Cell therapy uses living cells as a drug to treat diseases. In cell therapy, genetically modified or un-modified viable cells are injected into a patient to treat various diseases. GenScript offers END-TO-END SOLUTION to accelerate your journey from discovery to clinical research and commercial manufacturing by providing high-quality services, reagents, and instruments.

Cell Therapy Development Workflow

Lead Generation & Optimization 2

Target Discovery

Target Identification: Target identification and characterization begins with identifying the function of a possible therapeutic target (gene/protein) and its role in the disease.

Understanding the disease: Genetics plays a crucial role in understanding the origin of a disease and designing a pathway for treatment. Target genes involved in specific diseases can be identified by bioinformatics approaches and further validated using target enrichment NGS technology, CRISPR gRNA Libraries, qPCR, and other tools.

Target Gene Validation: Target validation ensures the engagement of the target in the progression of a disease and indicates that the target has potential therapeutic benefits. To validate targets, researchers study genomic sequences and modifications, generate cell-based and phenotypic models, and use techniques such as immunohistochemistry (IHC), fluorescence-activated cell sorting (FACS), western blotting, and other antibody and recombinant protein-based assays.

Lead Generation & Optimization

Lead Generation and Optimization: Lead optimization is a critical stage in drug discovery. The goal of this stage is to identify promising leads for the therapeutic agent and extensively optimize, in parallel, both the biological activity and the properties through in vitro screening and assays. In addition, to develop the candidate's safety profile, it is essential to advance a clear understanding of the mode of action, nature of the target and establish the biological relevance to qualify it to be an effective drug. In lead discovery for CAR T-cell therapy, upon identification of tumor antigens, the antibody recognizing the tumor antigens is produced as part of the process. The function of CAR can be significantly improved and toxicity reduced if the recognition of the tumor antigen by the antibody module is optimized and made more specific with greater binding affinity.

Preclinical Study

After target identification and lead optimization, identified leads are ready for preclinical validation and even downstream production, and the preclinical phase of drug development begins.

Preclinical data and models are necessary for two reasons:
1) To build confidence that the approach has some merit.
2) To understand the mechanism of action of the therapy.

During this stage, potential therapies are tested in vivo and ex vivo, including in animal models. Manufacturing information (CMC), clinical protocols, efficacy, toxicity, pharmacokinetic (PK) information, and side effects of the therapeutic are closely monitored and recorded for IND application.

Clinical Development

During this stage, the drug is produced in stage-appropriate cGMP settings and tested in human clinical trials involving a series of rigorous tests. Efficacy and side effects of the therapeutic are closely monitored and recorded. Treatment may be halted at any time if serious side effects are found. In parallel, a commercial manufacturing process is developed to meet market needs. Finally, when regulatory agencies approve a therapy, commercial scale manufacturing begins.

Manufacturing high quality, consistent, and effective cell therapy products depends on obtaining high-quality reagents from established manufacturing partners with supply chain security. GenScript offers various grades (RUO, GMP, etc.) of reagents to support every step of your cell therapy research and commercialization process.

Solutions Advantage

Experienced service and Support teams

GMP facilities and Manufacturing excellence

Quality and Regulatory Compliance

Flexibility – End-to-End CRO and CDMO Service

Cell Therapy Resources

Explore our technologies and learn more about Cell Therapy Solution. Find the information and resources you need by browsing through our educational material.

Explore our technologies and learn more about Cell Therapy . Find the information and resources you need by browsing through our educational webinar.

Alexander Marson, MD, PhD
Reprogramming Human T Cells with CRISPR

Alexander Marson, MD, PhD
Director, Gladstone-UCSF Institute of Genomic Immunology

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Anna Pasetto, PhD, Assistant Professor
TCR targets validation through T cell activation

Anna Pasetto, PhD, Assistant Professor
Managing director of pre-GMP facility
Department of Laboratory Medicine, Karolinska Institutet

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Joshua Burgess, PhD
Identification and Characterization of a Novel Biomarker for Cancer Therapy

Joshua Burgess, PhD
Advance Queensland Early Career Research Fellow of Translational Research Institute, Queensland University of Technology

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JJ Joseph Melenhorst, PhD
Response to Second Generation CAR T Cell Therapy: It takes (at least) Two to Tango

J Joseph Melenhorst, PhD
Professor, Pathology and Laboratory Medicine, University of Pennsylvania and Director, Biomarker Program, Parker Institute for Cancer Immunotherapy, UPenn

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Ramarao Vepachedu, PhD
CRISPR/Cas9-based genome editing for autologous CAR-T cell production

Ramarao Vepachedu, PhD
Development Scientist IV, Leidos Biomedical Research, Inc

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Matthew Porteus, MD, PhD
Targeted Integration in Stem Cells

Matthew Porteus, MD, PhD
Professor, Stanford University, School of Medicine

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Shondra M. Pruett-Miller, PhD
Operationalizing Genome Editing Across a Broad Range of Genomic and Cellular Targets

Shondra M. Pruett-Miller, PhD
Director, St. Jude Children's Research Hospital Comprehensive Cancer Center

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