Preclinical and CMC Services for AAV-based Therapies

Pharmaron’s team has more than a decade of experience supporting the development and approval of ocular therapies. Services include preclinical testing (efficacy, PK and biodistribution, safety and GLP toxicology studies), CMC development and manufacturing support for clinical trials. Our ocular experience in the preclinical and CMC disciplines spans the full drug development lifecycle, including IND, IMPD, BLA submissions and post market support.

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Download Whitepaper: NAb-bed Again: Challenges in Developing Neutralizing and Anti-drug Antibody Assays for Gene Therapy 

Services

• Preclinical study design, data acquisition
• Efficacy, biodistribution, GLP tox study execution and interpretation
• Authoring preclinical Study reports for regulatory submissions dossiers supporting clinical and market approval (US, EU and global)
• Supply of drug substance for preclinical, toxicology and clinical studies at a range of scales
• Analytical and product characterisation services
• Bioassay development and testing
• Authoring CMC sections for regulatory submission dossiers for clinical and market approval (US, EU and global)

Preclinical Capabilities

• Broad range of disease models in different species, including wet AMD, inherited retinal disorders, diabetic retinopathy, glaucoma
• Expertise across all routes of administration
• Access to expert ocular scientists and board certified veterinary ophthalmologists
• Experience collecting comprehensive tissue list for biodistribution
• Functional and structural evaluation of critical endpoints

CMC Capabilities

• 80,000 ft² state-of-theart development and manufacturing
• Platform manufacturing process up to 500L
• Scalable suspension culture systems
• Chromatography based purification with focus on high purity for ocular administration
• High throughput process development
• Suite of analytics
• Best-in-class product characterisation of ocular targets

Learn more about Pharmaron’s expertise by registering for our monthly CGT webinar series, which is focused on identifying key cell and gene therapy questions that arise during drug discovery and development. Register here.