CDMOs and Gene Therapy: Overcoming Manufacturing Roadblocks?
In the rapidly evolving landscape of gene therapy, small to mid-size biotech companies are racing against time to bring their products to clinical trials. One company making a significant impact in this race is Biovian, a contract development and manufacturing organisation (CDMO) offering a comprehensive service for the entire development process.
According to the Food and Drug Administration (FDA), by 2025, it is expected to approve 10 to 20 cell and gene therapy products annually. However, the current challenges in scaling and manufacturing of viral vectors for gene therapy are substantial. These challenges revolve around complex production processes, impurities, and maintaining quality control during scale-up.
Key Challenges
The unique impurity issues in viral vector production, such as residual host-cell DNA and plasmid DNA, require careful risk assessments and robust quality control to ensure safety. The process complexity and the lack of a standardized process across manufacturers make scale-up from lab to industrial levels difficult. Additional challenges include cost and manufacturing capacity, regulatory scrutiny, and the need for stringent quality and yield requirements in large-scale Advanced Adeno-Associated Virus (AAV) manufacturing.
Key Solutions and Approaches
To address these challenges, companies like Biovian are leveraging advanced bioprocess technologies, AI-driven optimization, and strategic manufacturing approaches. Transitioning from 2D cell culture to suspension bioreactors enables scale-up in controlled 3D environments, improving yields and consistency. AI and mathematical modeling are used to optimize viral vector design and manufacturing parameters, enhancing productivity, reducing costs, and accelerating time to market.
Other solutions include employing strategic scale-up plans, integrating proprietary platforms, and regulatory expertise through CDMOs, and using AI-driven automation in manufacturing and genome editing tools.
Biovian's Approach
Biovian, specifically, has a focus on AAV and adenovirus (AV) production. While detailed proprietary information is limited, Biovian likely uses advanced bioreactor systems, process development expertise, and quality control frameworks to address scalability and manufacturing challenges common in the industry.
The company's EMA-GMP compliant Quality Assurance/Quality Control (QA/QC) site is equipped to conduct analytical tests on product identity, purity, infectivity, endotoxin levels, and bioburden, as well as physical, protein-based, and cell-based assays.
To meet the growing demand for gene therapies, Biovian has doubled its capacity for GMP viral vector manufacturing. The company has also opened an aseptic filling line for recombinant proteins and plasmid DNA, expanding its existing biosafety level 1 and 2 viral vector fill and finish capabilities. The new filling line features a restricted-access barrier system to ensure aseptic quality without sacrificing process flexibility.
Biovian's manufacturing process of adenoviruses is sensitive to stress, temperature, and environmental factors that can lead to aggregation, affecting product yield and quality. The company takes full oversight of each project to ensure seamless integration from early development to the clinic and beyond, offering a unique, one-stop-shop concept: from gene to finished vial.
The Future of Gene Therapy
Gene therapy has become significantly safer and more effective due to the development of new virus backbone designs and mechanisms of action. Oncolytic virus therapy, when combined with checkpoint inhibitors, is regarded as a promising tool to attack cancer cells that evade the patient's own immune response. Gene therapies are no longer only targeting rare diseases, but larger patient populations.
Biovian aims to become the leading global player in the contract manufacturing of biologics, while maintaining its company culture that emphasizes personal contact, friendliness, and responsibility in customer relationships. The new filling line is fully automated and supports batches of up to 10,000 vials.
With notable gene therapy approvals such as Luxturna, Yescarta, Kymriah, and Imlygic in recent years, and the approval of Zolgensma for spinal muscular atrophy in children under two in 2019, the future of gene therapy looks promising. Companies like Biovian are playing a crucial role in overcoming the challenges in scaling and manufacturing, paving the way for more effective and accessible gene therapies.
- In the biotech world, Biovian, a CDMO, is utilizing advanced bioprocess technologies and AI-driven optimization to address the challenges in gene therapy's scale-up from lab to industrial levels.
- Biovian's focus on AAV and adenovirus (AV) production includes the use of advanced bioreactor systems, process development expertise, and quality control frameworks to combat common scalability and manufacturing issues.
- To meet the growing demand for gene therapies, Biovian has expanded its GMP viral vector manufacturing capacity and opened an aseptic filling line for recombinant proteins and plasmid DNA.
- Biovian aspires to be the leading global player in the contract manufacturing of biologics, offering a unique, one-stop-shop concept: from gene to finished vial, with a strong emphasis on personal contact and customer relationships.
- With Biovian's role in overcoming challenges in scaling and manufacturing, gene therapies for both rare diseases and larger patient populations are expected to become increasingly effective and accessible due to advancements in science, technology, and medical-conditions.
