Cell and Gene Therapy CDMOs: A Guide to Cell and Gene Therapy Contract Manufacturing

Your guide to choose the right Cell and Gene Therapy CDMOs for your company

The emerging world of cell and gene therapy (C&GT) is rapidly changing healthcare by using living cells and genetic modifications to treat diseases that were difficult to treat before.

At a time when 90% of biotechs rely on contract development and manufacturers, CDMOs play a crucial role in early stage and commercial launch of your products.

Understanding Cell and Gene Therapy

Cell therapy and gene therapy are overlapping fields of biomedical research and treatment. Both therapies aim to treat, prevent, or potentially cure diseases, and both approaches have the potential to alleviate the underlying cause of genetic diseases and acquired diseases. But, cell and gene therapies work differently.

Cell therapy aims to treat diseases by restoring or altering certain sets of cells or by using cells to carry a therapy through the body.

Gene therapy aims to treat diseases by replacing, inactivating or introducing genes into cells— either inside the body or outside of the body.

Some therapies are considered both cell and gene therapies. These therapies work by altering genes in specific types of cells and inserting them into the body.

Despite the long-standing similarities in traditional drug categories like small molecules, the US FDA and the EU EMA have yet to align their guidance on emerging therapies. This includes Cell and Gene Therapies (CGTs) and their EU equivalent, Advanced Therapy Medicinal Products (ATMPs).

Download the full ebook to understand the key differences between the US and EU in the Cell & Gene Therapy development and review processes.

Cell and gene therapy CDMOs

Cell & Gene Therapy CDMOs work as specialised extension of a biopharma’s team, giving you their knowledge, facilities, and tools to handle important parts of your C&GT development and manufacturing.

They cover everything from figuring out the process and making DNA to producing viral vectors and processing cells. They provide a complete set of services to make your journey from the early stages to delivering treatments as smooth as possible.

Subcategories of C&GT CDMOs:

The diverse C&GT CDMO landscape can be segmented based on service scope and expertise:

• Full-service C&GT CDMOs: These one-stop shops offer a complete suite of services encompassing preclinical to commercial-scale manufacturing.

• Niche C&GT CDMOs: These specialized players focus on specific areas like viral vector production, cell line engineering, or fill-finish operations.

• Emerging C&GT CDMOs: These new entrants offer innovative technologies and disruptive business models, often focusing on specific cell or gene therapy modalities.

Cell and Gene Therapy CDMOs Market Trends

The market is rapidly expanding with new cell and gene therapy products available, as researchers are focusing more on rare diseases.

The global cell and gene therapy CDMO market size was estimated at USD 4.99 billion in 2023 and is expected to grow at a compound annual growth rate (CAGR) of 27.9% from 2024 to 2030. (Source)

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Key market trends

A number of factors are driving growth in C&GT CDMO market:

• There is a trend towards consolidation in the market. The cell and gene therapy CDMO market is highly fragmented, with a large number of small and medium-sized players. However, there is a trend towards consolidation in the market, as larger players acquire smaller companies to expand their capabilities and geographic reach. Acquisition of Synteract by Syneos health in December 2020 and Biosciences being bought by genesis Biotechnology in august 2020.

• CDMOs are investing in new technologies. CDMOs are investing in new technologies, such as continuous manufacturing and artificial intelligence, to improve the efficiency and scalability of their processes. This is essential, as cell and gene therapies are often complex and require specialized manufacturing techniques. For instance, in September 2023, Charles River Laboratories unveiled its new technology Lentivation lentiviral vector (LVV) manufacturing platform. This platform is designed to streamline the manufacturing timelines for gene and gene-modified cell therapies, potentially reducing them by up to 60%, resulting in fewer than seven months compared to conventional manufacturing workflows.

• The focus is on geographic expansion. CDMOs are expanding their geographic reach to meet the growing demand for cell and gene therapies in emerging markets. This is especially true in Asia, where the market is expected to grow at a significantly faster rate than in other regions.

• The regulatory landscape is evolving. The regulatory landscape for cell and gene therapies is still evolving, which can create challenges for CDMOs. However, CDMOs are working closely with regulators to ensure that their processes meet the latest requirements.

Cell & Gene Therapy CDMOs

The number of CDMOs offering cell & Gene Therapies is expanding rapidly. This infographic summarises those operating facilities in Europe.

Download the European CDMO infographic here and the North American CDMO infographic here

Cell and Gene Therapy Contract Development and Manufacturing Organisations:

This table below ranks cell and gene therapy contract manufacturers based on their total revenue derived from contract services in 2023. Note: this is revenue from across their business, not specifically for cell and gene therapy.

Benefits of Partnering with C&GT CDMOs:

Working with Cell and Gene Therapy CDMOs can bring several advantages:

• Expertise and experience: Gain access to specialized knowledge and proven track records in C&GT development and manufacturing.

• Infrastructure and resources: Leverage state-of-the-art facilities, technologies, and equipment unavailable internally.

• Efficiency and speed: Accelerate your development timelines and optimise manufacturing processes through CDMO expertise.

• Cost optimisation: Access economies of scale and potentially reduce development costs compared to building internal capabilities.

• Regulatory compliance: Gain support from CDMOs experienced in navigating the evolving C&GT regulatory landscape.

Challenges of Partnering with C&GT CDMOs:

Cell and Gene Therapy CDMOs can come with some challenges:

• Selecting the right partner: Careful due diligence and evaluation are crucial to finding a CDMO that aligns with your specific needs and expertise.

• Intellectual property considerations: Clear agreements and confidentiality measures are essential to protect your intellectual property.

• Quality control and data integrity: Maintaining quality standards and data integrity requires robust oversight and communication with your CDMO partner. Integration and communication: Seamless integration of the CDMO into your development process and consistent communication are vital for project success.

• Integration and communication: Seamless integration of the CDMO into your development process and consistent communication are vital for project success.

Opportunities

Collaborating with Cell and Gene Therapy CDMOs offers various opportunities for innovators:

• Expertise and experience: Cell and gene therapy CDMOs have a deep understanding of the complex processes involved in developing and manufacturing these therapies. They can provide your company with the expertise and experience you need to overcome challenges and bring your therapy to market faster.

• Access to specialized facilities and technologies: Cell and gene therapy CDMOs invest heavily in specialized facilities and technologies that are essential for developing and manufacturing these therapies. Partnering with a CDMO can give you access to these resources without having to make a significant upfront investment yourself.

• Improved efficiency and scalability: Cell and gene therapy CDMOs have optimised their processes for efficiency and scalability. This can help you to reduce your costs and bring your therapy to market faster.

• Reduced regulatory burden: The regulatory landscape for cell and gene therapies is complex and ever-changing. Cell and gene therapy CDMOs have the expertise to help you navigate this landscape and ensure that your therapy meets all regulatory requirements.

• Focus on your core competencies: By partnering with a cell and gene therapy CDMO, you can free up your internal resources to focus on your core competencies, such as drug discovery and development.

• Cost savings: In some cases, partnering with a cell and gene therapy CDMO can be more cost-effective than building your own manufacturing capabilities. This is especially true if you are a small or mid-sized company with limited resources.

• Faster time to market: By partnering with a cell and gene therapy CDMO, you can accelerate your development timelines and bring your therapy to market faster. This can be critical for therapies that are needed to treat patients with serious or life-threatening diseases

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Finding the Right C&GT CDMO Partner:

Selecting the right C&GT CDMO is a critical decision. Follow these steps for a successful partnership:

Define your needs and objectives: Clearly identify your specific project requirements, desired outcomes, and timeline.

Research and shortlist potential CDMOs: Conduct thorough research, considering their expertise, experience, facilities, technologies, and track record.

Develop a Request for Proposal (RFP): Outline your project details, requirements, evaluation criteria, and quality standards in a detailed RFP.

Evaluate proposals carefully: Assess each proposal against your evaluation criteria, focusing on relevant expertise, experience, cost structure, quality systems, and compliance record.

Conduct due diligence: Verify the CDMO’s financial stability, regulatory compliance, and infrastructure.

Negotiate and finalise: Approach negotiation with a collaborative mindset, seeking mutually beneficial solutions that align with both parties’ goals.

By carefully considering these factors, you can identify a C&GT CDMO partner that aligns with your project goals and contributes to the success of your cell and gene therapy development.

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