BIVV003

DRUG2 trials

Sponsors

Sangamo Therapeutics

Conditions

Blood and Lymphatic DiseasesSickle Cell Disease

Phase 1

A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease

CompletedNCT03653247

Sangamo TherapeuticsSickle Cell Disease

Start: 2019-03-06End: 2025-07-17Updated: 2025-09-12

Unknown Phase

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

Active, not recruitingNCT05145062

Sangamo TherapeuticsBlood and Lymphatic Diseases

Start: 2021-12-21End: 2038-07-14Updated: 2025-11-12

Related Papers

Interim Safety and Efficacy Results from a Phase 1/2 Study of Zinc Finger Nuclease-Modified Autologous Hematopoietic Stem Cells for Sickle Cell Disease (PRECIZN-1)

Blood2022-11-1514 citations

Preliminary Safety and Efficacy Results from Precizn-1: An Ongoing Phase 1/2 Study on Zinc Finger Nuclease-Modified Autologous CD34+ HSPCs for Sickle Cell Disease (SCD)

Blood2021-11-0526 citations

Quantitative Systems Pharmacology Model of Sickle Cell Disease and Response to Gene Editing Therapy to Support Clinical Development of SAR445136 (BIVV003)

Blood2021-11-051 citations

Treating sickle cell anemia: A new era dawns

American Journal of Hematology2020-01-1124 citations

Zinc Finger Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer Results in Enriched Biallelic Editing, Increased Fetal Hemoglobin, and Reduced Sickling in Erythroid Cells Derived from Sickle Cell Disease Patients

Blood2019-11-137 citations

PF736 ZINC FINGER NUCLEASE‐MEDIATED DISRUPTION OF THE BCL11A ERYTHROID ENHANCER IN PLERIXAFOR MOBILIZED CD34+ CELLS RESULTS IN ENRICHED BIALLELEIC EDITING AND ALLELE‐ADDITIVE INCREASES IN FETAL HEMOGLOBIN

HemaSphere2019-06-01