Picking a CDMO? Gene therapy developers must demand a pilot study

Gene therapy developers should “demand” a pilot study before selecting a CDMO to help focus on process development, says expert.

Millie Nelson, Editor

April 22, 2024

4 Min Read

The topic of selecting a contract development manufacturing organization (CDMO) is one the industry has discussed at length. The conversations have ranged from the importance of timelines, knowing your molecule, and when it is time to dump your selected partner.

Pamela Whalley, associate director at Complement Therapeutics, told delegates at Phacilitate’s Advanced Therapies Week in Miami 2024 to “demand a pilot scheme” to reach the overall aim of having “an effective and diligent manufacturing partnership that will yield a drug product that can reach the patient in a cost-effective and timely manner and is safe to use.”

UK-based firm Complement Therapeutics is a spinout from Manchester University. Its lead asset is an adeno-associated virus (AAV) gene therapy CTx001, which aims to treat Geographic Atrophy (GA) where no licensed treatment is currently available to patients.

First steps

Whalley outlined the parameters to consider when selecting a gene therapy CDMO. She said there are multiple questions you must ask relating to the following:

  • Facility and capacity: “Do their capabilities, facilities, processes, and quality system match what you require?”

  • The people: “Do the team have the knowledge and technical experience for your needs?”

  • Costs and timelines: “Do their costs align with your budget and do they meet your timelines?”

  • Sponsor quality control and oversight: “Do they facilitate quality audits, inspections, visits and review of approved suppliers?”

  • GMP compliance and regulatory expertise: “Does the company culture drive compliance to quality and cGMP? Do they offer regulatory support? What is their experience in different territories?”

  • Innovation potential: “How do they compare to the state of the art? How eager are they to improve their technologies?”

While the considerations noted by Whalley for a gene therapy developer do not differ much from what a biologics or cell therapy firm would have to think of, she noted it is important to “demand” a pilot study before selecting your chosen CDMO.

The pilot study should be “small-scale” and take place “before [committing] to a partnership.” The benefits of doing so mean you can conduct a “parallel assessment of more than one CDMO to establish baseline performance of your product in their process.”

In turn, this helps support a gene therapy developer’s CDMO selection, while focusing on process development for the product.

Whalley’s advice to “demand” a pilot scheme relates predominately to how a product performs in the third party’s process. However, it also provides the developer with the experience of seeing how the CDMO operates with relation to its timelines, manufacturing processes, people, and communication style.

What next?

Once you have chosen your CDMO, the gene therapy developer should then do specific things to create a successful partnership. Key to its success lies within communication and Whalley said the developer should “set expectations early.”

The expectations include the frequency of communication you would like to have, the content it will revolve around, the format you would like this to be delivered in and the “recipients” who you will be communicating with. Additionally, she said you cannot shy away from issues and should “prompt communication of good, bad, and unexpected data.”

Whalley spoke about the dangers of making assumptions. She said to avoid assumptions, the developer should set out clear and “mutually agreed” expectations for the project. For example, expectations of the deliverables, the timelines, the budget, the milestones, quality measures, and performance indicators should be communicated.

While it is beneficial to have clear expectations, the developer should not forget to ask the question of “who is going to be responsible for what?” By having specific people dedicated to parts of the process, you can “eliminate any potential problems” and create a partnership where “both parties are on the same page.”

However, Whalley said the developer must anticipate some changes may need to be made. This is where agility and flexibility come into play as the “agreed” plan may have to change because of problems identified in the development, production, data, company priorities, and competitor arena.

To try and ease the burden of change, a gene therapy developer and its CDMO can proactively plan for such scenarios.

“In a partnership you are a ‘we’ and not a ‘you and them’. Work together and make space for all members of the team to be heard in relevant meetings so that ideas can be shared and heard by the decision makers.”

About the Author(s)

Millie Nelson

Editor, BioProcess Insider

Journalist covering global biopharmaceutical manufacturing and processing news and host of the Voices of Biotech podcast.

I am currently living and working in London but I grew up in Lincolnshire (UK) and studied in Newcastle (UK).

Got a story? Feel free to email me at [email protected]

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