EXAGAMGLOGENE AUTOTEMCEL

also known as CTX001 · Casgevy · Exa-cel

Developed for: sickle cell disease · beta thalassemia · age-related macular degeneration · anemia · disorder of iron metabolism and transport
Investigated by: Vertex Pharmaceuticals Incorporated · CRISPR Therapeutics AG · Cajal Therapeutics Inc. · Complement Therapeutics

Trials 9 · a red edge is where a trial was stopped

today NCT05951205 · Ph 3 NCT07577817 · Ph 0 NCT07392255 · Ph 1/2 NCT05477563 · Ph 3 NCT05356195 · Ph 3 NCT05329649 · Ph 3 NCT04208529 · Ph 3 NCT03745287 · Ph 2/3 NCT03655678 · Ph 2/3

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Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 01 trial
Phase 0	NCT07577817	Jun → Oct 2026 expected	anemia, chronic kidney disease, disorder of iron metabolism and transport	Cajal Therapeutics Inc.	Recruiting	No outcome recorded
Phase 1/21 trial
Phase 1/2	NCT07392255	Dec 2025 → May 2028 expected	age-related macular degeneration	Complement Therapeutics	Recruiting	No outcome recorded
Phase 2/32 trials
Phase 2/3	NCT03745287	Nov 2018 → Jul 2025	sickle cell disease	Vertex Pharmaceuticals Incorporated	Completed	No outcome recorded
Phase 2/3	NCT03655678	Sep 2018 → Nov 2025	beta thalassemia	Vertex Pharmaceuticals Incorporated	Completed	No outcome recorded
Phase 35 trials
Phase 3	NCT05951205	Jul 2027 → Dec 2033	sickle cell disease	Vertex Pharmaceuticals Incorporated	Withdrawn	No outcome recorded
Phase 3	NCT05477563	Aug 2022 → Jun 2027 expected	beta thalassemia, sickle cell disease	Vertex Pharmaceuticals Incorporated	Recruiting	No outcome recorded
Phase 3	NCT05356195	May 2022 → Nov 2027 expected	beta thalassemia	Vertex Pharmaceuticals Incorporated	Active not recruiting	No outcome recorded
Phase 3	NCT05329649	May 2022 → Jun 2027 expected	sickle cell disease	Vertex Pharmaceuticals Incorporated	Active not recruiting	No outcome recorded
Phase 3	NCT04208529	Jan 2021 → Sep 2039 expected	beta thalassemia, sickle cell disease	Vertex Pharmaceuticals Incorporated	Enrolling by invitation	No outcome recorded

Press releases naming this drug 39 releases

Date	Issuer	Release
2026-07-01	Vertex Pharmaceuticals Incorporated	Vertex Announces US FDA Approval for Expanded Use of CASGEVY® for the Treatment of People Ages 2 Years and Older With Sickle Cell Disease or Transfusion-Dependent Beta Thalassemia vrtx.com ↗
2026-06-11	Vertex Pharmaceuticals Incorporated	Vertex Presents New Data on CASGEVY®, Including First European Presentation of Data in Children Ages 5–11, at the European Hematology Association Congress and Announces Additional Global Regulatory Submissions vrtx.com ↗
2026-05-06	Vertex Pharmaceuticals Incorporated	Vertex Announces CASGEVY® Reimbursement Agreement for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia in Germany vrtx.com ↗
2025-12-06	Vertex Pharmaceuticals Incorporated	Vertex Presents New Data on CASGEVY®, Including First-Ever Data in Children Ages 5-11 Years, at the American Society of Hematology Annual Meeting and Announces Plan for Global Regulatory Submissions vrtx.com ↗
2025-09-18	Vertex Pharmaceuticals Incorporated	Vertex Announces CASGEVY® Reimbursement Agreement for the Treatment of Transfusion-Dependent Beta Thalassemia and Sickle Cell Disease in Italy vrtx.com ↗
2025-06-12	Vertex Pharmaceuticals Incorporated	Vertex Presents Longer-Term Data at the 2025 European Hematology Association (EHA) Congress Demonstrating Durability of CASGEVY® and Provides Update on Expanding Global Access to CASGEVY vrtx.com ↗
2025-01-31	Vertex Pharmaceuticals Incorporated	Vertex Announces CASGEVY® Reimbursement Agreement for the Treatment of Sickle Cell Disease in England vrtx.com ↗
2024-12-08	Vertex Pharmaceuticals Incorporated	Vertex Presents Positive Long-Term Data On CASGEVY™ (exagamglogene autotemcel) at the American Society of Hematology (ASH) Annual Meeting and Exposition and Provides Program Update vrtx.com ↗
2024-08-07	Vertex Pharmaceuticals Incorporated	Vertex Announces CASGEVY™ Reimbursement Agreement for the Treatment of Transfusion-Dependent Beta Thalassemia in England vrtx.com ↗
2024-06-14	Vertex Pharmaceuticals Incorporated	Vertex Presents Positive Long-Term Data On CASGEVY™ (exagamglogene autotemcel) at the 2024 Annual European Hematology Association (EHA) Congress vrtx.com ↗
2024-02-13	Vertex Pharmaceuticals Incorporated	European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia vrtx.com ↗
2024-01-16	Vertex Pharmaceuticals Incorporated	Vertex Announces US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia vrtx.com ↗
2024-01-09	Vertex Pharmaceuticals Incorporated	Vertex Announces Approval of First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™, for the Treatment of Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) in Kingdom of Saudi Arabia vrtx.com ↗
2023-12-15	Vertex Pharmaceuticals Incorporated	Vertex Receives CHMP Positive Opinion for the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia vrtx.com ↗
2023-12-11	Vertex Pharmaceuticals Incorporated	Positive Results from Pivotal Trials of CASGEVY™ (exagamglogene autotemcel) Highlighted in Oral Presentations at the American Society of Hematology (ASH) Annual Meeting and Exposition vrtx.com ↗
2023-12-08	Vertex Pharmaceuticals Incorporated	Vertex and CRISPR Therapeutics Announce US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Sickle Cell Disease vrtx.com ↗
2023-11-16	Vertex Pharmaceuticals Incorporated	Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia vrtx.com ↗
2023-11-02	Vertex Pharmaceuticals Incorporated	Vertex to Present New Data on Exagamglogene Autotemcel (exa-cel) at the American Society of Hematology (ASH) Annual Meeting and Exposition vrtx.com ↗
2023-06-09	Vertex Pharmaceuticals Incorporated	Positive Results From Pivotal Trials of exa-cel for Transfusion-Dependent Beta Thalassemia and Severe Sickle Cell Disease Presented at the 2023 Annual European Hematology Association (EHA) Congress vrtx.com ↗
2023-06-08	Vertex Pharmaceuticals Incorporated	FDA Accepts Biologics License Applications for exagamglogene autotemcel (exa-cel) for Severe Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia vrtx.com ↗
2023-04-03	Vertex Pharmaceuticals Incorporated	Vertex and CRISPR Therapeutics Complete Submission of Rolling Biologics License Applications (BLAs) to the US FDA for exa-cel for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia vrtx.com ↗
2022-09-27	Vertex Pharmaceuticals Incorporated	Vertex and CRISPR Therapeutics Announce Global exa-cel Regulatory Submissions for Sickle Cell Disease and Beta Thalassemia in 2022 vrtx.com ↗
2022-06-11	Vertex Pharmaceuticals Incorporated	Vertex and CRISPR Therapeutics Present New Data on More Patients With Longer Follow-Up Treated With exagamglogene autotemcel (exa-cel) at the 2022 European Hematology Association (EHA) Congress vrtx.com ↗
2022-06-02	Vertex Pharmaceuticals Incorporated	Vertex and CRISPR Therapeutics Announce Acceptance of Late-Breaking Abstract for CTX001™ at the 2022 Annual European Hematology Association (EHA) Congress vrtx.com ↗
2021-06-11	Vertex Pharmaceuticals Incorporated	Vertex and CRISPR Therapeutics Present New Data in 22 Patients With Greater Than 3 Months Follow-Up Post-Treatment With Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at European Hematology Association Annual Meeting vrtx.com ↗
2020-12-05	Vertex Pharmaceuticals Incorporated	CRISPR Therapeutics and Vertex Present New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at American Society of Hematology Annual Meeting and Exposition, Together With Publication in the New England Journal of Medicine vrtx.com ↗
2020-12-01	Vertex Pharmaceuticals Incorporated	CRISPR Therapeutics and Vertex to Host Investor Webcast to Review Data Presented at the 62nd American Society of Hematology Annual (ASH) Meeting and Exposition for Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001™ in Sickle Cell Disease and Beta Thalassemia vrtx.com ↗
2020-11-04	Vertex Pharmaceuticals Incorporated	CRISPR/Cas9 Gene-Editing Therapy CTX001™ for Severe Hemoglobinopathies Accepted for Plenary Presentation at the 62nd American Society of Hematology (ASH) Meeting and Exposition vrtx.com ↗
2020-09-22	Vertex Pharmaceuticals Incorporated	CRISPR Therapeutics and Vertex Pharmaceuticals Announce Priority Medicines (PRIME) Designation Granted by the European Medicines Agency (EMA) to CTX001™ for the Treatment of Sickle Cell Disease vrtx.com ↗
2020-06-12	Vertex Pharmaceuticals Incorporated	CRISPR Therapeutics and Vertex Announce New Clinical Data for Investigational Gene-Editing Therapy CTX001™ in Severe Hemoglobinopathies at the 25th Annual European Hematology Association (EHA) Congress vrtx.com ↗
2020-05-14	Vertex Pharmaceuticals Incorporated	New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001™ for Severe Hemoglobinopathies Accepted for Oral Presentation at the 25th European Hematology Association (EHA) Congress vrtx.com ↗
2020-05-11	Vertex Pharmaceuticals Incorporated	CRISPR Therapeutics and Vertex Pharmaceuticals Announce FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX001™ for the Treatment of Severe Hemoglobinopathies vrtx.com ↗
2019-11-19	Vertex Pharmaceuticals Incorporated	CRISPR Therapeutics and Vertex Announce Positive Safety and Efficacy Data From First Two Patients Treated With Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001® for Severe Hemoglobinopathies vrtx.com ↗
2019-04-16	Vertex Pharmaceuticals Incorporated	CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Beta Thalassemia vrtx.com ↗
2019-02-25	Vertex Pharmaceuticals Incorporated	CRISPR Therapeutics and Vertex Announce Progress in Clinical Development Programs for the Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001 vrtx.com ↗
2019-01-04	Vertex Pharmaceuticals Incorporated	CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Sickle Cell Disease vrtx.com ↗
2018-10-10	Vertex Pharmaceuticals Incorporated	CRISPR Therapeutics and Vertex Announce FDA Has Lifted the Clinical Hold on the Investigational New Drug Application for CTX001 for the Treatment of Sickle Cell Disease vrtx.com ↗
2018-05-30	Vertex Pharmaceuticals Incorporated	CRISPR Therapeutics and Vertex Provide Update on FDA Review of Investigational New Drug Application for CTX001 for the Treatment of Sickle Cell Disease vrtx.com ↗
2017-12-12	Vertex Pharmaceuticals Incorporated	Vertex and CRISPR Therapeutics to Co-Develop and Co-Commercialize CTX001 as CRISPR/Cas9 Gene Edited Treatment for Sickle Cell Disease and β-Thalassemia vrtx.com ↗

Evidence & citations 8 cited values

Every value below carries the sentence it was read from. 12 sources stand behind the page.

Field	Value	Cited text
Known as	EXAGAMGLOGENE AUTOTEMCEL	ChEMBL registry synonym — accepted as the source's own label CHEMBL5095474 ↗
Known as	Casgevy	ChEMBL registry synonym — accepted as the source's own label CHEMBL5095474 ↗
Known as	CTX001	ClinicalTrials.gov intervention name — accepted as the source's own label NCT05329649 ↗ 7 NCT03655678 ↗ NCT05477563 ↗ NCT05356195 ↗ NCT04208529 ↗ NCT07577817 ↗ NCT07392255 ↗ NCT03745287 ↗
Known as	Exa-cel	ClinicalTrials.gov intervention name — accepted as the source's own label NCT05951205 ↗
Action	Restore	“designed to reactivate fetal hemoglobin synthesis through ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing” PMID 38657265 ↗ Apr 2024
Modality	CRISPR / gene editing	“a one-time, ex vivo CRISPR/Cas9 gene-edited cell therapy” PMID 40857358 ↗ Dec 2025
Route	Intravenous	“Administered by IV infusion following myeloablative conditioning with busulfan” NCT03655678 ↗
Target	BCL11A	“CTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene)” NCT03655678 ↗