7:30 am Registration, Coffee & Networking
8:20 am Chair’s Opening Remarks
ALIGNING INDUSTRY LEADERS AROUND REGULATORY EXPECTATIONS
8:30 am FDA Perspective on Analytical Regulatory Expectations
Synopsis
- Provide insight in to the type of analytical data required to support agency licensure
- Present case studies reflective of specific requirements reflective of recent regulatory experience
- Share the need for adaptation to the specific and unique nature of the gene therapy program – how do expectations differ between indications and scales of manufacturing
- Review the validation necessary for new technologies – methods, instruments and software
- Offer insight into the current and future regulatory paradigms for gene therapy manufacturing
9:00 am Analytical Development Strategies for AAV Gene Therapy programs
Synopsis
- Considerations for development of analytical assays
– Assay throughput: In-process vs Release
– To qualify or not to qualify?
– In-house use vs outsourcing models - Development strategies for cell-based potency assays
– Phase appropriate development
– (case study) - Switching gears
– Adapting to program and assay changes
– (case study)
9:30 am Exploring Droplet Precision for Analytical Development
10:00 am Speed Networking & Morning Refreshments
11:00 am Analytical Methods: A Path to More Efficient Processes & Safer Gene Therapy Products
Synopsis
- A non-affinity AAV manufacturing process to manage formation of aggregates and result in extra low DNA contaminants will be presented
- Rapid and sensitive analytics for in-process control of AAV manufacturing with focus on HPLC and different in-line detectors will be shown
- Qualitative and quantitative information can be obtained in near real-time to assist with process decision-making
- New ultra-sensitive DNA assay showing major AAV-associated DNA contamination will be introduced
11:30 am Quantification of Vector Genomic DNA & Residual DNA in Gene Therapy Vectors by ddPCR
Synopsis
- Platform development of ddPCR assays
- Quantification of multiple targets by duplex ddPCR
- Need for optimization of parameters will be discussed
11:45 am Panel Discussion: Evaluating the Analytical Strategies of the Leading Companies in the Field
Synopsis
This interactive panel discussion will bring together the leading companies in the field to discuss key takeaways from pioneering gene therapy programs.
Topics to be covered include:
- Investigating common analytical challenges encountered across various programs in different indications and with different specifications
- Balancing the complexities of analytical requirements with the drive to progress therapeutics to market
- Establishing collaborative relationships with other departments within the organization
- Lessons learned from the recent approvals of gene therapy products – how has feedback from agencies changed and shaped analytical strategies moving forward?
12:15 pm Lunch & Networking
DEVELOPING EFFECTIVE AND CLINICALLY RELEVANT POTENCY ASSAYS
Synopsis
Session Chaired by:
Marina Feschenko, Principal Scientist, Analytical Development – Gene therapy and Biologics, Biogen
1:15 pm Potency Assessment for Novel AAV-Based Drugs: Exploration of Different Biological Activities & Stability Indicating Properties of the Assays
Synopsis
- There are three major types of in-vitro analytical assays for assessment of biological activity of AAV-based therapeutics: infectivity, target protein expression, and functional potency
- It is important to understand what information each assay can provide and how it can be used for process development and drug characterization
- Changes in AAV vector structure affect three biological assays in a different manner
- What strategy can we use if MOA is not clear?
1:45 pm Developing a Potency Assay for GeneRide Products: an AAV-based Genome Editing Platform
Synopsis
- The Gene Ride platform utilizes the natural process of homologous recombination to achieve targeted genome editing without the use of exogenous nucleases.
- Current guidance on potency assay development will be discussed, including specific obstacles for gene therapy products.
- Developing a potency assay for GeneRide vectors demonstrates additional challenges including low levels of integration which requires highly sensitive detection methods. Surrogate methods to investigate biological activity will be described.
2:15 pm A Platform Approach to Functional Potency Assay Development for Gene Therapy Products
Synopsis
- Streamlining development of functional potency assays across diverse
product types with custom and complex MOA - Bridging the gap between R&D and cGMP for commercially viable potency assays
- Case studies will be discussed for in vivo and ex vivo vector and non-vector based therapies
2:45 pm Interactive Roundtable Discussions: Deep Dives into Key Existing & Emerging Technologies Part 1
Synopsis
Participate in a focussed roundtable discussion to discuss key analytical technologies in an intimate environment. Each table will be led by an industry leader with experience in using the relevant methodology, so this is your opportunity to have your burning questions answered and benchmark your approach with your colleagues in the field. Roundtables will discuss the use of the following approaches
- qPCR and ddPCR
- Chromatography
- Ultracentrifugation
- Next Generation Sequencing
- AAV Titration ELISA
3:10 pm Afternoon Break
3:25 pm Interactive Roundtable Discussions: Deep Dives into Key Existing & Emerging Technologies Part 2
Synopsis
Participate in a focussed roundtable discussion to discuss key analytical technologies in an intimate environment. Each table will be led by an industry leader with experience in using the relevant methodology, so this is your opportunity to have your burning questions answered and benchmark your approach with your colleagues in the field. Roundtables will discuss the use of the following approaches
- qPCR and ddPCR
- Chromatography
- Ultracentrifugation
- Next Generation Sequencing
- AAV Titration ELISA
3:50 pm Afternoon Refreshments & Scientific Poster Session
Synopsis
The scientific poster session is an ideal opportunity to communicate your new results and expertise to a niche audience of industry experts, get feedback from peers and colleagues in the industry and learn how others in the field have been tackling similar challenges.
PIONEERING INNOVATIVE METHODOLOGIES TO EVALUATING CAPSID & GENOME QUALITY
4:20 pm Measure AAV Quality Attributes with SEC-UV-MALS-dRI
Synopsis
• Discussing a size exclusion chromatography (SEC) method coupled with UV, multi-angle light scattering (MALS), and differential refractive index (dRI) detectors to measure the following three important AAV quality attributes (QAs):
1. Total number of viral capsid particles
2. Relative capsid content (e.g., ratio of empty and full capsids)
3. Percentage of monomer or aggregates
• Comparing orthogonal methods using dynamic light scattering (DLS) and field flow fractionation (FFF)
4:30 pm Strategies and Technologies for AAV Genome Characterization
Synopsis
- Understanding the inherent complexities to AAV genome study
- Overview of traditional techniques for genome characterization and their limited capacity to meet regulatory requirements
- Comparison and evaluation of next generation sequencing platforms and their accompanying data analysis
5:00 pm Proven Answers for Protein, Vaccine and Gene Therapy Analysis
Synopsis
- Discover solutions for sizing, purity, and sequence analysis of your cell and gene therapeutics
- Easily analyze a wide range of nucleic acids, viral capsids, & proteins all on one platform
- Increase data confidence with robust solutions utilizing industry and regulatory proven technology
5:30 pm Analytical Ultracentrifugation: A Critical and Versatile Tool to Evaluate Gene Therapy Viral Vectors
Synopsis
- Analytical Ultracentrifugation is the only tool to provide direct quantification on empty, intermediate and full viral capsids
- Analytical ultracentrifugation using multi-wavelength detection offer greater confidence in characterizing the particle content
- Various forms of analytical ultracentrifugation can provide additional information that other separation techniques cannot
6:00 pm AAV Capsid Purity: the Control Strategy & the Evaluation of CE-SDS with LIF Detection
Synopsis
- Insights into the current strategies and gaps for determining capsid protein purity of AAV gene therapy candidates
- Providing an overview of novel labeling approaches to enable CE-LIF detection of AAV capsid proteins
- CE-LIF could be a QC-friendly method for routine monitoring of capsid purity compared with other CE-based analysis
6:30 pm Chair’s Closing Remarks
6:35 pm Networking Drinks Reception
Synopsis
After the formal presentations have finished, digest the full day of content with your
fellow delegates in a more informal setting at our evening drinks reception