GlaxoSmithKline Intellectual Property Development Limited
发明人:
Chaminda Salgado
申请号：
GB201704968
公开号：
GB2560933A
申请日:
2017.03.28
申请国别(地区):
GB
年份:
2018
代理人:
摘要：
The invention relates to a composition comprising a high concentration of transduced cells, in particular transduced stem cells for use in methods of gene therapy. The composition comprises transduced cells, particularly haematopoietic stem cells, at a concentration of at least 0.5 x 108 cells/mL. The cells may be transduced with a lentiviral vector and may contain a transgene encoding human beta globin. The compositions may comprise a cryoprotectant. Methods for preparing said transduced cell compositions are also disclosed, wherein the methods can involve suspending transduced cells at a concentration of least 0.5 x 108 cells/mL, optionally in a cryoformulation comprising DMSO, and optionally HSA and/or saline. The compositions can be administered to a patient in methods of gene therapy.
