8:50 am Chair’s Opening Remarks

BUILDING A PHASE APPROPRIATE ANALYTICAL STRATEGY

9:00 am Combinational Product Development – Bridging Science With Compliance

  • Cynthia Brysch Senior Director - Quality Assurance, Sarepta Therapeutics

Synopsis

• Harmonised approaches to developing a successful global analytical strategy

• Quality based infrastructure development from concept to commercialisation

• Traversing the programmatic lifecycle (integrated approaches to the development of novel analytical methods and compedial applications)

9:30 am Measuring Strength of AAV Therapeutics

Synopsis

• Supporting drug discovery and drug development

• Quest for MoA-reflective methods

• Optimisation and automation of the infectious titre method

10:00 am Morning Refreshments

BIOASSAY & MOLECULAR BIOLOGY TRACK

POTENCY ASSAY STRATEGIC DEVELOPMENT

11:00 am (CASE STUDY) (NEW DATA) Quantity & Potency Assays for SVec, a Novel SV40 Based Viral Vector Platform

Synopsis

• Introduction to SVec as gene therapy vector and development of the VP2-qPCR assay to quantify vector genomes and the TCTD50-LUC potency assay to support initial development of the SVec production process

• Development of the Reverse transcriptase Q-PCR Potency Assay (RQPA), a relative potency assay for generic application.

• Product specific potency assays are presented for AMA005, Amarna’s lead product candidate targeting Hemophilia B

11:30 am (CASE STUDY) Potency Reference Standard Strategy: Laying a Clinical Foundation for Commercial Success

  • Shana Boyer Critical Reagent Lead, Spark Therapeutics

Synopsis

• Meeting regulatory expectations and overcoming the unique challenges presented by smaller scale clinical and commercial production

• Key aspects of planning and implementing a sound commercial two tiered reference standard strategy with a focus on robust potency methods

PHYSIOCHEMICAL TRACK

CHARACTERISING PRODUCT RELATED IMPURITIES

11:00 am Speaking Position Reserved for Bio-Techne

  • Chris Heger Director - Applications Science, Bio-Techne Corporation

11:30 am Analytical Methods of AAV Vectors for Process Development & Quality Control

Synopsis

  • Analytical methods for AAV for process development and for quality control will be introduced
  • Pros and Cons of each method will be explained
  • Current situation of analytical methods for AAV characterisations especially from scientific perspectives

12:00 pm Speaking Position Reserved for Refeyn

  • Racha Majed Sales & Applications Specialist, Refeyn Ltd

12:10 pm Lunch & Networking

ENHANCE YOUR GENOME SEQUENCING TO BETTER UNDERSTAND YOUR GENOME IDENTITY

1:00 pm (CASE STUDY) (NEW DATA) Quantitative Analysis of AAV Concentration and Integrity by Digital PCR

  • Jinpu yang Senior Manager - Analytical Development, Vertex Pharmaceuticals

Synopsis

  • Evaluation of multiple digital PCR platforms
  • Optimisation and automation of PCR-based AAV titering
  • Vector genome integrity by linkage analysis

1:30 pm (NEW TECH) (CASE STUDY) Genome Integrity Using DDPCR

  • Oscar Relf Expert, Science & Technology, Gyroscope

Synopsis

  • Definition of vector integrity: sequence variation or sequence fragmentation?
  • Analytical assays for the characterisation
  • Development of a 2D-ddPCR method for vector integrity determination

CHARACTERISING PRODUCT RELATED IMPURITIES

2:00 pm (CASE STUDY) Mass Spectrometric VP Characterisation of rAAV Gene Therapy Products

Synopsis

  • Intro to our LCMS approach for Intact mass, peptide mapping, and PTM profiling
  • Example of minor VP protein ID using combination of LC-MS and CE-SDS
  • Example of PTM profiling to support formulation selection

2:30 pm Afternoon Refreshments & Networking

MASTERING AAV INFECTIVITY

3:00 pm (CASE STUDY) AAV Infectivity & Its Role in the Potency Matrix

  • Cullen Mason Associate Director & Head of Gene Therapy, Bioassay Development, Biogen

Synopsis

• Development of a platform ddPCR-based method and comparability to TCID50

• Use cases for implementation and correlations to other potency readouts

3:30 pm (CASE STUDY) Relative Infectivity Assay Method

Synopsis

• Overview of current infectivity approaches

• REGENXBIO case study of relative infectivity approach, challenges and opportunities 

4:00 pm Chair’s Closing Remarks