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.
NEW! Free On-Demand Course: Application of HSPCs in Cell Therapy
From the fundamentals of hematological disease progression and hematopoietic cell therapy products, to key workflow and compliance considerations, you’ll learn best practices for applying HSPCs in cell therapy development and gain insight into the unique challenges and opportunities. Whether you're already working with HSPCs in translational workflows or just getting started, this self-paced virtual course will help move your HSPC-based cell therapy closer to the clinic.
Register Now >- New Tools for the Ex Vivo Expansion of Human Hematopoietic Stem and Progenitor CellsThe development of standardized ex vivo HSPC expansion methods is needed to generate clinically relevant cell numbers for transplantation. This webinar will review key concepts in human HSPC biology, including standardized methods and recently demonstrated strategies for expanding HSPCs in culture. Additionally, º£½ÇÆƽâ°æ products for the entire workflow of HSPC expansion will be covered, from isolation to expansion and differentiation, and analysis.
- How to Use Primary Cells to Develop the Next TherapyHuman primary cells are cells isolated directly from tissues, including blood and bone marrow. The use of human primary cells increases the physiological relevance of cell culture systems, enabling you to generate meaningful data that is more predictive of in vivo outcomes. Learn about the advantages of starting with human primary cells for your immunology and cell therapy research workflows. Key considerations for choosing the ideal starting material, donor characterization, regulatory challenges, and tips to streamline your cell-based assays with human primary and cultured cells are also covered.
- The CFU Assay: In Vitro Functional Potency Assessment for Hematopoietic Stem and Progenitor CellsThe colony-forming unit (CFU) assay remains the only in vitro assay that provides a correlative assessment of hematopoietic stem and progenitor cell (HSPC) engraftment potential in vivo. For many years, regulatory bodies have recommended functional assessment and the CFU assay for labs working with cellular therapies or transplantable products derived from HSPCs. Recent updates from regulatory bodies such as FACT underscore the crucial role of the CFU assay in these workflows, particularly after any processing step, such as cryopreservation, that may impact the functional properties of HSPC products. <br><br> In this webinar, Selena Hallahan introduces the utility of the CFU assay for functional assessment of hematopoietic cell products and º£½ÇÆƽâ°æ Technologies’ comprehensive portfolio of reagents, training, and services designed to support high-performing and standardized workflows for hematopoietic cell therapy products.
- Hematopoietic Stem Cell Fitness and Function During Sickle Cell DiseaseChronic insults, such as inflammation and replicative stress, impair and exhaust blood-sustaining hematopoietic stem cells (HSCs), leading to dysfunction and selection for leukemia-associated mutations. Dr. McKinney-Freeman’s laboratory is currently studying how sickle cell disease (SCD), an inherited hemolytic anemia with a large inflammatory component and increased hematopoietic demand, compromises the fidelity and function of hematopoietic stem cells (HSCs) in both mice and individuals with SCD. Mounting evidence indicates that SCD patients may experience enhanced rates of clonal hematopoiesis, as well as MDS (myelodysplastic syndrome) and AML (acute myeloid leukemia), in general, and following allogeneic HSC transplantation or autologous HSC gene therapy. Considering that these are the only curative therapies for SCD, it is important to better understand and prevent SCD-induced insults to HSCs and their microenvironment. <br><br> In this webinar, Dr. McKinney-Freeman from St. Jude Children’s Research Hospital describes, in detail, what her laboratory has learned about how SCD affects HSCs. Additionally, Dr. McKinney-Freeman is joined by Dr. Eric Norris, Account Executive, Cell Culture, º£½ÇÆƽâ°æ Technologies in the Q&A session.
- Implementing the Colony-Forming Unit (CFU) Assay As a Potency Assay for Hematopoietic Cell Therapy ProductsAssessing the functional potency of cells for use in cell therapy research presents unique challenges. For hematopoietic stem and progenitor cells (HSPCs) in particular, potency can be measured in vitro by assessing the ability of these cells to differentiate into progenitor cells using the colony-forming unit (CFU) assay. Validating the CFU assay as a potency assay requires demonstrating its specificity, accuracy, precision, linearity, and reproducibility. Once validated, the CFU assay can assess the quality and consistency of prospective hematopoietic cell therapy products (HCTPs) at multiple stages of the processing and manufacturing workflow. <br><br> Join Dr. Colin Hammond and learn about the regulatory guidance around the potency testing of HCTPs and how to validate the CFU assay as a potency assay that can be integrated into cell therapy manufacturing workflows.
- Tools for Optimizing Human Immune Cell ResearchObtaining consistent and reliable results when culturing immune cells can be challenging. Watch this webinar to discover how to obtain high yields of functional T cells, NK cells, B cells, dendritic cells, and macrophages for your research applications. The speaker, Evan Karas, also explains how to expand primary T cells without feeders or serum.
- Determining the Potency & Stability of Hematopoietic CellsIn this webinar, Dr. Jackie Damen of º£½ÇÆƽâ°æ Technologies describes the effects of cryopreservation protocols on the engraftment potential of hematopoietic cells and the need for standardized potency assays, including the Colony-Forming Unit (CFU) assay, for hematopoietic cell products.
