Cell & Gene Therapies

Extensive CAR T-cell and other cell-based therapy experience and technologies to enable early product screening and future development

• Molecular biology and imaging technologies for assessing novel engineered adeno-associated viruses (AAVs) and other viral capsids
• On-site gene-modified cell therapy production and advanced in vitro services by flow cytometry, IHC, ddPCR, MSD
• Multiple-modality imaging capabilities
  
• More than 300 solid tumor and hematologic malignancy models (more than 100 luciferase expressing)
• Combinatorial immunosuppressant therapy studies
• AAV Capsid-directed evolution strategies for evaluating diverse capsid libraries and variants with desired tropism in in vivo models
• Ocular safety assessment, PK, and efficacy studies in partnership with OSODopens in a new tab

Comprehensive safety toxicology, tumorigenicity, biodistribution, analytical testing and data support for investigational new drug (IND)/investigational medicinal product dossier/clinical trial application submissions

• Unique animal models requiring complex methods and routes of administration
• Large-and small-animal model stereotaxic delivery (e.g., magnetic resonance imaging, intracranial volume)
• Phase-appropriate assay development for chemistry, manufacturing and control (CMC) regulatory expectations
• Emerging pluripotent-derived cell experience
• Key therapeutic area support (e.g., ocular, neurodegenerative and oncology)

Decision-enabling data to support the preparation and filing of biologics license application/marketing authorization application and other submissions

• Proprietary data set with unique insights from diagnostic test data, clinical trial data and direct patient access
• Comprehensive analytical and biomarker testing, including bespoke assay development, validation and implementation through a global laboratory network
• CMC assay development, optimization and validation
• Central analytical lab to remove risk of analytical variation or bias from multiple CDMO/analytical partners—from drug development to commercialization to enable bespoke, comprehensive and independent assessment of product quality

Full-service testing support for extended long-term follow-up

• Parallel development of therapeutic and diagnostic and CDx commercialization
• Decentralized laboratory sample collection solutions to decrease patient burden and optimize engagement
• CMC commercial batch release of marketed products