The global biopharmaceutical industry is continually innovating and looking forward to discover the next class of therapies to treat and cure patients around the world.
Cell and gene therapies, which fall into the broader category of drugs known as Advanced Therapy Medicinal Products (ATMPs) have emerged as an incredibly promising class of treatments that have shown the ability to treat and even cure many individuals with diseases for which there was previously little to no hope.
Cell and gene therapies work by substituting the missing or defective gene and resulting protein or cell causing the disease’s symptoms. Cell therapy treats diseases by restoring or altering targeted sets of cells or by using cells to carry a therapy throughout the body. Gene therapy operates differently by replacing, inactivating or introducing genes into cells — either inside the body (in vivo) or outside of the body (ex vivo). Some therapies are considered both cell and gene therapies. These therapies work by modifying genes in specific types of cells and inserting them into the body.
Current market forecasts predict the cell and gene therapy market to grow by almost $10B by 2026. A compound annual growth rate of more than 20%.1
Clearly the cell and gene therapy market indicates immense growth potential. But, with this massive increase, comes the need for the research, development, testing, and manufacturing services to bring these products to patients quickly, efficiently, and with this highest possible quality and safety.
Eurofins BioPharma Product Testing (Eurofins BPT) supports the development of ATMPs both for traditional use as well as for use in personalized medicine. The company provides comprehensive cGMP-compliant CMC testing support to ensure the identity, potency, purity, and safety of starting materials, intermediate products, vectors, and final drug products as well as support for manufacturing process development and validation.
Responding to a Growing Market
Eurofins BPT is experiencing significant growth in cGMP testing of both traditional biologics (mAb/protein therapeutics) as well as Cell & Gene Therapy (CGT) products or ATMPs. To address the growing demand for testing across biologics and the challenges posed by the growing ATMP sector, Eurofins BPT is increasing the size of many of its laboratories and adding a new lab focused specifically on viral vectors and ATMP product testing. This is all part of an overall strategy to support the ATMP product testing demands as well as expansion of the viral safety, biochemistry, and molecular testing services that the company’s ATMP clients require.
Commenting on this growth, Marian McKee, PhD, Vice President of Biosafety said, “The ATMP lab was designed to accommodate many of the tests required to release viral vectors for use in cell and gene therapy applications. The space includes cell-based assay capabilities with various endpoints, including a PCR suite and immunoassay set up to accommodate endpoint analysis. The first methods to be launched out of the CGT/ATMP lab will be the replication competent and viral vector quantitation assays. The space is also designed to accommodate basic biochemistry set up for release testing and affords space for future expansion as demand grows.”
A Closer Look at ATMPs and Cell and Gene Therapy Services
“Eurofins BPT has a long history of offering cGMP-compliant CMC testing of starting materials, intermediate products, vectors, and final drug products and supporting manufacturing process development and validation,” says Marian McKee. “This includes testing of raw materials, cell and viral bank services, plasmid and viral vector testing, lot release testing, bulk and finished product testing, and stability studies.”
For cell and gene therapy products, Eurofins BPT provides cGMP testing services to support both autologous and allogeneic cell therapies, including rapid sterility (BAcT/Alert 3D technology) and mycoplasma (MycoSEQ Real Time Detection System for Mycoplasma). Within the Eurofins BPT network of companies, analytical ultracentrifugation to support regulatory expectations around empty/full capsid analysis has been added recently. Eurofins BPT has also invested in technology to allow rapid sterility testing requirements for autologous cell therapies and digital droplet PCR for more precise quantitation of viral particles. Investments in Eurofins BPT facilities expand CGT capabilities and include laboratories being updated to accommodate biosafety level 2 (BSL2) work to maximize space utilization and to optimize workflows.
Investments in Cell and Gene Therapy Testing Technologies
With the industry’s drive to bring more cell and gene therapy products to patients, comes the inevitable need to implement the latest technologies and instruments to test and analyze these ATMPs. Eurofins BPT is making significant investments in facilities and instruments to ensure the company has the most up-to-date capabilities.
"As clients' needs and market trends evolve, we've been in lockstep by expanding our lab space and capabilities specifically with our new transmission electron microscopy (TEM) lab in a newly built laboratory space designed to support the company’s ATMP testing offering,” said Marian McKee.
The addition of electron microscopy allows Eurofins BPT to offer a full panel of in vitro test methods required for bulk harvest (UPB) testing and cell line characterization (CLC). The TEM lab will complement the cell and gene therapy testing services for viral vector characterization, including analysis of capsid architecture and empty/full analysis.
Marian McKee continues, “In addition to the new lab space for TEM, Eurofins BPT is investing in expansion of molecular biology, biochemistry, sterility, mycoplasma, viral safety, and raw materials testing, as well as stability laboratories across its Lancaster campus. Much of these enhancements are in response to the growing demand from the advanced therapy market.”
The addition of new instrumentation and technology is designed to handle the smaller sample volumes inherent to cell and gene therapy and for specialized testing. Examples include ddPCR, Maurice, Ella, Flow Cytometers and Analytical Ultracentrifugation (AUC).
Global Support for ATMP Capabilities
Eurofins BPT has the largest network of harmonized bio/ pharmaceutical cGMP product testing labs around the world in order to provide services to the global biopharmaceutical industry.
As the need for ATMP services in general and cell and gene therapies specifically grows, Eurofins BPT is investing in facilities, technologies and personnel worldwide to meet the burgeoning demand.
“In the United States, the Lancaster, PA, laboratory is the lead site in development of advanced capabilities and offers transfer of methods to support clients working with Eurofins BPT sites in Columbia, MO, and San Diego, CA,” says Marian McKee.
As the modalities employed for gene therapy change, Eurofins BPT is able to adapt existing capabilities or add new ones to address the emerging market. Currently, Eurofins BPT is able to support testing and release of:
- Viral vectors for in vivo and ex vivo use
- Nanoparticles
- Plasmid based
- iRNA/mRNA
- Oncolytic Viral Therapies
- Allogeneic and Autologous Cell Therapies
In addition, Eurofins BPT has built global teams that are focused on various product modalities, including traditional biologics and cell/ gene therapy.
“The global teams are tasked with sharing information about regulatory trends, new testing approaches, and market demands from both local and international geographies,” says Stanley Prince, Senior Scientific Advisor Manager.
The company’s laboratories in Europe and Asia Pacific are also making significant investments to support the cell and gene therapy cGMP testing requirements. Some of the investments include ddPCR, NexGen Sequencing, Viral Safety, and many more.
For product specific methods such as potency, infectious titer, and others, Eurofins BPT has the ability to transfer methods for cGMP testing between sites to accommodate product release in the various geographies under various regulatory umbrellas.
The Future of ATMP and Cell and Gene Therapy Testing Services
As the global market for these therapies continues to expand, Eurofins BPT is committed to bringing the latest tools and technologies into use for its current and future clients.
“We are continuing to gather information from our clients directly as well as from conferences,” says Stanley Prince. “The information we gather helps us to evaluate investments into new technologies and methodologies to support the cGMP testing requirements of these therapeutics.”
In addition, Eurofins BPT also participates in various regulatory strategy review committees to review new guidance documents and provide feedback to the agencies, discusses traditional biologics testing problems that may negatively impact the cell and gene therapy industry, and works collaboratively with companies globally to bring new alternative approaches to help further progress the development of these much-needed therapeutics.
References
- https://www.reportlinker.com/p06325351/Global-Cell-and-Gene-Therapy-Market. html?utm_source=GNW
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