Hematopoietic Cell Therapy

The field of hematology has been at the forefront of regenerative medicine for more than six decades. Emerging from the pioneering bone marrow transplants of the 1950s and 60s, hematopoietic stem cell transplantation (HSCT) has since become the standard of care for numerous hematological disorders and malignancies. The application of CD34+ cells in translational workflows continues to expand, bringing new possibilities for curative cell and gene therapies.

The emergence of FDA-approved autologous gene therapies such as Casgevy and Lyfgenia have signaled a new era for hematopoietic cell therapy, and the field continues to push beyond conventional transplantation toward increasingly precise, scalable, and transformative therapeutic approaches. High manufacturing costs and complex logistics remain key challenges, driving researchers to pursue innovative strategies such as ex vivo-expanded universal donor cells, hypoimmune induced pluripotent stem cell (iPSC)-derived hematopoietic stem and progenitor cells (HSPCs), and even in vivo gene editing (the goal being to eliminate the need for transplantation entirely). The realization of these next-generation therapies will depend on the advancement of scalable gene-editing workflows, reliable cGMP-compliant media, robust ex vivo expansion methods, and potency assays that provide meaningful measures of HSPC function.

The scientific resources below have been curated to support you through the key stages of hematopoietic cell therapy development and manufacturing, including sourcing, expanding, and gene-editing HSPCs, tools and assay selection, regulatory compliance pathway navigation, and more.

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