8:30 am Registration and Morning Networking

8:55 am Chairperson’s Opening Remarks

DEFINING THE UPDATED REGULATORY AGENCY REQUIREMENTS FOR GENE THERAPY ANALYTICS

9:00 am The Regulatory Expectations For Acceptance Criteria And For Demonstrating Extended Characterization Of Gene Therapy Products

  • Axel Ståhlbom Current Member, EDQM Gene Therapy Products working party

Synopsis

  • Determine how regulators define what constitutes a Gene Therapy Medicinal Product (GTMP)
  • Discuss how regulators are thinking around extended characterisation studies and their relevance for release testing and criteria of a GTMP
  • Discuss how much data is considered ‘sufficient’ to effectively demonstrate safety, efficacy, quality and consistency of GTMPs
  • Determine the regulators perspective on creating gold standard analytical methods to support with benchmarking activities

9:30 am Panel Discussion: Are We Any Closer To Creating A Gold Standard Amongst A Landscape That Is Rife With Orthogonal Methods?

  • Ernest Milian CMC Manager, VCN Biosciences
  • Niamh Kinsella Global Regulatory CMC Early Development Gene Therapy Lead, Biogen
  • Sol Ruiz Head of Biologics, Biotechnology, and Advanced Therapies, Spanish Medicines Agency (AEMPS)

Synopsis

  • In a landscape where different methods that measure the same attributes generate variable data, how can we determine which methods are considered acceptable and preferable?
  • This panel discussion will explore if and how specifications can be created to differentiate orthogonal methods and identify gold standards

10:15 am TEM Analysis for Insights on AAV Full/Empty/Intermediate Capsid Ratios and Other Process-Critical Quality Parameters

Synopsis

  • Transmission electron microscopy (TEM) image analysis provides unique visual insights and capabilities to identify structural parameters related to important biological functions for quality control, efficacy, and safety
  • We present how GMP-compliant TEM analyses can be applied from preclinical stages to commercial manufacturing in gene therapy development, including specific examples of cryoTEM characterization of full/empty/intermediate capsid ratio in AAV products

10:45 am Speed Networking

Synopsis

Join us for face-to-face networking. We will pair you up with fellow attendees so you can get one on one time with the brightest minds working in the gene therapy field in order to establish meaningful business relationships to pursue for the rest of the conference.

11:30 am Morning Coffee Break

BIOASSAYS & MOLECULAR BIOLOGY TRACK

A SHIFT TOWARDS

FUNCTIONAL ASSAY DEVELOPMENT

12:00 pm AV Antibodies for Capsid Variants?

Synopsis

  • Bottleneck: AAV antibody affinity
  • AAV capsid variants – considerations for ELISA development
  • Reliable standards for AAV variants

 

12:30 pm Determine The Need For Alternative Potency Assay Strategies in the Case of Nucleic Acid Based Therapeutics – The Matrix Approach

Synopsis

  • Potency assays: regulatory enviroment, classical approach, business need and specific challenges for nucleic acid based therapeutics. A potential strategy is the matrix approach

PHYSICOCHEMICAL TRACK

APPROACHES TO STRENGTHEN VIRAL AND GENOMIC TITRE QUANTIFICATION

12:00 pm Considerations for Implementing New and Improved Genome Quantification Methods for Late- Stage Clinical Development

  • Xiaohui Lu Senior Director, Analytical Development, Ultragenyx

Synopsis

  • Leverage the insights of gene therapy developers who are exploring the use of ddPCR methods to accurately quantify transgene expression
  • Case studies on clinical dosing decision during method changes

12:30 pm The latest innovations in gene therapy product characterization using capillary electrophoresis and LC-MS

Synopsis

1:00 pm Lunch

REINFORCING POTENCY ASSAY DESIGN

ASSAY DESIGN

2:30 pm Developing Functional Analytical Assays for the Characterisation of Dual AAVs

Synopsis

  • The dual vector approach consists in dividing the gene of interest in two parts, one vector encoding the 5’ sequence and the other the 3’ sequence.
  •  In vitro bioassays to demonstrate the dual otoferlin vector expresses full-length human otoferlin mRNA and protein.
  • Determination of the optimal ratio vector for efficient reconstitution.
  • In vivo functional assays to show the recovery of auditory function

3:00 pm How Critical are Critical Quality Attributes of AAVBased Gene Therapy Products (GTP) In Terms Of Product Potency

Synopsis

  • Unravel the significance of identifying various CQAs of AAVbased GTPs throughout the clinical development pathway that can potentially impact the potency of the product

NEXT GENERATION TECHNOLOGIES FOR EMPTY/ FULLY CHARACTERIZATION

2:30 pm Wyatt Technology – The Light Scattering Toolkit for LNP and Virus

Synopsis

  • The presentation will show how light scattering can be used to measure various quality attributes for gene therapy drugs
  • This will be illustrated by the example of AAV and LNP’s, showing different techniques from analysis with DLS to separation via SEC-MALS and FFF-MALS
  • The advantages and disadvantages of the different methods are listed, and it is shown how light scattering is ideally used for gene therapeutics

3:00 pm Exploring The Advantages of Flow Cytometric Analysis of Empty:Full Capsids

Synopsis

  • Review how flow cytometry is being utilized for empty full analysis and the advantages of this approach
  • Further analysis of viral vectors using flow cytometry/flow virometry

3:30 pm Skip the hassle for AAV empty/full and titer data with Stunner

Synopsis

Getting a quick answer on AAV titer, empty/full ratio, and aggregation state is critical to most decisions in the lab. Stunner’s dye-free, label-free, and standard-free AAV Quant delivers all that data using just 2 µL of sample, in less than a minute – way faster than AUC, ELISA and ddPCR. In this session, we’ll showcase studies and published data so you can see how Stunner can accelerate your AAV characterization.

4:00 pm Afternoon Break

4:01 pm
IMPROVING SPECIFICITY AND EFFICIENCY OF ANALYTICAL ASSAYS

4:30 pm Identifying mRNA production related impurities with a HPLC analytical platform

  • Blaz Bakalar Product Manager, PATfix solutions, BIA Separations Sartorius

Synopsis

  • Optimizing the IVT reaction for a high mRNA yield requires construct-specific considerations. While the IVT reaction might seem simple compared to cell lysates, containing mostly nucleotides and their by-products, effective IVT and subsequent purification process development needs to separate & identify these species, so they can be tracked & removed. The PATfix mRNA analytical platform is specifically designed to tackle this task.

5:00 pm Evaluating The Analytical Tool Box for Accurate Determination of Empty/ Full AAV Particles

Synopsis

  • Here we present a range of techniques that have been explored for the accurate determination of empty/full AAV particles, detailing the advantages each methodology brings

5:30 pm let’s go digital: Droplet Digital PCR as a part of your cell and gene therapy analytical toolbox

  • Caroline Weydert PharmD PhD, EMEA Sales Specialist Genomics - Biopharma, Bio-Rad Laboratories N.V.

Synopsis

Droplet Digital PCR (ddPCR) is becoming the standard for viral vector titer determination. Absolute quantification, with great precision, accuracy and reproductibility, makes it an excellent technique for this purpose.

But that’s not all, you can get a lot more out of ddPCR. Genome integrity, copy numbers, transduction efficiency, host cell detection, transgene expression and mycoplasma testing with ddPCR could become a part of your analytical toolbox.

6:00 pm Closing Remarks

6:10 pm Close of Conference