7:50 am Registration & Coffee
8:50 am Chair’s Opening Remarks
ESTABLISHING ROBUST PHASE-APPROPRIATE ANALYTICAL METHODS
9:00 am (CASE STUDY) Establishing a Robust & Efficient Upfront Potency Assay Strategy to Prevent Setbacks Downstream
Synopsis
- Establishing a lean matrix approach to phase I/II, keeping in mind a regulatory strategy for late and commercial phase readiness
- Discussing contemporary tools for early potency assay development to increase the likelihood of meeting potency CQAs
- Navigating regulatory hurdles to maximise the chance of approval success
9:30 am PANEL DISCUSSION: Advancing Gene Therapy Manufacturing: Automated Analytical Tools for Precision & Efficiency
Synopsis
- Characterising methods to assess CQAs throughout manufacturing, storage, and shipment
- Increasing analytical throughput for in-process and release quality testing to GMP compliance in preparation for phase three or commercial launch
- Introducing automation or in-process analytics to establish effective parameters for reproducibility
10:15 am Morning Refreshments & Speed Networking
Synopsis
This session is the ideal opportunity to get face-to-face time with many of the brightest minds working in the gene therapy analytics field and establish meaningful business relationships to pursue for the rest of the conference.
BIOASSAYS/MOLECULAR BIOLOGY
USING PCR TO QUANTIFY VIRAL TITRE & DETERMINE GENOME INTEGRITY
11:00 am (CASE STUDY) Investigating & Improving Methods for Genome Integrity Quantification
Synopsis
- Outlining and discussing methods for target specific genome integrity evaluation
- Quantification aspect of vector genome integrity by different approaches
- Case study on multiplex dPCR and novel BLI platform to inform full-length vector genome titre
- Signifying the importance of vector genome integrity to support process development
11:30 am Mastermind Session: qPCR vs ddPCR vs dPCR: Which is the Best Technique to Use for My Viral Titer?
Synopsis
With the emergence of more and more molecular biology tools, it is becoming difficult to assess which is the best to use in practice. This is only complicated further by the lack of an exact match across PCR titer methods, making it hard to compare relative precision and accuracy.
This mastermind will give each group the same set of questions surrounding this central problem. Answers will be collated at the end of the session and distributed to all attendees post-conference to gain insights into current perspectives in the field and suggestions and ideas of relevant actions we can take as a collective to navigate misconceptions.
Topics to cover include:
- Examining the differences in precision and accuracy among qPCR, ddPCR, and dPCR methods for viral titre determination.
- Investigating the reasons behind the lack of exact match between various PCR techniques.
- Identifying existing technological gaps in sample preparation, automation, and throughput and discussing strategies for improvement in these areas.
- Using multiplex PCR methods to comprehensively quantify genome integrity
- Technical considerations for sample preparation
PHYSICOCHEMICAL CHARACTERISATION
CHARACTERISING PROCESS & PRODUCT-RELATED IMPURITIES TO INFORM PRODUCT EFFICIENCY
11:00 am (NEW DATA) Exploring Analytical Tools to Investigate Photodegradation of AAV
Synopsis
- Outlining sample preparation for AAV photodegradation
- Investigating degradation pathways with analytical tools
- Using mass spectrometry to analyse AAV post-translational modifications
- Identifying sources of observed High Molecular Weight Substances
11:30 am (NEW DATA) Utilising Physicochemical Analytical Methods to Quantify the Impact of AAV Degradation Impurities, Provide Novel Insights on their Impact on Efficacy & Potency
Synopsis
- Presenting novel data on physically and chemically induced forced degradation
- Characterising whether degradation leads to novel capsid or PTM formations
- Understanding the impact of degradation-related impurities on efficacy and potency
12:00 pm (NEW DATA) (NEW TECH) Understanding AAV Impurities & Product Attributes Through Advanced Bioanalytics for Bioprocess Characterisation
Synopsis
- Using orthogonal Mass Spectrometry (MS) and Capillary Electrophoresis methods to calculate VP identity & ratio
- AAV capsid PTM assessment using a MAM MS-based workflow
- Host-cell protein (HCP) profiling using MS tools
- Determining the impact of bioprocess parameters on HCP levels and capsid CQAs
12:30 pm Lunch & Networking
STRATEGIC POTENCY ASSAY DESIGN FOR ALTERNATIVE DELIVERY MODALITIES
1:30 pm Speaking Position Reserved for Progen
2:00 pm (NEW DATA) Developing Novel Potency Assays for LNP-formulated Self-Amplifying mRNA to Enhance Drug Product Characterisation
Synopsis
- Comparing analytical methods assessing formulated vs naked RNA molecules
- Presenting data on a novel transgene expression ELISA used to quantify mRNA release and stability
- Discussing the analytical challenges associated with mRNA-LNP delivery platforms and relating these to critical CQAs
STREAMLINING INDUSTRY APPROACHES TO EMPTY-FULL RATIO CHARACTERISATION
1:30 pm PANEL DISCUSSION: Choosing a Method Of Empty-Full Characterisation. How Do We Ensure Industry Standardisation?
Synopsis
- Discussing best practice for empty-full characterisations, including AUC, HPLC, and ELISAs
- Considering the balance of cost, accuracy, reproducibility, speed, and precision
- Discussing whether methods ensure GMP during commercial upscaling
- Risk/Benefit ratio of investing in one vs multiple analytical tools
2:00 pm (CASE STUDY) (NEW TECH) Contrasting Data on Multiple Methods of Empty-Full AAV Characterisation to Better Streamline Industry Approaches
Synopsis
- Is AUC still the gold-standard technique for characterising empty-full ratios?
- Exploring SEC-MALS, Mass Photometry, IEX, Capsid/vg ratio, and A260/280 to mitigate the drawbacks of analytical ultracentrifugation
- Outlining data using CD-MS and Mass Photometry to establish their relevance as emerging tools
2:30 pm Afternoon Refreshments & Poster Session
REGULATORY GUIDANCE ON ANALYTICAL DEVELOPMENT
3:15 pm PANEL DISCUSSION: Unpicking The Complexities of Batch-Batch Variability to Enable the Setting of Feasible Thresholds & Avoid Regulatory Obstacles
4:00 pm (CASE STUDY) Where Is the Current Regulatory Focus? Key Insights Gained From Discussions With the Regulators
Synopsis
- Setting realistic expectations before entering discussions with regulatory experts
- Sharing regulatory perspectives on Validating Assays, Setting Standards, and Ensuring Data Integrity in Gene Therapy Analytical Development
- Highlighting how to interact effectively with regulatory authorities
4:30 pm Understanding the Changes in Regulatory Scrutiny Throughout the EarlyLate Stage Transition in Order to Optimise Phase-Appropriate Assay Design Strategy
Synopsis
- Discussing ICH guidelines during progression towards clinical trials
- Establishing regulatory perspectives on late-phase cGMP
- Ensuring appropriate data integrity and instrument qualification through the early-late stage transition to suit regulatory demands
- What are the most important factors to show regulators during AAV extended characterisation