Outsourcing Next-Gen Therapies Relies on Expertise and Technology

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Next-generation biotherapeutics pose challenges to the development of manufacturing processes and analytical characterization because of their complexity and, in some cases, novelty. From cell and gene therapies (CGTs) and new vaccines to antibody-drug conjugates (ADCs), successful development and commercialization of these next-generation therapies may hinge on partnerships with service providers that offer specific focus, expertise, and technological experience. At certain points in the discovery and development process, developers—especially smaller firms—come to realize the unmet needs that prompt them to seek an outsourcing provider and/or partnership.The first order of business would be to bring in a partner with the correct expertise and technology to tackle the challenges of bringing a complex biotherapeutic to market.

Challenges

Bringing next-generation therapies to market brings up multiple challenges, according to Monika Paule, CEO, Caszyme. These challenges range from scientific and technical hurdles to regulatory and societal barriers. “One of the major obstacles is the social perception and acceptance of novel technologies such as CRISPR [clustered regularly interspaced short palindromic repeats]. Addressing public concerns, providing accurate information, and engaging in meaningful dialogue about the benefits, risks, and ethical considerations of these technologies are crucial,” Paule says.

Ali Pashazadeh, CEO, Treehill Partners, a transaction and drug development advisory firm focused on the biotech industry, points to the fact that the biopharmaceutical industry is currently experiencing its fourth year of financial drought, which has been one of the biggest challenges lately to bringing next-generation therapies to market. “Whilst next-generation technologies are incredibly exciting in their potential, these really require, for the first time, excellence in drug development to be combined with excellence in manufacturing,” Pashazadeh says.

Speaking specifically on nucleic acid-based therapies, Bernard Sagaert, CEO of etherna, emphasizes that the challenges are still big, even after the success of the COVID-19 vaccines. The challenges on the RNA front are tied in with RNA quality, where sometimes an inferior quality of RNA is used, which always leads to project failure in discovery or preclinical stages, cost of goods, or duration of expression, he explains. “On the delivery side, targeted expression and off-target expression reduction, balancing immune expression (regulate up or down, or be immune-neutral), and stability are the main challenges. All of these challenges can be different based on the therapeutic area where the products need to be used, whether it is in prophylactic or therapeutic vaccines, autoimmune diseases, gene editing, oncology, protein replacement, etc.,” Sagaert says.

Meanwhile, other challenges include technical and regulatory complexities, such as optimizing gene-editing precision and efficiency, ensuring scalable and cost-effective manufacturing, navigating complex regulatory requirements, and addressing safety and ethical considerations. “Overcoming these hurdles requires broad scientific expertise, strong analytical capabilities, and strategic collaborations among key ‘players’ to accelerate development while minimizing risks,” says Paule.

Complexity of manufacturing

For the most part, it is the complexity of manufacturing involved in next-generation biotherapies that drives outsourcing in this sector. “These therapies require specialized expertise in areas such as gene and cell therapy production, advanced analytics, and regulatory compliance—expertise that many biopharma companies do not have in-house. Of course, everyone would love to do everything in-house, but the amount of initial investment needed, the need for speed in development, cost efficiency, and enhanced scalability are the main factors driving outsourcing,” says Paule.

“A certain level of complexity multiplies directly in line with” the scale of manufacturing capacity,” adds Jason Jones, global business development lead, Cellular Origins. Jones explains that if processes are complex, variable, and expensive when manufacturing 1000 doses of a next-generation therapy, then these processes will be 100x more complex, variable, and expensive if they are continued to be produced the same way as production scales up 100x.

Read the article in Pharmaceutical Technology’s Bio/Pharma Outsourcing Innovation eBook.

About the author

Feliza Mirasol is science editor at Pharmaceutical Technology.

Article Details

Pharmaceutical Technology®
Bio/Pharma Outsourcing Innovation eBook
February 2025
Pages: 4–10

Citation

When referring to this article, please cite it as Mirasol, F. Outsourcing Next-Gen Therapies Relies on Expertise and Technology. Pharmaceutical Technology Bio/Pharma Outsourcing Innovation eBook, February 2025.