Course Description:
A number of gene-based cell therapies are in development with several products already in commercial phase. Human primary cells and tissues, plasmids and vectors represent raw materials of biological origin and as such cannot be subjected to sterilization steps during their manufacture or processing. Crucial quality considerations which can impact product consistency, potency, carry the risk of adventitious agents or present supply chain challenges that must be addressed through phase appropriate good manufacturing practices for these complex biological processes. For example, key quality considerations for vectors include high packaging capacity, stable gene expression and low immunogenicity. Above all the vectors must be deemed safe and demonstrate the absence of replication competent virus for viral vectors. Under 21 CFR 312.23 (a) (7) (i) sponsors must show, in their IND filing, a section listing all components used in the manufacturing of the gene therapy product. This information includes a detailed description of the manufacturing process, all components including vector, master cell bank, master viral bank and reagents. Description, history and details on the derivation of the vector and plasmid construct including the sequence analysis must be included. Guidance issued by a number of regulatory bodies (FDA, EU) and standard setting agencies (USP, ISO, ICH) have provided developers with phase appropriate CMC guidance in selecting those starting materials for human gene therapy Investigational New Drug Applications. Selecting a Contract Development and Manufacturing Organization (CDMO) that offers the experience necessary for high quality vector and plasmid production services becomes an essential part of early product development. The vendor qualification process should include a comprehensive quality audit conducted onsite or in the age of COVID-19, where the opportunity of an onsite audit may be limited, a virtual or remote audit needs to be considered. Thus, critical materials management adds a necessary complexity to the development of investigational protocols. Risk analysis and mitigation during early development planning can ensure the safety of subjects in gene based clinical investigations and improve chances for the commercial viability of these promising therapeutic modalities.
The live version of this recording took place on April 12, 2021 and features
a presentation by Gary du Moulin, Ph.D., MPH
Who should participate:
- Analytical chemists
- QA/QC analysts
- R&D scientists, managers
- Team members in CMC development projects
- Manufacturing scientists, managers
- Regulatory affairs specialists
- Contract research organizations
- Contract manufacturing organizations
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