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

Phase 1/2 Completed 7 enrolled Sangamo Therapeutics

NaSingle-groupOpen-labelTreatment

Summary

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 hematopoietic stem cell transplantation using BIVV003.

Timeline

Start: 2019-03-06
Primary completion: 2025-07-17
Completion: 2025-07-17

Publications

Brusson M, Miccio A. Genome editing approaches to beta-hemoglobinopathies. Prog Mol Biol Transl Sci. 2021;182:153-183. doi: 10.1016/bs.pmbts.2021.01.025. Epub 2021 Mar 1.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	BIVV003	Cell therapy	—	Intravenous
Background	Busulfan	Small molecule	—	Intravenous
Background	Plerixafor	Small molecule	—	Subcutaneous

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

Summary

Timeline

Publications

Drugs

Indications