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

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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.

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