EXTERNAL-RESOURCE

Proposed solutions for cell and gene therapy validation challenges

27 October 2022
Advanced Therapy Medicinal Products
Proposed solutions for cell and gene therapy validation challenges

Cell and gene therapy (CGT) products are a class of advanced therapeutics that have tremendous potential to treat diseases. However, their manufacturing challenges are complex and successful commercialization of CGT products requires a multidisciplinary approach that integrates patient needs and product knowledge with the capability to commercially manufacture these complex products consistently and reliably.

The basic principles of current good manufacturing practices and general process validation concepts and practices apply to CGT manufacturing processes and analytical methods; however, following validation, concepts established for general biologics can result in challenges for CGT product validation due to their unique characteristics. Therefore, CGT validation requirements are still in development. Specific challenges in validating CGT product manufacturing processes include identifying quality attributes, establishing specifications, and validating analytical methods.

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.

ICH Guidance Information
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Helping the ICH refine its guidance on analytical procedures
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Now you can have a simple, compliant, and valuable equipment revalidation strategy
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Facing challenges on CGT method validation controls? Read our guide
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BioPhorum introduces – CGT considerations within product transfer and validation
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How to use a phase-appropriate approach for assay validation in CGTs

Advanced Therapy Medicinal Products

Advanced Therapy Medicinal Products support the quest for better and faster development of cell, gene and RNA therapies​ through connecting therapy developers, contract manufacturing, and testing organizations to gain an understanding and respond to the challenges faced by the advanced therapeutics industry.

Development Group

Development Group accelerates and improves the development process of biopharmaceutical medicines for the benefit of the patient. Replacing isolation with collaboration by providing a “safe” space where subject matter experts can work on sharing solutions and best practice on emerging industry trends, implementation of new technologies and common issues whilst ensuring their intellectual property and confidentiality is protected.

Drug Substance

Drug Substance advances excellence in drug substance manufacturing and testing, acting as an industry voice to accelerate technology adoption, implement post-approval change more rapidly and increase confidence in maintaining compliance.

Fill Finish

Fill Finish accelerates development and acceptance of sustainable world-class filling and packaging operations for drug product which meets the future needs of patients. Through innovative solutions resulting from the sharing of expertise, we can overcome common performance challenges and deliver on quality and regulatory compliance.

Information Technology

Information Technology accelerates digital maturity across the ​global pharmaceutical manufacturing industry.

Quality

BioPhorum Quality provides a dedicated, safe space for quality professionals from biomanufacturers and contract organizations to coalesce and enhance effective collaboration to address shared industry challenges.

Regulatory-CMC

Regulatory CMC brings leaders together who have a common goal – to improve access to medicines through innovation in the Regulatory ecosystem. It provides a dedicated space for strategic leadership and a coordinated industry voice, to move from divergence to convergence.

Supply Chain to Patient

Supply Chain to Patient transforms the performance of global pharmaceutical clinical and commercial outbound supply chains through industry collaboration by creating transparent patient-centric, resilient and agile E2E supply chains, that consistently deliver high-quality medicines ensuring an uninterrupted supply to patients.

Supply Partner

Supply Partner is a trusted advisory group and collaboration that is the engine for driving change across the global industry for all things relevant to the inbound supply chain. Addressing the challenges of complexity, resilience, cost, sustainability, and innovation.

Sustainability

Sustainability enables the industry’s transition to a low carbon, circular future – supporting members to improve patient health while respecting the planet. We activate multi-disciplinary teams drawn from our network to deliver environmental sustainability improvements across the value chain.

Technology Strategy

Technology Strategy acts as one voice of the industry to define strategy, accelerate technology transformation and support technology translation and adoption. With a purpose of enabling the future state of flexible, resilient, sustainable end-to-end biomanufacturing.

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An industry perspective on understanding AAV capsid content variants
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EXTERNAL-RESOURCE
An intercompany perspective on practical experiences of predicting, optimizing and analyzing high concentration biologic therapeutic formulations
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The Drug Substance 2.0 Strategic Value Framework
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BioPhorum’s holistic approach to container closure integrity
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Digital Plant Maturity Model 3.0
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Inaugural BioPhorum Quality face to face – get involved
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The next step in building regulatory as a capability to enable strategic innovation across the BioPhorum community
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Delivering a Cell and Gene Therapy Outbound Supply Chain Pathway for the Industry
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A vision for the biopharmaceutical industry’s inbound supply chain
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A major milestone – a harmonized approach to product carbon footprint data
Technology Roadmap V 2.0
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BioPhorum Technology Roadmapping roadmap vision 2.0

Publications

Documents including best practices, vision statements and peer reviewed papers to help you take value back to your business.

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Discussing all aspects of the biopharmaceutical industry, from digitization and knowledge management to raw materials, supply chain, sustainability and more. Learn from the perspectives of those at the forefront of the industry.

Podcasts

BioPhorum Connect is our podcast series that keeps you up to date with the latest news and trends in the biopharmaceutical industry. From experienced professionals to thought leaders, these podcasts bring you the insights and perspectives of experts from around the world.

Tools

A variety of resources including design tools and user requirement specifications to help you improve and streamline your business processes.

Benchmarks and Surveys

Research conducted among the BioPhorum membership providing evidence to support change and identify best practice.

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WEBINAR
Bioreactivity testing in single-use system biomanufacturing
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Bioreactivity testing in single-use system biomanufacturing
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Bioreactivity testing in single-use system biomanufacturing
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Digital Plant Maturity Model 3.0
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Bioreactivity testing in single-use system biomanufacturing