In their efforts to bring groundbreaking therapies to market, developers have leaned heavily on contract development and manufacturing organisations (CDMOs) that prioritise innovation.
Pioneering CDMOs provide developers with the tools necessary to overcome today’s hurdles of quality, cost and speed to clinic, says Daniel Spurgin, Director of Strategic Partnerships at ReciBioPharm.
To combat these challenges, CDMOs have taken three key approaches to best serve developers.
The development of flexible gene therapy platforms
During the past 5 years, gene therapies have consistently been ranked as the “most expensive therapy in the world.”4–8
With 2111 gene therapies currently under development, CDMOs must balance product quality and efficiency with cost-efficiency, all while accelerating production timelines so that products can reach patients faster.9
To address this, CDMOs are increasingly focusing on developing platforms to produce gene therapies at accelerated rates; this includes flexible adeno-associated viral vector (AAV) platforms that offer significant advantages to developers, including the following:
- customisable reagents and cell lines: developers can leverage their preferred reagents or cell lines rather than being restricted to a predetermined option
- optimisation using design of experiment (DoE): DoE methodology streamlines AAV production optimisation, facilitating the evaluation of multiple variables to achieve optimal titre, quality and safety
- adoption of advanced technologies: flexible AAV platforms simplify the adoption of new technologies and processes, ensuring the delivery of the most effective therapies with the latest innovations.
Overall, flexible AAV platforms empower developers with greater control of the production process, enabling them to achieve cost-effective, high-quality and efficacious AAV-based therapies.
Advances in RNA therapeutics
RNA therapeutics have seen a rapid rise in demand, with 897 currently in the development pipeline as of April 2023 targeting rare conditions, cardiovascular diseases and genetic respiratory disorders.10,11
Recent advances in RNA therapies have significantly improved their stability, shelf-life and efficacy. Among these innovations, the shift from batch production to continuous mRNA manufacturing has gained considerable traction in the past year.
This transition has enabled developers to accelerate timelines and reduce production costs. As a result, many RNA therapies are poised for global reach.
CDMOs are vital in terms of advancing continuous mRNA manufacturing and possess the necessary specialised expertise, infrastructure and resources.
Recognising this, the US FDA has launched a programme combining CDMO, industry and academia expertise to develop a comprehensive template for continuous mRNA manufacturing for the biopharmaceutical industry.12
Innovative technologies transforming RNA manufacturing
CDMOs must evolve to incorporate cutting-edge technologies into the production processes to remain at the forefront of RNA therapeutics.
For instance, integrating automation into analytical testing can improve sampling efficiency and reduce analysis time. Machine learning (ML) and artificial intelligence (AI) can create models that allow real-time monitoring and control throughout the entire processing line.
Analytical testing plays a pivotal role in continuous RNA production, ensuring that therapies meet stringent quality standards and are safe for patient use.
By integrating inline process analytical technologies (PAT), manufacturers gain a comprehensive and robust monitoring testing system to consistently produce high-quality mRNA therapies.
The future of ATMPs in biopharma
In ATMP production, CDMOs are vital when it comes to driving transformative trends — utilising flexible AAV platforms, transitioning towards continuous mRNA manufacturing and integrating cutting-edge technologies — to introduce cost efficiency, enhance product quality and increase productivity.
As the landscape evolves, CDMOs must continue to grow to maintain their position at the forefront of innovation.
References
- www.fieldfisher.com/en/sectors/life-sciences/life-sciences-law-blog/the-future-of-medicine-atmps.
- https://doi.org/10.3402/jmahp.v4.31036.
- https://doi.org/10.14740/jocmr3964.
- www.fiercepharma.com/pharma/most-expensive-meds-u-s-topped-by-novartis-and-spark-gene-therapies
- www.fiercepharma.com/pharma/20-most-expensive-pharmacy-drugs-u-s-2020.
- https://assets.ctfassets.net/4f3rgqwzdznj/6QhNMRveZFs4ekOPLZ9m2O/a8b2c9de10161cc89b6d5334f6c67b56/most_expensive_drugs__july_2021_goodrx.pdf.
- https://pharmaoffer.com/blog/10-most-expensive-drugs-in-the-world/.
- www.statista.com/statistics/765652/ten-most-expensive-drugs-and-their-list-price/.
- www.asgct.org/global/documents/asgct-citeline-q4-2023-report.aspx.
- https://doi.org/10.3390/biom13101497.
- https://investors.modernatx.com/news/news-details/2023/Moderna-Announces-Advances-Across-mRNA-Pipeline-and-Provides-Business-Update/default.aspx.
- https://news.mit.edu/2023/mit-researchers-lead-new-center-mrna-manufacturing-0713.