Clinical Trial

Disease: Sickle Cell Disease, SCD, (NCT03653247)

Disease info:

Sickle cell disease is a group of disorders that affects haemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. People with this disorder have atypical haemoglobin molecules called haemoglobin S, which can distort red blood cells into a sickle or crescent shape.

The production of haemoglobin A, which is the principle type of haemoglobin in humans, is governed by 3 genes: HBA1, HBA2, and HBB. Each haemoglobin A molecule consists of two alpha and two beta chains, and mutations in either of the HBA or the HBB genes may result in abnormal haemoglobin molecules with reduced or diminshed function. Sickle cell diseaase arises from a single point mutation in the 6th codon of the beta-globin gene (HBB), which results in a valine instead of a glutamic acid in the haemoglobin beta-chain.

Abnormal haemoglobin ultimately leads to anaemia as well as other symptoms, depending on the exact mutations present. Diseases caused by defective haemoglobin fall into a larger category of diseases known as the "haemoglobinopathies" which also include the thalassemias, a related group of diseases that are characterised by reduced or deficient rather than abnormal haemoglobin. 

Sickle cell disease affects approximately 100,000 individuals in the USA and more than 3 million worldwide.
Official title:
A Phase 1/2, Open-Label, Multicenter, Single-Arm Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease


Medical Monitor

Phone: 510-307-7266



United States, California

UCSF Benioff Children's Hospital, Oakland, California, United States, 94609

University of California Davis Comprehensive Cancer Center, Sacramento, California, United States, 95817


United States, Georgia

Children's Healthcare of Atlanta, Atlanta, Georgia, United States, 30322


United States, Maryland

Investigational Site Number 101, Bethesda, Maryland, United States, 20892


United States, Michigan

Karmanos Cancer Institute, Detroit, Michigan, United States, 48201


United States, Pennsylvania

Children's Hospital of Philadelphia_Investigational site number 108, Philadelphia, Pennsylvania, United States, 19104


Study start:
Mar. 6, 2019
8 participants
Gene editing method:
ZNF-Zinc Finger Nucleases
Type of edit:
Gene disruption
BAF chromatin remodelling complex subunit (BCL11A)
Delivery method:
Non-viral - Ex-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


This is an open label, multicenter, Phase 1/2 study in approximately eight adults with severe Sickle Cell Disease (SCD). The study will evaluate the safety, tolerability, and efficacy of autologous haematopoietic stem cell transplantation using SAR445136 (formerly known as BIVV003).

Participants will receive plerixafor as subcutaneous (SQ) administration followed by myeloablative conditioning therapy with intravenous (IV) busulfan. SAR445136 will then be administered as a 1-time IV infusion of autologous Cluster of Differentiation 34 + Haematopoietic Stem/Progenitor Cell (CD34+HSPC) transfected ex vivo with zinc finger nuclease (ZFN) messenger ribonucleic acid (mRNAs) targeting the B-cell lymphoma/leukemia 11A (BCL11A) locus.


  • Biological: Plerixafor
  • Drug: Busulfan
  • Genetic: SAR445136
Last updated: Feb. 27, 2024
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