8:00 am Check-In & Coffee
8:55 am Chair’s Opening Remarks
Uncovering Scalability & Commercialization of Viral Vector Production While Achieving Higher Yields for Enhanced Market Access
9:00 am Panel Discussion: Bridging Perspectives on Collaborating for Scalable Solutions in Viral Vector Manufacturing
Synopsis
- Capacity Constraints in Bioreactors: Discuss the limitations in operating bioreactors beyond 500 liters and the impact on production scalability
- Inefficiencies in Current Production Strategies: Examine the challenges and costs associated with aggregating multiple smaller runs, highlighting the need for more effective manufacturing solutions
- Innovative Solutions for Tech Transfer: Explore best practices for technology transfer and collaborative approaches between service providers and manufacturers to enhance efficiency and overcome logistical hurdles in scaling up production
10:00 am Streamlining Tech Transfer in Viral Vector Manufacturing to Prevent Introduction of Quality Variations
Synopsis
- Implement standardized protocols for tech transfer that encompass detailed risk assessments and process validation to ensure seamless transitions between manufacturing sites while maintaining quality and compliance
- Focus on developing cross-functional training programs that equip teams with the necessary expertise in viral vector production, fostering collaboration and knowledge sharing to uphold quality standards during transitions
- Design and utilize flexible quality management systems that adapt to changes in manufacturing processes, incorporating real-time monitoring and robust documentation practices to ensure product integrity and regulatory adherence
10:30 am
Standardized Full Capsid Enrichment & PCR Sample Preparation Platforms for AAV Process Development
Presented by Teknova
Synopsis
- Outlining the need for standardization in the manufacturing of AAV gene therapies to ensure reproducible and scalable processes
- Demonstrating two products to provide a platform solution for critical steps within the AAV workflow
- Presenting a serotype specific AEX buffer screening kit to facilitate quick and scalable identification of the optimal equilibration and elution formulation needed for high recovery and purity
- Illustrating a standardized AAV sample preparation platform for use in PCR applications that demonstrates robustness across serotypes and steps within the manufacturing workflow
11:00 am Morning Break & Networking
11:45 am Round Table Discussion: Scaling Up Viral Vector Manufacturing While Maintaining Quality & Regulatory Compliance
Synopsis
- Share practical experiences and insights gained from transitioning from small-scale development to commercial production, emphasizing key pitfalls to avoid
- Discuss the critical importance of maintaining product quality while scaling up manufacturing processes, including strategies for ensuring compliance with regulatory standards
- Consider the importance of integrating efficient process development practices, such as automation and real-time monitoring, to enhance scalability and reduce time-to-market for viral vector products
12:45 pm Presentation by NanoFCM
Synopsis
- Session and speaker details to be announced
1:00 pm Lunch Break & Networking
Leveraging Advanced Viral Vector Platform Technologies & New Therapeutic Modalities to Reduce Time-To-Market & Delivering More Effective Treatments
2:00 pm Novel Approach to Treating Rare Disease Using an AAV Vector to Deliver Gene Editing Tools to the Brain
Synopsis
- Provide overview of the development of LETI-101, a preclinical stage AAV5-delivered Life Edit® nuclease and guide RNA for mutant allele-specific editing in Huntington’s Disease
- Share approach for assessing manufacturability of LETI-101 through linking preclinical and CMC efforts
- Discuss the importance of biotech “owning” the future CMC problems at an early stage of pharmaceutical development to de-risk clinical trials and commercialization
2:30 pm CMC Strategies for Ensuring AAV Vector Potency and Infectivity in Gene Therapy
Synopsis
- Potency Assays for AAV Vectors: Discuss the development and validation of reliable potency assays to ensure that AAV vectors retain their ability to effectively transduce target cells and deliver the therapeutic gene
- Balancing Yield and Infectivity: Address the challenges of maximizing viral yield without compromising infectivity, focusing on process optimization, transfection efficiency, and the importance of maintaining high-quality vector production
- Quality Control and Regulatory Alignment: Highlight best practices for aligning manufacturing processes with regulatory expectations for quality control, potency testing, and infectivity assessments in clinical development
3:00 pm Afternoon Break & Networking
3:30 pm Unlocking Potential: The Promise of Transient Transfection in Viral Vector Production
Synopsis
- Transient transfection enables rapid generation of viral vectors, eliminating the lengthy stable cell line development process and facilitating quicker access to early-stage research and clinical trials
- This method allows for easy modifications and testing of various vector constructs, empowering researchers to tailor vectors for specific applications and enhance performance through iterative design
- With the potential for significant viral yields and reduced production costs, transient transfection offers an efficient alternative for both small-scale and large-scale production of viral vectors, addressing the growing demand in gene and cell therapies
4:00 pm Overcoming Downstream Impurity Challenges in Lentiviral Manufacturing: A Point-of-Use Approach
Synopsis
- Sharing insights from the point of use on common impurities faced during lentiviral vector production, such as residual host cell proteins, DNA, and product-related contaminants, and their practical impact on downstream processes
- Highlighting effective, on-the-ground strategies for dealing with impurities, from optimizing filtration methods to implementing process control techniques that ensure the purity of lentiviral vectors in real-time
- Discussing the challenges and solutions for ensuring impurity control and product consistency during scale-up, with a focus on practical techniques and decisions made in the lab or manufacturing environment to meet regulatory requirements