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exagamglogene autotemcel

Generic: exagamglogene autotemcel·Brand: Casgevy

exagamglogene autotemcel: developed by 2 companies · 7 clinical trials tracked.

exagamglogene autotemcel evidence translation

What we can confirm

7 tracked trials

What it means

More than one developer is source-backed. Until you choose one, AI stays drug-only and does not guess ownership.

What to watch

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

Prepared research question

What is verified about exagamglogene autotemcel, and what remains unknown?

Run cited research

Traceable citations · Unknowns marked

See linked entities and source evidence8 indications · 1 study sponsors

Related indications

Severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) in patients 12 years and olderSickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT)Beta-ThalassemiaGenetic Diseases, InbornHematologic DiseasesHemoglobinopathiesSickle Cell DiseaseThalassemia
Development Pipeline (2)
CompanyPhase
CRISPR Therapeutics AG (CRSP)
Severe sickle cell disease (SCD) and transfusion-dependent b…
Approved
Editas Medicine, Inc. (EDIT)
Sickle cell disease (SCD) and transfusion-dependent beta tha…
Approved
Clinical Trials (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

NCT05951205·Phase 3·VRTX
Withdrawn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

NCT05477563·Phase 3·VRTX
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

NCT05356195·Phase 3·VRTX
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

NCT05329649·Phase 3·VRTX
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)

NCT04208529·Phase 3·VRTX
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

NCT03745287·Phase 2/Phase 3·VRTX
Completed

Primary endpoint: Proportion of subjects who have not experienced any severe vaso-occlusive crisis (VOC) for at least 12 consecutive months (VF12)

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

NCT03655678·Phase 2/Phase 3·VRTX
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

Who is developing exagamglogene autotemcel?

exagamglogene autotemcel is being developed by CRISPR Therapeutics AG, Editas Medicine, Inc..

How many clinical trials involve exagamglogene autotemcel?

BioSniper tracks 7 clinical trials involving exagamglogene autotemcel.