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
9:45 am Enhancing Reproducibility: Standard Operating Procedures & Quality Controls in Viral Vector Development
Synopsis
- Explore the critical role of standardized operating procedures (SOPs) in optimizing viral vector production workflows, reducing variability, and ensuring consistent outcomes across multiple development batches
- Examine best practices for quality control that integrate advanced analytical techniques and real-time monitoring, ensuring the integrity and efficacy of viral vector products throughout the development lifecycle
- Discuss how adherence to SOPs and rigorous quality controls can accelerate regulatory approval processes, ultimately paving the way for successful commercialization of viral vector-based therapies
10:15 am Process intensification of rAAV purification with column-free BioRMB platform
Synopsis
- Introduction to BioRMB – a real moving bed column-free chromatography platform with fully closed single-use flowpath
- AAV purification data with multiple serotypes featuring significant improvements in productivity (10-20X)
- Discussion on mechanisms of action vs status quo column purification that lead to more robust product quality and higher recoveries
10:25 am Morning Break & Networking
11:25 am The Trend of Plasmid-based AAV Manufacturing in HEK293 Cells
Synopsis
- Current challenges in plasmid-based AAV manufacturing platforms
- Technical innovations that improve rAAV titer and purity with reduced cost
- Potential future directions towards off-the-shelf AAV manufacturing
11:55 am Presentation by NanoFCM
Synopsis
- Session and speaker details to be announced
12:10 pm Lunch Break & Networking
1:10 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
Leveraging Advanced Viral Vector Platform Technologies & New Therapeutic Modalities to Reduce Time-To-Market & Delivering More Effective Treatments
1:40 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
2:10 pm Afternoon Break & Networking
3:10 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
3:40 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