9:00 am Registration and Morning Networking

9:30 am Chairperson’s Opening Remarks

LEVERAGING AUTOMATION TO OVERCOME VARIABILITY IN ANALYTICS

10:00 am Leveraging Automation of Analytics to Reduce Resource Burden and Improve Sensitivity and Efficiency of Analytical Data Generation

  • James Norton MSAT Analytical Development Lead, MeiraGTx Gene Therapy Facility

Synopsis

  • Explore the investments being made into automation of analytics including qPCR sequencing and library preparation automation Discover the advantages and limitations automation offers gene therapy developers in real world applications
  • Uncover how automation is overcoming capacity limitations and site variability
  •  Review how gene therapy developers and industry partners are working together to optimise workflow

10:30 am Best Approaches to Designing and Executing Risk Assessments for Transferring Technology Between Sites

  • Simon Briggs Head of Gene Therapy Technical Product Development, Biogen

Synopsis

  • Uncover how to build an effective risk assessment that determines the impact of changes in process development and the suggested actions to minimise variability across manufacturing sites

11:00 am DGE-AUC: Adapting the Power of Density Gradient Separations for Gene Therapy Analytics

  • Dr. Lutz Ehrhardt Senior Manager Field Marketing Centrifugation Europe, Beckman Coulter Life Sciences

Synopsis

  • Adapting gold standard CsCl isopycnic gradient purification methods to AUC
  •  How DGE-AUC compares to the “classical” methods
  • Achieve more with less math! Simplified data visualization and analysis – a walkthrough with Excel
  • Application of DGE-AUC in the Gene Therapy environment: AAV, adenovirus, plasmids

11:30 am Morning Coffee and Networking Break

BIOASSAYS & MOLECULAR BIOLOGY TRACK

ANALYTICAL ASSAYS FOR PURITY/IMPURITY MONITORING

12:00 pm Key Considerations for Developing a Highly Sensitive and Quality Control-Friendly CE-SDS Method for Monitoring AAV Purity

Synopsis

  • Exploring the key factors for CE method development on different AAV serotypes.
  • Method validation following ICH guidelines.
  • Use force degradation studies to determine if methods are stability indicating

12:30 pm Leveraging Host Cell Protein Detection and Quantification to Support Process Development and Risk Mitigation for Gene Therapy

  • Judy Shimoni Technical Development Lead, Pharma Technical Development Project and Portfolio Management, Genentech

Synopsis

  • Gene therapy manufacturing for clinical supply must ensure robust and consistent production of safe and efficacious products.
  • Removal of product- or process-related variants or impurities is required to meet the pre-determined specifications.
  • The ability to detect and identify residual impurities including host cell proteins is essential for risk assessment and mitigation.
  • Readily available immunoassays combined with advanced applications of orthogonal methods provide tools to support process development and quality control.

PHYSICOCHEMICAL TRACK

DEEP CHARACTERIZATION METHODS

12:00 pm Characterization of gRNAs and Ribonucleoproteins for CRISPR Applications

  • Steven Wolk Vice President, Analytical Chemistry & Site Head Boulder, Editas

Synopsis

  • gRNA sequences can form alternate intra-molecular and intermolecular folds, which can impact complexation with the Cas protein Formation is sequence dependent, and more likely for longer sequences
  • Manifestation also is condition dependent
  • RNPs represent another level of complexity due to noncovalent nature of complex
  • SEC, IEX, PAGE, IM data shows that the secondary structures formed by the guides impact the complexation
  •  Some method dependence of results, which is not surprising for these noncovalent complexes of large biomolecules

12:30 pm Assessing Critical Quality Attributes of Viral Vectors for Gene Therapy

  • Dr. Klaus Richter Leader Analytical Ultracentrifugation (AUC) Group, Coriolis Pharma

Synopsis

Several quality attributes of AAV-gene therapy vectors are critical, including particle size and concentration, protein stability, monomer/dimer content, quantification of impurities (e.g., host-cell proteins) and the differentiation between empty and full capsid. In this talk, data from different analytical techniques (incl. DSF, TEM, IEX and AUC) are shown and the benefits and limitations of each technique on measuring final DP or during the manufacturing or purification steps are discussed.

1:00 pm Presentation by Refeyn

Synopsis

1:10 pm Lunch

LEVERAGING ANALYTICS TO SUPPORT PROCESS DEVELOPMENT

2:30 pm Capillary Electrophoresis As A Platform Tool For Characterisation Of Gene Therapies

  • Bernd Innthaler Lead Separation Methods, Gene Therapy Process Development, Takeda

Synopsis

  • Capillary electrophoresis (CE) has established itself as a high-throughput method for process monitoring. With CE methods, AAV LVV and pDNA production can be easily monitored.
  • In this talk, an overview of implemented CE methods will be given and compared with established methods such as gel electrophoresis, UPLC, ELISA and AUC.

3:00 pm Determine How To Leverage Analytics To Support Process Improvements For Autologous Gene Therapies

Synopsis

  • Assess how analytical comparability strategies can be leveraged to strengthen process development
  • Discuss how analytics can support both process developers needs whilst also meet the health authority expectations

3:30 pm Afternoon Break

DISRUPTING ANALYTICS WITH NEXT GENERATION TOOLS

4:00 pm Manufacturing Challenges And Strategies For Autologous Gene Modified Cell Therapies

Synopsis

  • This presentation will aim to outline the challenges of commercial manufacture and release of Autologous Gene Therapies
  • The presentation will explain how solutions such as use of high throughput analytics will complicate the logistics whilst also increasing the cost of goods
  • The risks involved in the solution of technically upskilling the commercial workforce by means of apprenticeship schemes will also be challenged
  • Strategies of introducing mature solutions from sectors outside of Pharmaceuticals and Life Sciences will be discussed

4:30 pm Exploring Antibody Kinetics In A Mice Model After Treatment With An Igg Cleaving Enzyme Imlifidase

Synopsis

  • Imlifidase belongs to a group of proteases that specifically cleaves the hinge region of IgG, resulting in complete temporary removal of antibodies within a few hours
  • The use of a IgG cleaving enzymes as pre-treatment to gene therapies creates an antibody free window for the delivery of gene of interest, which may allow for the inclusion of patients with high titre of neutralising antibodies against the viral vector
  • Cleaving of IgG will result in one F(ab’)2 fragment and one Fc fragment. Here, we share our findings of the assays used to study the kinetics of antibodies after IgG cleaving with imlifidase in a mice model

5:00 pm Closing Remarks

5:10 pm Close of Conference