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
Exagamglogene Autotemcel for Severe Sickle Cell Disease.
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Jun 2023 – Aug 2025Where it's discussed
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X / Twitter140 posts
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News24 articles
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Blogs6 articles
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YouTube3 videos
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Facebook3 posts
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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.
Almost all discussions express strong support for the findings and approach presented in the publication.
The discussions indicate a very high level of curiosity and engagement with the topic of gene therapy for sickle cell disease.
Many posts dive into specific aspects of the research, including methodologies and implications, reflecting deep engagement with the material.
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 BlueskyOriginal 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
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
Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/X1oFd3e8Be
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
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
All mentions
Gene Editing and Its Transformative Impact on Science and Medicine
Discover the revolutionary power of CRISPR-Cas9 gene editing technology, which is transforming science and medicine by enabling disease resistance, bacterial plastic digestion, and potential cures for genetic disorders.
CRISPR Therapy: A Breakthrough in Sickle Cell Disease Treatment
What if you could switch off disease by turning on a forgotten gene? Exagamglogene autotemcel is a groundbreaking FDA-approved CRISPR therapy for sickle cell disease, enhancing fetal hemoglobin production. Results…
CRISPR Cell Therapy Shows Promise in Sickle Cell Disease
CRISPRedited stem cells have shown remarkable results, stopping severe pain crises in 29 of 30 sickle cell patients. Exagamglogene autotemcel (exa-cel) employs CRISPR-Cas9 technology, resulting in 97% of patients free…
Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/y4YVpHtz7H
@john_evans3 Hey John. Is this study different from "Exagamglogene Autotemcel for Severe Sickle Cell Disease" one?
RT @EL_KUT: @Danjauro_ @muasaidu Very good data. Main issues are TRM from MAC and possible off target. Otherwise very good science https://…
@Danjauro_ @muasaidu Very good data. Main issues are TRM from MAC and possible off target. Otherwise very good science https://t.co/Yy4QvAAhJo
Interesting phase 3 trials too: https://t.co/0DyUf07Yor https://t.co/kK6utbYIlf BBC article: https://t.co/CpJLyGRYRH
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
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
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.
RT @thelimbichaemUK: WATCH: https://t.co/jjp4SjI9WH @richardbuka and Dr Phillip Nicholson host a live version of their podcast “Don’t Just…
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
We'll be discussing Frangoul et al. Exagamglogene autotemcel for severe sickle cell disease. https://t.co/8Ch4bvoOzr
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
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.
RT @clairepomeroy: Reactivation of fetal hemoglobin synthesis by gene editing with exa-cel eliminated vaso-occlusive crises in 97% of #Sick…
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
RT @SarahCannonDocs: SCRI Research Leader @hfrangoul helped pioneer exciting research published today in @NEJM showing results of CRISPR/Ca…
RT @SarahCannonDocs: SCRI Research Leader @hfrangoul helped pioneer exciting research published today in @NEJM showing results of CRISPR/Ca…
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
Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/lNXN9mycKV
An exciting time for medicine - will be very interesting to see what the future holds for these gene editing therapies https://t.co/joH2A9RDwx
RT @MedHHU: Wegweisender Behandlungserfolg bei den schwerwiegenden Erbkrankheiten Beta-Thalassämie und Sichelzellkrankheit durch zielgerich…
Wegweisender Behandlungserfolg bei den schwerwiegenden Erbkrankheiten Beta-Thalassämie und Sichelzellkrankheit durch zielgerichtete Geneditierungstherapie: https://t.co/banQiFLl2G + https://t.co/FQf17KCAZl
Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/WWk8vvHnGy
Is this the cure for sickle cell disease ? Treatment with exa-cel eliminated vaso-occlusive crises in 97% of patients Wow ! Game changing science
RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…
Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/3LnDbVbr9b
RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…
RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…
RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…
RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…
RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…
RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…
RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…
RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…
RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…
RT @Transplant_Doc: Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso…
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
Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine
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
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
RT @NEJM: Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia https://t.co/LiTmJPo0pS Exagamglogene Autotemcel for Severe Si…
RT @NEJM: Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia https://t.co/LiTmJPo0pS Exagamglogene Autotemcel for Severe Si…
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
Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/RUicUbuCEz Eliminated vaso-occlusive crises in 97% of patients!
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
RT @LAB_BIOC: Patients with severe sickle cell disease, treated with CRISPR editing, appeared to stop severe vaso-occlusive crises, for at…
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
Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/3LnDbVbYYJ
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
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…
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…
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…
second trial here: Exagamglogene Autotemcel for Severe Sickle Cell Disease #itsthecells! #helpisontheway! DOI: 10.1056/NEJMoa2309676 | April 24, 2024 https://t.co/BkicYMZJPU
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
RT @_alltimebrianna: Seems like a great time to share the publication around this therapy. If you’re curious about the treatment, my work a…
https://t.co/v4j6DNWO0N
RT @norionakatsuji: 元論文→ Reference: Frangoul Haydar, Locatelli Franco, Sharma Akshay, et al. Exagamglogene autotemcel for severe sickle cel…
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…
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
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
RT @_alltimebrianna: Seems like a great time to share the publication around this therapy. If you’re curious about the treatment, my work a…
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
RT @_alltimebrianna: Seems like a great time to share the publication around this therapy. If you’re curious about the treatment, my work a…
RT @_alltimebrianna: Seems like a great time to share the publication around this therapy. If you’re curious about the treatment, my work a…
RT @_alltimebrianna: Seems like a great time to share the publication around this therapy. If you’re curious about the treatment, my work a…
RT @_alltimebrianna: Seems like a great time to share the publication around this therapy. If you’re curious about the treatment, my work a…
RT @_alltimebrianna: Seems like a great time to share the publication around this therapy. If you’re curious about the treatment, my work a…
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
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
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…
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…
RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…
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…
RT @CARTTherapy: Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/X1oFd3e8Be
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…
RT @salvadorpayan: ¡Se resistían! Publicados en @NEJM los resultados de exa-cel (terapia CRISPR) en #talasemia y #drepanocitosis.
RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…
RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
https://t.co/J14vFCcYOl
RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…
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
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…
RT @CARTTherapy: Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/X1oFd3e8Be
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
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…
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…
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…
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…
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
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…
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
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…
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
RT @CARTTherapy: Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/X1oFd3e8Be
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…
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
掲載誌: The New England Journal of Medicine **Using generative AI: Claude 3 Opus** https://t.co/mznkVdctQ6
RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…
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:
8. Journal: The New England Journal of Medicine **Using generative AI: Claude 3 Opus** https://t.co/mznkVdctQ6
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.
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
RT @NEJM: Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cell…
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。
RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…
RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…
RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…
RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…
RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…
RT @NEJM: Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) https://t.co/l5varniA16 Editorial: Ex…
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
RT @CARTTherapy: Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/X1oFd3e8Be
RT @MySickleCare: Exagamglogene Autotemcel for Severe Sickle Cell Disease https://t.co/SxsdazA9qn? @akshatdoctor @TheSickleDoc @acweyand @D…
RT @CARTTherapy: Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/X1oFd3e8Be
Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/X1oFd3e8Be
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
Exagamglogene Autotemcel for Severe Sickle Cell Disease https://t.co/wdKvBtiMje https://t.co/CVCnN8eMEh
RT @MySickleCare: Exagamglogene Autotemcel for Severe Sickle Cell Disease https://t.co/SxsdazA9qn? @akshatdoctor @TheSickleDoc @acweyand @D…
RT @MySickleCare: Exagamglogene Autotemcel for Severe Sickle Cell Disease https://t.co/SxsdazA9qn? @akshatdoctor @TheSickleDoc @acweyand @D…
Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/3LnDbVbYYJ
Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/pmUDRZiFQc
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
RT @UsuarezMD: Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/60ZqsTnjO9
RT @UsuarezMD: Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/60ZqsTnjO9
Exagamglogene Autotemcel for Severe Sickle Cell Disease | New England Journal of Medicine https://t.co/60ZqsTnjO9
RT @SarahCannonDocs: Earlier today, @hfrangoul's oral presentation titled, "Exagamglogene Autotemcel for Severe Sickle Cell Disease,” was s…
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
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
Exagamglogene Autotemcel for Severe Sickle Cell Disease #GeneTherapy #ASH23 https://t.co/aqfdYhzvE7 @hfrangoul https://t.co/N2aRAlwpi3
Vertex Pharma Announces FDA Acceptance of BLA for exagamglogene autotemcel for Severe Sickle Cell Disease and... - https://t.co/D7Wd4Qi8xv