With ongoing advances in science and technology, the cell and gene therapy pipeline has grown especially robust over the past few years. At present, ClinicalTrials.gov shows more than 4,500 active gene therapy trials globally. In the United States, McKinsey experts expect to see 10 to 20 cell and gene therapy approvals per year over the next five years.
This rise in supply has created a heightened demand for contract development and manufacturing organizations (CDMOs) with biotech expertise. CDMOs typically supply materials and handle production and manufacturing, allowing life sciences companies to focus on innovation and marketing.
The bottleneck stems from a shortage of CDMOs with gene therapy expertise and resources. Considering the critical need for safe, effective gene therapies and the rapid pace of development, it's important for pharma and biopharma to find a CDMO with both gene therapy capabilities and availability to take on new partners now—not 18 months from now.
"Full-service CDMOs that can assist with both development and manufacturing are in highest demand," said Richard Welch, PhD, vice president, development services for Emergent BioSolutions, a global CDMO and specialty life sciences company headquartered in Gaithersburg, Maryland. "As pharma and biopharma companies move from early phase to late phase, CDMOs need experience with process characterization and process validation as well as commercial production and supply chain."
"The supply chain is much more complex," added Tarek Abdel-Gawad, senior director of commercial strategy for Emergent BioSolutions. "You aren't just growing cells. You're ensuring viruses, helper viruses, and plasmid DNA work together to produce the molecule of choice. Few companies have the capabilities, equipment, and GMP expertise."
The Benefits of a "Single-Source" CDMO
Much of the gene therapy development as of late has stemmed from smaller biotech companies or research universities according to a McKinsey report. Large pharmaceutical companies may partner with these organizations on rare disease or oncology treatments – two therapeutic areas where much of the research lies.¹
Many small to midsize companies have the idea and investor support, but do not have the employees, infrastructure or manufacturing space. "A CDMO is a good partner in those cases," said Mukesh Mayani, PhD, principal scientist, gene therapy at Sanofi. "You can test your hypotheses and work with a CDMO that has the platform, the people, and the preclinical models. This arrangement speeds up the timeline and allows these innovative companies to focus on other modalities and molecules."
Pharma and biopharma companies of all sizes can learn from this "single-source" approach. Partnering with a CDMO earlier in the process—from preclinical development through packaging—frees up resources to focus on innovation and communication.
"It is neither simple nor cheap to develop and manufacture gene therapies," said Dr. Welch. "A CDMO has the built-in skill set to grow viruses at the densities necessary to meet early-phase studies while hitting safety margins. With the clinical trial failure rate as high as it is, working with a CDMO that has experience in different technologies and products makes for a more efficient, cost-effective process."
What to Look for in a CDMO
Although there is a high demand now for CDMOs with gene therapy expertise, the market is quickly growing. According to Grand View Research, the CDMO market is expected to grow from $115.6 billion in 2020 to $157.7 billion in 2025, outpacing the pharmaceutical industry as a whole. New cell and gene therapy CDMOs are emerging and established CDMOs are expanding capabilities.
Before you start your CDMO search, consider the following two factors:
- How much support do you need? Larger, established pharmaceutical companies may have broad capabilities, but want expert support for a particular product or therapeutic area. A new biotech startup may want to outsource more functions—from preclinical through phase four—to a CDMO that has GMP production facilities and the ability to scale.
- What intellectual property do you and the CDMO own? Evaluate whether, or to what extent, your intellectual property (IP) portfolio allows you to contract with a CDMO or what IP rights are owned by the CDMO. "The CDMO IP ties you to the technology being used for manufacturing of products and therefore adds to the cost of patient treatment due to milestone payments during development and royalty payments once the product is commercialized," said Dr. Mayani. "There may not be much competition in a particular therapy now, but as time moves on, and competition puts pressure on cost of therapy, you'll have to keep in mind how the IP impacts cost.
When vetting CDMOs for your gene therapy studies, consider the strengths and weaknesses of your company as well as your potential CDMO partner. A few points to consider include:
- Track record. "The CDMO needs development know-how and a history of successful delivery of molecules that move programs through the clinical trials," said Dr. Mayani.
- Full-scope manufacturing. Look for all manufacturing capabilities needed to produce, process, and purify cells, viruses, and genes. Drug product fill finish and analytics capabilities are core competencies that a single-source CDMO should have.
- Scalability. Look for a CDMO with both manufacturing and production expertise that can be a partner from beginning to end. They also need the ability to scale up in development as needed.
- Experts. Your CDMO must have experienced culture and virology experts as well as skilled biochemical engineers and biochemists. Look for a team you can trust with your company's cell culture, viral production, viral purification, and data analytics.
- Synergy between development and manufacturing sites. Emergent BioSolutions uses the same single-use bioreactors in its development and manufacturing sites. It uses the same isolators in its drug product development and fill sites. That type of synergy should extend to people and processes: the manufacturing and development operations teams should share the same methodologies and operational execution targets.
Your CDMO: Partner and Navigator
As gene therapy research continues to expand, innovators in this space will need CDMOs with highly specific expertise, facilities, and equipment. Choose a partner that can assist from the earliest phases of product development all the way to commercialization.
¹ Capra, Emily, et al. "Gene therapy coming of age: Opportunities and challenges to getting ahead." McKinsey, October 2, 2019