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Cell therapy refers to the use of living cells as therapeutic agents to repair, replace, or regulate biological functions in the body. This broad field encompasses a wide spectrum of approaches: from established procedures such as hematopoietic stem cell transplantation to engineered immune cell therapies like CAR T and CAR NK cells, gene-edited hematopoietic or pluripotent stem cells, and regenerative applications using mesenchymal stromal cells (MSCs) or pluripotent stem cell-derived lineages.

While the specific cell type, engineering strategy, and manufacturing process may vary, all cell therapies share the goal of restoring or enhancing function through the precise delivery of living cells. These diverse approaches represent one of the most dynamic areas of translational medicine—the bridging of basic cell biology, genetic engineering, and advanced manufacturing to create “living drugs” with transformative clinical potential.

A wide range of cell types are used in cell and gene therapy, each selected for their unique biological properties and therapeutic potential:

• Immune cells (e.g. T cells, NK cells, and dendritic cells) are frequently modified to enhance antigen recognition, persistence, or cytotoxic activity, forming the basis of engineered immunotherapies like chimeric antigen receptor (CAR) T cells or T cell receptor-engineered T (TCR-T) cells.
• Hematopoietic stem and progenitor cells (HSPCs) serve as self-renewing vehicles for gene addition or correction, enabling sustained, long-term correction of inherited blood and immune disorders.
• Human pluripotent stem cells (hPSCs) offer a renewable platform for generating defined, functional cell types—including cardiomyocytes, pancreatic islet cells, and neural progenitors—for regenerative applications or disease modeling.
• Mesenchymal stromal cells (MSCs) and their extracellular vesicles (EVs) are being studied for their immunomodulatory and trophic effects in tissue repair and inflammation.

Collectively, these cell types form the foundation of modern cell and gene therapy development, supporting both autologous and allogeneic treatment paradigms across a growing range of clinical indications.

As the field transitions from discovery to commercialization, researchers face complex challenges across both the scientific and operational spectrum:

• Scalability and reproducibility: Developing robust, standardized culture and expansion systems that maintain cell identity and potency.
• Genetic and phenotypic stability: Minimizing variability introduced during long-term culture or genome editing.
• Regulatory compliance: Ensuring that ancillary materials, media, and reagents meet relevant cGMP and Quality by Design (QbD) standards.
• Cost and manufacturing logistics: Addressing the time and expense associated with autologous workflows and scaling allogeneic platforms.
• Process integration: Bridging discovery research, process development, and clinical manufacturing within defined, closed systems.

Overcoming these challenges requires coordinated use of high-compliance reagents, validated protocols, and scalable manufacturing solutions, supported by strong documentation and regulatory expertise.

Gene editing technologies such as CRISPR-Cas9, TALENs, and base or prime editors are transforming cell therapy by enabling precise, stable modifications that improve efficacy and safety. These tools are used to correct genetic defects, enhance persistence or targeting in immune cells, and develop universal or hypoimmunogenic allogeneic products. Integration of gene editing requires compatible, xeno-free culture systems and validated reagents to preserve cell quality, genetic integrity, and regulatory traceability.

Reliable progress in cell and gene therapy depends on standardized reagents and reproducible workflows. Key components include:

• Animal origin-free, cGMP-compliant media (e.g. TeSR™-AOF, StemSpan™-AOF, ImmunoCult™-XF) for culture, maintenance, and expansion.
• Passaging, activation, and differentiation reagents designed for high compliance and scalability.
• Validated cryopreservation media (e.g. CryoStor® CS10) for consistent cell recovery and function.
• Analytical tools for cell identity, potency, and genomic stability assessment.

�����ƽ�� Technologies supports these needs with well-characterized reagents, complete documentation, and technical expertise through our Services for Cell Therapy Program.

Scalable cell therapy manufacturing requires bioreactor systems that support consistent, high-quality cell expansion while maintaining the functional integrity of sensitive cell types. Systems that provide gentle mixing, precise environmental control, and process scalability are best suited for this purpose. For example, PBS-MINI Bioreactors are designed to minimize shear stress with a Vertical-Wheel™ impeller design, an essential factor for maintaining the viability and pluripotency of shear-sensitive cells such as human pluripotent stem cells (hPSCs).

Building compliance into early development reduces risk later. Using reagents manufactured under relevant cGMPs and following standards for ancillary materials—such as USP <1043>, ISO 20399, and ICH Q7—simplifies regulatory submissions. �����ƽ�� provides detailed Certificates of Analysis, traceability documentation, and change notifications to support IND and CTA filings, along with expert guidance on reagent qualification and quality assurance.