8:50 am Chairs Opening Remarks
REGULATORY GUIDANCE & FEEDBACK FOR ANALYTICAL DEVELOPMENT
9:00 am British Pharmacopoeia Advanced Therapy Medicinal Products Analysis Guidance
Synopsis
• Who the BP are, what we do (traditionally and in ATMP)
• Why we are creating these guidance documents (who they are aimed at, where to find them, usefulness)
• Contents of the published guidance documents (likely to be covered by a
• collaborator (not confirmed))
9:30 am Navigating The Regulatory Landscape Of GTPs in Europe
Synopsis
• Overview of current European regulatory landscape
• Key regulatory hurdles faced by analytical teams in Gene Therapy drug development
• What might future regulatory guidelines look like?
10:00 am PANEL DISCUSSION: Getting to Grips With Potency Assay Regulatory Guidance & Guidelines
Synopsis
• Current potency guidance is largely designed to address challenges for biologics, thus what trends or shifts are we likely to observe (which may not be stated in any kind of guidance)
• What is currently driving the trends of potency assay development and regulatory communications?
• Are we any closer to an industry wide standard for potency?
• For potency readouts what’s necessary and what’s sufficient?
• Examining a potential industry knowledge gap in terms of defining function
10:30 am Speed Networking
Synopsis
This session is a great opportunity to introduce yourself to the attendees that you would like to have more in depth conversations with. This session is the ideal opportunity to get face-to-face time with many of the brightest minds working in the Gene Therapy analytical field and establish meaningful business relationships.
11:00 am Morning Refreshments
BIOASSAY & MOLECULAR BIOLOGY TRACK
PHASE APPROPRIATE POTENCY ASSAY DEVELOPMENT
11:30 am (CASE STUDY) Functional Potency Assay Development to Support Late Stage AAV Gene Therapy
Synopsis
• Custom cell line implementation
• Assay optimisation and performance
12:00 pm (CASE STUDY)Potency Assay Development From Early Stage Through to Approval: Assay Development, Validation & Implementation Into Stability Studies
Synopsis
• Intro to Orchard and our technology
• Potency for ATMPs: an overview
• Method development, optimisation, qualification and validation
12:30 pm PANEL DISCUSSION: Addressing Phase Appropriate Potency Assay Development Challenges
Synopsis
• Discuss the fields most common and pressing challenges in translating your early phase potency assays into late phase assays
PHYSIOCHEMICAL TRACK
EMPTY FULL CHARACTERISATION
11:30 am ROUND TABLE DISCUSSION: AUC – The Gold Standard for Empty, Full & Partial Characterisation, But For How Long?
Synopsis
Despite well known challenges, analytical ultracentrifugation (AUC) is the industry’s current gold standard. Ultimately there exists a need for an orthogonal technique that is faster and easier to perform. This roundtable will seek to assess current characterisation techniques and determine what analytical approaches may provide a superior alternative.
12:00 pm (NEW TECH) Opportunities & Challenges Using Mass Spectrometry / Photometry for rAAV Vector Characterisation
Synopsis
• Overview and discussion of current mass spec and mass photometry methods potential future MS applications case study data
12:30 pm Speaking Position Reserved for Coriolis Pharma
1:00 pm Lunch & Networking
BIOASSAY & MOLECULAR BIOLOGY TRACK
POTENCY ASSAY MATRIX DEVELOPMENT
2:00 pm (CASE STUDY) QC-Friendly Potency Method Development, Bridging, & Life Cycle Strategies
Synopsis
Discuss the fields most common and pressing challenges in translating your early phase potency assays into late phase assays
2:30 pm (CASE STUDY) (NEW DATA) Potency Matrix Approach at BridgeBio Gene Therapy & the Development of an In Vitro Potency Assay for Galactose-1-Phosphate Uridylyl¬transferase (GALT) Enzyma
Synopsis
• In-Vitro Gene/Protein Expression and Functional Assays and the potency matrix approach at BridgeBio Gene Therapy
• Development of an In Vitro Potency Assay for Galactose-1- Phosphate Uridylyl-transferase (GALT) Enzymatic Activity in AAV-Transduced Cells
• The (GALT) enzymatic activity assay exhibited a high level of repeatability across multiple executions, demonstrating the utility of the assay in supporting lot release and assessing lotto-lot consistency
PHYSIOCHEMICAL TRACK
CHARACTERISATION OF PARTIALLY FULL CAPSIDS
2:00 pm Speaking Position Reserved for PROGEN
2:30 pm Filling The Gaps in Understanding the Content Of Viral Vector Particles by Using Multiplex Dpcr & HighThroughput Sequencing
Synopsis
• Moving from empty/full ratio towards understanding of capsid content
• Advanced multiplex dPCR approaches can help in quantification of genome integrity
• How can high-throughput sequencing, especially long-read sequencing help in characterisation of AAV samples
3:00 pm Afternoon Refreshments & Networking
HIGHLIGHTING NEW TECHNOLOGIES IN THE FIELD
3:30 pm (NEW TECH) (CASE STUDY) CE-Western Method Development to Support AAV Extended Characterisation
Synopsis
• Size and charge heterogeneity are critical quality attributes for biotherapeutics due to their potential impact on drug potency and stability
• The developed CE-Western based method allowed for the assessment and quantitation of individual VP charge heterogeneity under denatured and reduced conditions
• This method demonstrated great value in supporting process development and forced degradation stability study
4:00 pm Building Secure Knowledge & Data Foundations for Successful Filings of Advanced Therapies
Synopsis
• How do we build a knowledge management strategy which encompasses the requirements of the agencies
• What do digital data repositories need to do?