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.
Cell Engineering Optimization
Non Viral Service and reagent
Viral: Service and reagent
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.
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.
Cell Selection/ Isolation
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
Director, Gladstone-UCSF Institute of Genomic Immunology
Gal Cafri, PhD
Immunotherapy and Genetic Engineering Group Leader, Sheba Medical Center
Anna Pasetto, PhD, Assistant Professor
Managing director of pre-GMP facility
Department of Laboratory Medicine, Karolinska Institutet
Joshua Burgess, PhD
Advance Queensland Early Career Research Fellow of Translational Research Institute, Queensland University of Technology
J Joseph Melenhorst, PhD
Professor, Pathology and Laboratory Medicine, University of Pennsylvania and Director, Biomarker Program, Parker Institute for Cancer Immunotherapy, UPenn
Ramarao Vepachedu, PhD
Development Scientist IV, Leidos Biomedical Research, Inc
Matthew Porteus, MD, PhD
Professor, Stanford University, School of Medicine
Shondra M. Pruett-Miller, PhD
Director, St. Jude Children's Research Hospital Comprehensive Cancer Center
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