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As we enter a new era of precision medicine, the rapid emergence and evolution of cell and gene therapy (CGT) has offered hope for the treatment of both rare and common genetic disorders as well as previously incurable life-threatening diseases such as cancer. This fast-paced development is reflected in the rapid growth of the global cell and gene therapy market which reached nearly $4.4 billion in 2020 with a compound annual growth rate (CAGR) of 25.5% over 5 years. Moreover, the rising prevalence of chronic diseases coupled with an aging population is expected to propel the growth of this market to almost $34 billion by 2030.[i]
The evolution of cell-based therapeutics has also brought to the forefront an emphasis on biomanufacturing innovations. Development for CGT requires improvements in biomanufacturing efficiency and flexibility in manufacturing.
Some of the key biomanufacturing trends that have emerged in the pandemic era include the explosive growth of new modalities and the need for rapid industrialisation. Novel platforms such as cell therapy, gene therapy, and mRNA are complementing more traditional platforms such as mAbs, and as such, researchers are looking at more therapeutic areas such as Alzheimer’s disease, Parkinson’s disease and Infant Spinal Muscular Atrophy.
These sources of research and development, along with the usage of these platforms to support a wide landscape of therapeutic areas, bring about opportunities to collaborate and transform the industry. Digitalisation and rapid adoption of Industry 4.0 technologies will also revolutionize all aspects of biopharma.
Though accelerated development in the CGT sector is promising, it needs to be accompanied by a fundamental shift in the traditional biomanufacturing model. However, challenges for CGT exist at every level. Scalability and manufacturability remain two closely linked challenges that the CGT industry faces to fulfil its clinical potential.
The specialized biologic material used in CGT makes it imperative that the open systems inherited from the traditional research and development space cannot be applied due to the frequent need for sampling and the associated risk of contamination. There also is a lack in ready-to-use packaged materials as well as GMP availability of raw materials with supply chain reliability and controlled manufacturing.
Against this backdrop, how can industry players pinpoint core challenges, and navigate complex paths towards better outcomes? Some specific solutions are outlined below.
Coordinating with suppliers to evaluate and implement cGMP materials early in the developmental process can help expedite scale-up. Early risk assessment along with subsequent reviews at every level of the development process can also improve efficiency. Elimination of manual steps along with sustained analysis of the ongoing processes are crucial to maintaining an optimized pharmaceutical quality system.
Several upscaling risks can also be minimized by utilizing single-use and closed-system processing. Customizable sampling platforms such as Avantor’s OmniTop Sample Tubes® system with an adjustable volume sampling system (AVSS)[ii] are beneficial in CGT due to the precision and control they offer while drawing the sample, eliminating volume loss. On the other hand, the implementation of closed-system processing using ready-to-use sterilized solutions enhances speed and flexibility while facilitating automation.
A robust life sciences ecosystem requires collaborative approaches, hence companies who are doing this life saving work need suppliers they can trust.
Avantor offers that value to our customers – whether it’s through the sheer scope of our products and services, our global network and infrastructure or the technical expertise that is brought to our customers. We offer our customers access to a whole new world of services and support that would be nearly impossible to replicate on their own.
Optimizing processes and establishing efficient production work environments are the key to improving overall production. Technical expertise can be used to enhance services across key production service segments such as Design Consulting, Planning and Materials Management, Production Readiness and Critical Environment and Sanitation. Overall, coordinating with an end-to-end supplier can help enhance every step of the CGT cascade ranging from consumables such as cGMP chemicals, single-use solutions, equipment and sera, production, logistics support, environmental control, and staffing expertise.
Adaptive digital ecosystems to optimize workflow needs for lab inventory, chemical and equipment management, as well as biological samples help connect scientists to manufacturing processes without compromising their work. Digital sample managers can provide full visibility for secure storage and enable on-demand retrieval of biological samples from biorepository and archiving facilities. These cloud-based digital solutions can be used to track and document every stage of the scientific journey, making lab and inventory management easy to replicate. These efficient, flexible and simple tools help overcome supply chain hurdles, and enhance coordination between scientists.
Though CGT represents the next frontier in biomanufacturing, several capability gaps exist making upscaling of the therapies from the lab to the patient a challenge. The unique demands of CGT biomanufacturing necessitate an early-stage collaboration between suppliers and biomanufacturers to enable successful process implementation and regulatory compliance. Through collaborative efforts between manufactures and suppliers, resilient support systems can be established by incorporating innovative/flexible material workflows, reducing system-based risk and implementing robust formulation strategies.
Investing in building robust end-to-end supply chains can also ensure quicker access to high quality materials. For example, to better serve its customers in the AMEA region, Avantor in May announced a new manufacturing and distribution hub in Singapore, by integrating its existing distribution facility with new manufacturing operations. The new hub, which provides a range of services including quality control and inventory management expertise, aims to bring Avantor solutions closer to regional customers and strengthen global supply chain capabilities.
Lastly, utilization of cGMP reagents and chemicals, process optimization with aseptic fluid handling technologies and digitization of workflows can be the key drivers toward overcoming the challenges associated with commercializing new therapies. Successful integration of innovative clinical development with improvements in process technologies can help companies face the constantly evolving CGT landscape.
