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

  • David Dobnik Senior Research Associate, National Institute of Biology

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

  • Huriye Dagdas MSAT Protein Group Leader - Gene Therapy, Meira GTx

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?

  • Nasser Sadr Senior Director - Global Analytical Development, uniQure
  • Asa Curry Principal Scientist – Quality Control, Freeline Therapeutics
  • Marco Thomann Principal Scientist - Gene Therapy & Technical Development Analytics, Roche
  • Mojca Janc PhD student, National Institute of Biology
  • Paul Devine Associate Principal Scientist, AstraZeneca

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

  • Shreya Ahuja Senior Principal Scientist - Analytical Development, Eli Lilly

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

3:30 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

  • Uditha deAlwis Vice President, Analytical Sciences, Sarepta Therapeutics

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

5:00 pm Chair’s Closing Remarks

End of Day 1

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