This document outlines a general strategy for drug substance and/or drug product manufacturing processes expected to undergo process performance qualification with considerations unique to gene therapy. It addresses the specific requirements of the gene therapy industry, as general validation templates from the biologics industry may not be entirely applicable.
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This presentation, developed by the BioPhorum Validation team reviews the regulatory challenges faced in cell and gene therapy with a focus to identify and use appropriate analytical controls when dealing with both limited regulatory guidance and limited materials.
This paper evaluates the applicability of the current understanding of viral clearance to the products and processes employed in CGT manufacture to assure the viral safety of these therapies. Unique considerations for preventing contamination through raw material risk mitigation are highlighted. Guidance on viral clearance strategies for inactivation and removal are also provided. This guidance focuses on adeno-associated viral vectors but may also apply to other viral vectors.
A BioPhorum member only survey to understand viral purification and clearance practices member companies are undertaking in CGT.
A BioPhorum member only survey is to gather information to benchmark what analytical controls member companies are currently using to establish methods to test identity, strength, potency and purity.
A BioPhorum member only benchmarking survey to understand the current challenges and strategies undertaking regarding leveraging process characterization data/use of platforms to accelerate validation.
Commentary provided to ‘Chemistry Today’ following an interview with one of the lead authors of the BioPhorum CG&T Validation challenges publication. This article highlights, from the lead authors perspective, the unique challenges and possible solutions when attempting to validate cell and gene therapy products.
This commentary highlights the published work from the BioPhorum Validation workstream indicating the unique challenges and possible solutions when attempting to validate cell and gene therapy products.
CGT considerations when assigning responsibilities during a product transfer between a sponsor and a contract development manufacturing organization
With over 1000 CGT developers worldwide, the FDA expects to approve 10-20 CGT products per year annually from the year 2025. Based on this information, there is an industry emphasis on streamlining the technology transfer process for new products into CDMOs. There are many considerations that are unique to CGT and if discussed early in the NPI process, could help reduce the risk of unplanned disruptions and align expectations in the early stages. This paper presents a common understanding and proposed ownership of these unique aspects, developed via a consensus of CGT sponsor (client) and CDMO participants in the Validation workstream of BioPhorum Cell and Gene Therapy. These recommendations could serve well as pre-established expectations or as an agenda for an NPI–kickoff meeting upon the initiation of a CGT sponsor CDMO relationship.They could also be used to support master supply agreement and quality agreement discussions.