Preclinical Studies
CGT Preclinical Studies
In drug development, preclinical studies assess the preliminary safety and efficacy of new drug products and guide clinical trial design about biologically active dose levels, route of administration, patient eligibility criteria, and physiologically relevant endpoints. This poses several challenges for CGT products, including highly specialized dose routes, a lack of relevant animal models, and species-specific immunogenicity.
The in vivo capabilities at Pharmaron have been tailored to address these challenges and characterize the product-specific safety and efficacy of CGT products to assist project teams in decision-making on key milestones.
Capabilities
In Vivo Pharmacology Studies
Pharmaron provides specialized pharmacology support for CGT programs, focusing on key endpoints such as
- Drug distribution/persistence, viral shedding
- Cell fate dynamics
- Gene expression & biomarker expression
- Disease or tumor models
Driving Innovation in Preclinical CNS Research
Pharmaron empowers the development of cutting-edge CGT therapies for neurological disorders through a comprehensive suite of specialized capabilities. Our team leverages advanced neurodegenerative disease models, precise stereotaxic injections, and robust neurobehavioral assessments to deliver actionable insights. Combined with functional readouts and state-of-the-art bioanalytical assays, we provide end-to-end, compliant preclinical evaluations that accelerate your path from discovery to clinical translation.
Nonclinical Safety Assessment / Toxicology Studies
Pharmaron provides IND-enabling CGT studies with comprehensive evaluation of potential toxicities and systemic effects, including immunogenicity, histopathology, and on/off-target effects. Using FDA-recommended disease and injury models, we ensure biological relevance, often integrating safety parameters into pharmacology studies. Our CGT experts have deep experience across ocular, CNS, cardiovascular, and metabolic indications, supported by state-of-the-art facilities for imaging, bioanalysis, and partnerships in clinical pathology and histopathology.
Therapeutic Area Expertise – Ophthalmology
Pharmaron advances CGT products for eye disorders with specialized ocular disease models, multimodal imaging, and bioanalytical assays, supported by board-certified veterinary ophthalmologists to deliver comprehensive, compliant efficacy assessments.
Regulatory Affairs and Submission Support
Comprehensive guidance for cell and gene therapy programs, including strategy, preparation, and submission of regulatory dossiers (INDs, BLAs, and global filings) to accelerate development and ensure compliance with FDA, EMA, NMPA, and other health regulatory authorities.
Contact Our Team
Request more information about our drug discovery, preclinical and clinical development services
