NCT04208529ENROLLING_BY_INVITATIONanonymous

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)

Sponsor

Source record

Vertex Pharmaceuticals Incorporated

Phase

Source record

Phase 3

Modality

AI-normalized

protein therapy

Target

AI-normalized

CRISPR-Cas9 modified hematopoietic stem cells

Indication / condition

AI-normalized

Beta-Thalassemia

Intervention

Source record

CTX001

Source & freshness

Source record

NCT ID

NCT04208529

Original source

ClinicalTrials.gov

Source last updated

Mar 25, 2026

Ingested at

Jun 17, 2026

Internal sync

Jun 17, 2026

Model version

trialsignal-ai-v1

Normalized confidence

96%

Validation status

validated

Open original registry record

View original source fields

NCT ID

NCT04208529

Title

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)

Sponsor

Vertex Pharmaceuticals Incorporated

Status

ENROLLING_BY_INVITATION

Phase

Phase 3

Condition raw

Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia

Condition normalized

Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia

Modality raw

protein therapy

Modality normalized

protein therapy

Target raw

CRISPR-Cas9 modified hematopoietic stem cells

Target normalized

CRISPR-Cas9 modified hematopoietic stem cells

Interventions

CTX001

Public preview

Source record

Vertex Pharmaceuticals, in collaboration with CRISPR Therapeutics, is advancing CTX001, a CRISPR-Cas9 gene-editing therapy targeting β-thalassemia and sickle cell disease (SCD). The long-term follow-up study aims to assess the safety and efficacy of CTX001 in patients previously treated in parent studies. The market for gene therapies in hematologic diseases is expanding, driven by increasing prevalence and demand for innovative treatments. Successful outcomes could position CTX001 as a leading therapy in this niche, potentially capturing significant market share and enhancing Vertex's portfolio, especially as competition intensifies with other gene-editing technologies and therapies in development.

AI-generated analysis supports research triage only. Verify source records, publications, sponsor disclosures and IP databases before making diligence decisions. Model: trialsignal-ai-v1.

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TrialSignal

Clinical trial intelligence report

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)

Source-linked diligence brief with registry provenance, taxonomy normalization and premium analytical context.

Generated

Jun 17, 2026

NCT ID

NCT04208529

Status

ENROLLING_BY_INVITATION

Phase

Phase 3

Sponsor

Vertex Pharmaceuticals Incorporated

Executive brief

Investment-Ready Snapshot

Source & freshness

Provenance

SourceClinicalTrials.gov

Source updatedMar 25, 2026

Internal syncJun 17, 2026

Model versiontrialsignal-ai-v1

https://clinicaltrials.gov/study/NCT04208529

Indication

Beta-Thalassemia

Modality

protein therapy

Target

CRISPR-Cas9 modified hematopoietic stem cells

Intervention

CTX001

Source record