers View post →
Makenzie Beaman-Cooper, MD PhD
@makenziebeaman · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 25, 2024 · 152 shares · 770 followers View post →
村田悠典 | 医学論文AI総合サイト MVidEra | 論文全文検索AI 開発
@yusuke_m_MU · X

掲載誌: The New England Journal of Medicine **Using generative AI: Claude 3 Opus** https://t.co/mznkVdctQ6

Apr 25, 2024 · 1 like · 2,905 followers View post →
Zahra M, PharmD, MBA, BCOP, FHOPA
@kczmj · X

RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…

Apr 25, 2024 · 16 shares · 1,394 followers View post →
村田悠典 | 医学論文AI総合サイト MVidEra | 論文全文検索AI 開発
@yusuke_m_MU · X

1. 要約: - 97%の患者で12ヶ月以上、血管閉塞性発作がみられなかった - 100%の患者で12ヶ月以上、血管閉塞性発作による入院がなかった - 中央追跡期間: 19.3ヶ月 2. 論文タイトル:  Exagamglogene Autotemcel for Severe Sickle Cell Disease 3. 筆頭著者: Frangoul H 4. 所属:

Apr 25, 2024 · 2,905 followers View post →
村田悠典 | 医学論文AI総合サイト MVidEra | 論文全文検索AI 開発
@yusuke_m_MU · X

8. Journal: The New England Journal of Medicine **Using generative AI: Claude 3 Opus** https://t.co/mznkVdctQ6

Apr 25, 2024 · 1 like · 2,905 followers View post →
村田悠典 | 医学論文AI総合サイト MVidEra | 論文全文検索AI 開発
@yusuke_m_MU · X

1. Summary:    - 97% of patients were free from vaso-occlusive crises for ≥12 months    - 100% were free from hospitalizations for vaso-occlusive crises for ≥12 months    - Median follow-up: 19.3 months 2. Title: Exagamglogene Autotemcel for Severe Sickle Cell Disease 3.

Apr 25, 2024 · 2,905 followers View post →
Rijk Gans
@Gansrob · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 25, 2024 · 152 shares · 790 followers View post →
Maruhen Datsch Silveira
@MaruhenSilveira · X

RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…

Apr 25, 2024 · 152 shares · 182 followers View post →
フロッガー
@drfrogger · X

Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/qkBDr3lcsl CRISPR-Cas9による遺伝子治療が出て来たな。

Apr 25, 2024 · 1 like · 2,513 followers View post →
Eritropatologia_ES
@eritropatologia · X

RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…

Apr 25, 2024 · 16 shares · 1,471 followers View post →
David Beneitez, MD
@beneitez_david · X

RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…

Apr 25, 2024 · 16 shares · 1,284 followers View post →
Fyodor Urnov
@UrnovFyodor · X

RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…

Apr 25, 2024 · 16 shares · 16,084 followers View post →
Dhruva Biswas, MD/PhD
@dhruva_biswas · X

RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…

Apr 25, 2024 · 16 shares · 590 followers View post →
J.A.S
@lesyeuxetoiles · X

RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…

Apr 25, 2024 · 16 shares · 221 followers View post →
Elizabeth Rhodes
@docesr · X

RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…

Apr 25, 2024 · 16 shares · 596 followers View post →
NEJM
@NEJM · X

Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Extending Gene Medicines to All in Need https://t.co/JbDFObRWR0 #Hematology https://t.co/4FVV1rw79w

Apr 25, 2024 · 53 likes · 16 shares · 935,685 followers View post →
Ahmed AlRefaei
@alrefaei_ahmed · X

RT @CARTTherapy: Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/X1oFd3e8Be

Apr 25, 2024 · 5 shares · 162 followers View post →
Mick Jagger
@JaggerMlck · X

RT @MySickleCare: Exagamglogene Autotemcel for Severe Sickle Cell Disease https://t.co/SxsdazA9qn? @akshatdoctor @TheSickleDoc @acweyand @D…

Apr 25, 2024 · 3 shares · 1,036 followers View post →
Dr. José A. Queizán Hernández
@queizan · X

RT @CARTTherapy: Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/X1oFd3e8Be

Apr 25, 2024 · 5 shares · 1,280 followers View post →
Chimeric Antigen Receptor T-Cell (CAR-T) & Gene Rx
@CARTTherapy · X

Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/X1oFd3e8Be

Apr 25, 2024 · 9 likes · 5 shares · 4,456 followers View post →
You Make Me Sick! Podcast
@makemesickpod · X

Small sample size, but great results, this @nejm article highlights really cool science to reboot bone marrow to treat sickle cell disease with gene editing. Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/xuf594rtJf

Apr 25, 2024 · 394 followers View post →
Dennis Lal
@LalDennis · X

Exagamglogene Autotemcel for Severe Sickle Cell Disease https://t.co/wdKvBtiMje https://t.co/CVCnN8eMEh

Apr 25, 2024 · 3,311 followers View post →
Eritropatologia_ES
@eritropatologia · X

RT @MySickleCare: Exagamglogene Autotemcel for Severe Sickle Cell Disease https://t.co/SxsdazA9qn? @akshatdoctor @TheSickleDoc @acweyand @D…

Apr 25, 2024 · 3 shares · 1,471 followers View post →
David Beneitez, MD
@beneitez_david · X

RT @MySickleCare: Exagamglogene Autotemcel for Severe Sickle Cell Disease https://t.co/SxsdazA9qn? @akshatdoctor @TheSickleDoc @acweyand @D…

Apr 25, 2024 · 3 shares · 1,284 followers View post →
旅人 tabito
@tabibito250 · X

Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/3LnDbVbYYJ

Apr 25, 2024 · 1 like · 1,071 followers View post →
Alejandro De Los Angeles, DPhil, MPH
@delosangeles520 · X

Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/pmUDRZiFQc

Apr 24, 2024 · 86 followers View post →
Salveo Therapeutics
@MySickleCare · X

Exagamglogene Autotemcel for Severe Sickle Cell Disease https://t.co/SxsdazA9qn? @akshatdoctor @TheSickleDoc @acweyand @DrPowerHays @DrAmandaBrandow @subarnac @drzsicklecell @nfayanders @seethal_jacobmd @sicklecelldoc @DrTitiFasipe @JuliaXuMD @DrScottHadland

Apr 24, 2024 · 8 likes · 3 shares · 297 followers View post →
Santhosh Ambika
@RenoHemonc · X

RT @UsuarezMD: Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/60ZqsTnjO9

Apr 24, 2024 · 4 shares · 4,006 followers View post →
Fabio Torres
@FabioTorressav · X

RT @UsuarezMD: Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/60ZqsTnjO9

Apr 24, 2024 · 4 shares · 671 followers View post →
Uriel Suárez
@UsuarezMD · X

Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/60ZqsTnjO9

Apr 24, 2024 · 2 likes · 4 shares · 3,341 followers View post →
Paul Shaughnessy
@PaulS1594 · X

RT @SarahCannonDocs: Earlier today, @hfrangoul's oral presentation titled, "Exagamglogene Autotemcel for Severe Sickle Cell Disease,” was s…

Dec 12, 2023 · 1 share · 247 followers View post →
Sarah Cannon Docs
@SarahCannonDocs · X

Earlier today, @hfrangoul's oral presentation titled, "Exagamglogene Autotemcel for Severe Sickle Cell Disease,” was selected as a #BestofASH at the @ASH_hematology annual meeting! We are so proud of this important work. https://t.co/eysrm4NP2l

Dec 12, 2023 · 2 likes · 1 share · 3,855 followers View post →
Greg5thTrad
@Greg5thTrad · X

Friday - Dec 8 It is the @US_FDA action date on Vertex Pharmaceuticals $VRTX for the investigational treatment exagamglogene autotemcel for severe sickle cell disease. https://t.co/fW35fYCFhM

Dec 3, 2023 · 1 like · 1,327 followers View post →
Chimeric Antigen Receptor T-Cell (CAR-T) & Gene Rx
@CARTTherapy · X

Exagamglogene Autotemcel for Severe Sickle Cell Disease #GeneTherapy #ASH23 https://t.co/aqfdYhzvE7 @hfrangoul https://t.co/N2aRAlwpi3

Nov 17, 2023 · 7 likes · 4,456 followers View post →
Investing.com News
@newsinvesting · X

Vertex Pharma Announces FDA Acceptance of BLA for exagamglogene autotemcel for Severe Sickle Cell Disease and... - https://t.co/D7Wd4Qi8xv

Jun 9, 2023 · 229,533 followers View post →