End-To-End, Fully Automated Manufacturing of Hematopoietic Stem and Progenitor Cell Therapy Via CCR5-Targeted Integration

An automated, high-throughput platform produced CCR5-targeted anti-HIV antibody knock-in gene-edited HSPCs with high purity and editing efficiencies comparable to small-scale processing.

• Why it matters: Manual HSPC gene-therapy manufacturing is costly and hard to scale, limiting patient access, so an end-to-end clinical-scale workflow is needed.
• What they did: The platform ran all steps from post-thaw inoculation through Cas9 electroporation, AAV6 transduction, and harvest to generate CCR5-locus geHSPCs.
• The result: After transplantation into mice, geHSPCs engrafted and reconstituted multiple hematopoietic lineages, indicating preserved repopulation capacity and supporting scalable manufacturing.

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