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exagamglogene autotemcel

Generic: exagamglogene autotemcel·Brand: Casgevy

exagamglogene autotemcel: sourced relationships with 1 company · 7 clinical trials tracked.

exagamglogene autotemcel evidence translation

What we can confirm

What it means

Source-backed records connect exagamglogene autotemcel to EDIT; that link alone does not prove the drug works.

What to watch

Watch trial status, endpoints, and the next source-backed update.
BioSniper AIPage evidence connected

Prepared research question

How could exagamglogene autotemcel → EDIT affect its developer, and what evidence supports the link?

Run cited research

Traceable citations · Unknowns marked

See linked entities and source evidence7 indications · 0 study sponsors

Related indications

Sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT)Beta-ThalassemiaGenetic Diseases, InbornHematologic DiseasesHemoglobinopathiesSickle Cell DiseaseThalassemia

Study sponsors

No additional listed-company sponsor is linked.

Dated Pipeline Records (1)
CompanyDated record stage
Editas Medicine, Inc. (EDIT)
licensee · Reported 2025-03-05 · Current development: unknownSource
Sickle cell disease (SCD) and transfusion-dependent beta tha…
Approved
Clinical Trial Registry Records (7)

A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects With Severe Sickle Cell Disease, βS/βC Genotype

NCT05951205Registry updated 2026-03-25·Phase 3
Withdrawn

Primary endpoint: Proportion of Participants with an Average Fetal Hemoglobin (HbF) Greater Than or Equal To (>=) 20 percent (%) on or After 6 Months

Interventionalopen0 targetStarted 2027-07Completion 2033-12Vertex Pharmaceuticals Incorporated

A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease

NCT05477563Registry updated 2026-03-23·Phase 3
Recruiting

Primary endpoint: Fetal Hemoglobin (HbF) Concentration Over Time

Interventionalopen26 targetStarted 2022-08Completion 2027-066 sites · Germany, Italy, Saudi Arabia, United StatesVertex Pharmaceuticals Incorporated

A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-Thalassemia

NCT05356195Registry updated 2026-07-01·Phase 3
Active, not recruiting

Primary endpoint: Proportion of Participants who Achieve Transfusion Independence for at Least 12 Consecutive Months (TI12)

Interventionalopen16 targetStarted 2022-05Completion 2027-116 sites · Canada, Germany, Italy, United Kingdom…Vertex Pharmaceuticals Incorporated

A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease

NCT05329649Registry updated 2026-07-01·Phase 3
Active, not recruiting

Primary endpoint: Proportion of Participants who do not Have any Severe Vaso-occlusive Crises (VOCs) for at Least 12 Consecutive Months (VF12)

Interventionalopen13 targetStarted 2022-05Completion 2027-067 sites · Germany, Italy, United Kingdom, United StatesVertex Pharmaceuticals Incorporated

A Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated With Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)

NCT04208529Registry updated 2026-08-11·Phase 3
Enrolling by invitation

Primary endpoint: New malignancies

Interventionalopen160 targetStarted 2021-01Completion 2039-0920 sites · Belgium, Canada, Germany, Italy…Vertex Pharmaceuticals Incorporated

A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

NCT03745287Registry updated 2026-09-16·Phase 2/Phase 3
Completed

Primary endpoint: Percentage of Participants Who Have Not Experienced Any Severe Vaso-occlusive Crisis (VOC) for at Least 12 Consecutive Months (VF12) After Exa-cel Infusion

Interventionalopen63 enrolledStarted 2018-11Completion 2025-0717 sites · Belgium, Canada, France, Germany…Vertex Pharmaceuticals Incorporated

A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects With Transfusion-Dependent β-Thalassemia

NCT03655678Registry updated 2025-12-17·Phase 2/Phase 3
Completed

Primary endpoint: Proportion of participants achieving transfusion independence for at least 12 consecutive months (TI12)

Interventionalopen59 enrolledStarted 2018-09Completion 2025-1114 sites · Canada, Germany, Italy, United Kingdom…Vertex Pharmaceuticals Incorporated

Frequently asked questions

Which companies have sourced relationships with exagamglogene autotemcel?

exagamglogene autotemcel has dated SEC/FDA relationship records for Editas Medicine, Inc..

How many clinical trials involve exagamglogene autotemcel?

BioSniper tracks 7 clinical trials involving exagamglogene autotemcel.