ultra-HSC lentiviral vector

Ultra-HSC is an engineered lentiviral vector (LVV) platform developed by Syenex for the efficient transduction of primary, resting human CD34+ hematopoietic stem cells (HSCs). Conventional lentiviral vectors typically require HSCs to be stimulated or activated to achieve efficient gene delivery, a process that can alter the cells' phenotype and reduce their long-term stemness. Ultra-HSC overcomes this by utilizing a library of engineered pseudotypes, including specific affinity reagents and fusogens, that target proteins on the surface of quiescent HSCs. This allows for high-efficiency delivery of genetic cargo and large protein payloads, such as Cas9/gRNA ribonucleoproteins, without the need for chemical enhancers or cell activation. The platform is intended to improve the manufacturing and efficacy of HSC-based gene therapies for inborn genetic disorders.