Sickle Cell Disease

BIVV003, formerly known as SAR445136
  • Status
    Phase 1/2
  • Partner
    wholly owned
  • Technology
    Autologous gene-edited cell therapy

Disease

Sickle Cell Disease (SCD) is a blood disorder caused by a mutation in the hemoglobin gene, resulting in distorted red blood cells (sickle cells). These sickle cells either die early or cause blockages in small blood vessels. SCD’s main symptom is agonizing pain – either chronic or episodic – due to ischemia, a restriction in the blood flow to certain organs.

graphic showing what sickle cell disease is

Genomic medicine approach

BIVV003 is a zinc finger nuclease (ZFN) gene-edited cell therapy candidate. BIVV003 is manufactured by ex vivo gene editing of a patient’s own (autologous) hematopoietic stem cells using non-viral delivery of ZFN technology targeting the BCL11a gene erythroid-specific enhancer. As this gene naturally stops the production of fetal hemoglobin (HbF) in the first few months of life, its deactivation by BIVV003 is expected to restore the production of functional HbF and relieve SCD symptoms.


Phase 1/2 PRECIZN-1 Study

The Phase 1/2 Alta study is an open-label, dose-ranging, multicenter clinical trial designed to assess the safety and tolerability of giroctocogene fitelparvovec in patients with severe hemophilia A. The mean age of the 11 male patients assessed across four dose cohorts (9e11 vg/kg – 2 patients, 2e12 vg/kg – 2 patients, 1 e13 vg/kg – 2 patients and 3e13 vg/kg – 5 patients) is 30 years (range 18-47 years). Patients in this study will be assessed every six months until they enroll in a long-term follow-up study.

Designations

BIVV003 has received Fast Track Designation from the U.S. Food and Drug Administration (FDA) and Orphan Medicinal Product from the European Medicines Agency (EMA).

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