Post-Conference Discussion Day

Thursday, December 3 2021 | 09.00AM - 4.00PM EST

A DEEP DIVE INTO CHARACTERIZING IMPURITIES  09.00am-12.00am EST

Recombinant adeno-associated viruses (rAAV) are extensively used as gene delivery vectors in clinical studies, and several rAAV based treatments have already been approved. Significant progress has been made in rAAV manufacturing, and large-scale vector production and purification methods have been developed. However, better and more precise characterization techniques of both the vector and the payload (typically the transgene) are still needed to guarantee the purity and safety of gene therapy products.

As such this discussion day will set to explore some of the following themes in a panel:

  • CQAs
  • Characterization of the payload
  • Characterization of Carrier
  • Characterizing the viral structure, presence of aggregates & presence of impurities

Yan Zhi, Director Analytical Development, Spirovant Sciences

Quantifying Residual Host Cell DNA in AAV

  • Importance of measuring residual host cell DNA as a Quality Attribute
  • Different approaches to measure encapsidated and total residual host cell DNA

Aishwarya Shevade, Staff Engineer, Regeneron

From R&D to QC - Establishment & Tech Transfer of Analytical Methods for Characterization of AAV Derived Gene Therapy Products

Kamila Pytel, Associate Director, Gyroscope Therapeutics

Residual Impurity Quantification & Clearance in AAV-Based Gene Therapy Product Development

  • Analytical methods with high sensitivity for residual impurity quantification
  • Purification methods for residual impurity removal

Yi Pu, Scientist, Biogen

 

This session will also discuss expectations of current regulatory guidance in accordance with product characterization & highlight why and how the results of characterization will inform the iterative refinement of production for yield, purity and potency of your vector.

Lastly this session will host a panel to discuss why current analytical methods to measure these attributes commonly suffer from long turnaround times or low throughput for process development, and what needs to be done in order for rapid, high-throughput methods to be developed and commercialized.

BEYOND AAV VECTORS: ANALYTICAL STRATEGIES AND COMMON HURDLES 1.00pm-4.00pm EST

This discussion day will be dedicated to dissecting analytical hurdles and challenges for those gene therapy drug developers outside of the AAV bubble. There are currently major hurdles prohibiting quantitative assessments of LV and LNP products because current analytical tools for both often are low throughput and lack robustness and standardization is still required. This discussion day will mainly look to focus on gene therapy drug development utilizing both Lentiviral and Lipid Nano-Particle (LNP) approaches.

In light of this, the discussion day is set to cover:

PANEL: Common Analytical Pitfalls & Challenges Associated with Working on LV & LNP-Based Gene Therapy Products

  • What are the industry’s most common analytical hurdles?
  • How can they be overcome?

Session Leaders:

Nolan Sutherland, Scientist, bluebird bio
Luigi Barbarossa
, Senior Research Associate, AVROBIO
Taylor Zhang
, Senior Scientific Director, BMS

Best Analytical Approaches to Determine Lentiviral & LNP CQAs

  • Physiochemical & Identity
  • Potency & Bioactivity
  • Safety & Microbial Purity
  • Product & Process Impurities

Analytical Strategies for Characterizing Residuals in Autologous Chimeric Antigen Receptor (CAR) T-Cell Therapy

  • The manufacturing of autologous CAR T-cell products starts with highly heterogeneous leukapheresis materials from patients and involves the use of various raw materials.
  • This complex process may result in a formulation containing process- and product-related residuals.
  • A phase-appropriate, risk-based analytical development strategy for possible residual characterization will be presented.

Taylor Zhang, Senior Scientific Director, BMS

Lentiviral Characterization: An Overview of Analytical Approaches to Characterize LV:

  • Aggregates
  • Impurities
  • Stability & Formulation

The Importance of Novel Technologies in Clinical Assays 

  • Generation of calibrators and ddPCR bioanalytical assay development for accurate vector copy number assessment in gene therapy drug product

Luigi Barbarossa, Senior Research Associate, AVROBIO

Transduction Assays for LV & LNP

  • Analytical approaches to determine how many copies of your transgene are delivered to the target cell