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How to speed up the delivery of CGT therapies

4 March 2024
Advanced Therapy Medicinal Products
Kathleen O’Hagan
Global Change Facilitator

An increasing number of cell and gene therapies (CGTs), with diverse delivery vehicles and routes of administration, are being approved by regulatory authorities to address a variety of diseases. Many more products are being developed, and the number is expected to grow. However, the CGT industry faces many challenges, including the restricted availability of materials, a limited product understanding and analytics, and a high cost of goods.

To address some of these, there is a strong desire to create common approaches to manufacture novel therapies, which in some cases only differ by their genetic payload. Such ‘platforming’ approaches require a good understanding of areas such as regulatory requirements, manufacturing facilities and equipment design, and production process design across therapeutic technology platforms.

Yet despite the interest in such approaches and some initiatives in this area, there is currently no available guidance or best practice to help CGT developers.

To fill this gap, we have published Leveraging platform and process characterization data to accelerate CGT validation and commercialization .

In this paper, CGT companies from BioPhorum have evaluated and summarized the opportunities to leverage process development and characterization knowledge across similar therapeutic technology platforms. The aim is to accelerate process validation activities using experience gained with similar product types. The evaluation focuses on manufacturing and covers both cell and gene therapies.

“CGT process characterization/validation timelines are challenging due to their relative uniqueness,” said Sanjay Nilapwar, Principal Scientist I, Purification Development at Abbvie. “There is an untapped opportunity to leverage data across projects; however, there is currently no best practice for CGT platforms. This whitepaper summarizes strategies that will be highly beneficial for organizations looking to accelerate PC/PV and will be very useful for accelerating the drug commercialization cycle.”

The paper covers regulatory and business considerations, what a process platform is, phase-appropriate process development and characterization, control strategy, and data management. There is also a detailed appendix containing the results of a benchmarking survey looking at leveraging process characterization data to accelerate validation.

It contains strategies and best practices to enable CGT developers to create platforms for the development of their products. For each section, a high-level ‘how to’ guide is provided, followed by suggested strategies for leveraging internal company or cross-industry approaches.

We believe this initiative will be a valuable resource to CGT developers as the industry strives to move forward with such platforming and prior-knowledge-based approaches, which are already well-established for other therapeutic modalities.

All CGT developers should increase the level of knowledge sharing and transparency around certain CMC (chemistry manufacturing and controls) knowledge, which can benefit the entire CGT industry. By continuing to share such approaches more openly, we have a great opportunity to collectively learn from each other’s experiences and help speed the delivery of these life-transforming therapies.

Want to know more?

We are running a webinar on 10 April 2024 that showcases the paper and will discuss opportunities to leverage process development and characterization knowledge across similar therapeutic technology platforms. For more details and to register, visit here.

For more information, download the paper here  and contact Kathleen O’Hagan, Global Change Facilitator, at kathleen.ohagan@biophorum.com

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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.

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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.

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Supply Resilience 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.

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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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