Cambridge Healthtech Institute’s Inaugural

Gene Therapy Analytics and Manufacturing

Strategies, Analysis, Process Development, and Scale-Up of Vector-Based Gene Therapies

January 21-22, 2020

Part of the Cell & Gene Therapies pipeline

Cambridge Healthtech Institute’s Inaugural Gene Therapy Analytics and Manufacturing conference will take an in-depth look at the challenges facing the formulation, characterization, analysis and scale-up of gene therapies. The conference will examine critical challenges facing the analysis, characterization, quality control and bioproduction process development and scale-up, of viral vector-based gene therapies such as AAV, lentivirus and retrovirus.

Final Agenda



1:00 pm Registration (Sapphire West Foyer)

1:30 Refreshment Break in the Exhibit Hall with Poster Viewing (Sapphire Ballroom)

Opportunities, Regulations, and Challenges
Aqua D

2:00 Chairperson’s Opening Remarks

Jim Richardson, PhD, Senior Science and Standards Liaison, Global Biologics, United States Pharmacopeia


2:05 Living in the World of RNAi Therapeutics

Muthiah (Mano) Manoharan, PhD, Senior Vice President, Drug Discovery, Alnylam Pharmaceuticals, Inc.

The approval of ONPATTRO® by the US FDA in 2018 paves the way for a new class of RNA-based medicines that can be available for needy patients soon. This talk will discuss current challenges and opportunities in RNAi delivery. Furthermore, we will discuss the case study of ONPATTRO, talk about the challenges faced during discovery and development and how they were overcome.

2:45Regulation and Challenges in Developing Vector-Based Gene Therapies

Heidaran_MoMo Heidaran, PhD, Vice President, Technical, PAREXEL Consulting, PAREXEL International

While it is assumed that regulatory approval often leads to commercial success, this may not necessarily become an overwhelming trend in the field of gene therapy. The major reasons for this potential disconnect will be discussed in my presentation, but full understanding of how these products work (knowledge of the product in context of applicable CGMPs) and how these products could be consistently manufactured at commercial scale sustainably remain to be fully demonstrated or established.

3:15 Strategy and Challenges for Developing Potency Assays for Cell and Gene Therapy Products

Radhika Raheja, PhD, Scientist I, Method Development and Qualification, Analytical Development
bluebird bio

Autologous cell and gene therapy products are currently being developed for the treatment of rare genetic diseases and multiple cancer indications. To ensure long-term vector and drug product quality, it is important to demonstrate manufacturing consistency. Here we discuss some of the challenges, considerations and strategies for generating potency assays to support regulatory submissions. 

3:45 Refreshment Break in the Exhibit Hall with Poster Viewing (Sapphire Ballroom)

Formulation Development and Stability
Aqua D

4:30 Lyocycle Development for AAV-Based Gene Therapy Applications- Challenges and Opportunities

Tabish_TanvirTanvir Tabish, MSc, Head of Formulation Development, Formulation, Fill and Finish, Takeda

Biopharmaceuticals show varying levels of stability in aqueous solutions for short periods of time. Lyophilisation is a technique commonly used to improve the stability profile of biomolecules through the removal of water resulting in the increasingly restricted mobility of the reacting species. The Factor IX (FIX) gene therapy product was formulated and lyophilized. A stability study was established with the lyophilized material to determine its stability profile at the accelerated temperature at 5°C.

5:15 Extended Q&A with the Speaker 

5:30 Close of Day

5:30 - 5:45 Short Course Registration (Sapphire West Foyer)

5:45 - 8:45 Recommended Dinner Short Course*

SC5: Protein Aggregation: Mechanism, Characterization, and Consequences - Detailed Agenda


Thomas Laue, PhD, Professor Emeritus, Biochemistry and Molecular Biology; Director, Biomolecular Interaction Technologies Center (BITC), University of New Hampshire

Kevin Mattison, PhD, Principal Scientist, Malvern Pananalytical, Inc.

*Separate registration required




7:45 am Registration (Sapphire West Foyer) and Morning Coffee (Sapphire West & Aqua West Foyer)


8:15 Chairperson’s Remarks

Lake N. Paul, PhD, President, BioAnalysis, LLC

8:20 USP Standards for Gene Therapy

Jim Richardson, PhD, Senior Science and Standards Liaison, Global Biologics, United States Pharmacopeia

This presentation will provide updated information on existing USP Standards relevant for developers of Gene Therapies, such as Chapter <1047> Gene Therapy Products as well as Chapter <1043> Ancillary Materials for Cell, Gene, and Tissue-Engineered Products. It will also cover USP’s development of new physical reference materials to aid developers of Gene Therapies.

8:50 Beyond Empty Capsids: Biophysics in Gene Therapy Manufacturing

Lake N. Paul, PhD, President, BioAnalysis, LLC

Currently the gene therapy space is underutilizing the power of biophysics, specifically Analytical Ultracentrifugation (AUC). Besides the quantitation of empty capsids, AUC can also be used to evaluate the physicochemical properties along establishing the CQA of the viral vector. From QC and IPC (decision point) perspectives, AUC can be an invaluable tool for evaluating the DP during the entire manufacturing process.

Wyatt-logo-New-Logo-only 9:20 Measure AAV Quality Attributes with SEC-UV-MALS-dRI

Chen_MichelleMichelle Chen, PhD, Vice President of Analytical Services, Wyatt Technology

In this presentation, we discuss a size exclusion chromatography (SEC) method coupled with UV, multi-angle light scattering (MALS), and differential refractive index (dRI) detectors to measure the following three important AAV quality attributes: total number of viral capsid particles, relative capsid content, and percentage of monomer or aggregates.

9:50 Coffee Break in the Exhibit Hall with Poster Viewing (Sapphire Ballroom)

10:35 Strategy in Building In-House Analytical Capability for Gene Therapy Products

kang_jichaoJichao (Jay) Kang, PhD, RAC, Director, Analytical Development, Amicus Therapeutics

Compared with the traditional biologic products, gene therapy products are extremely heavy in CMC development. With limited CMO capacity and high requirements for quality and timeline, it is critical for a serious gene therapy company to develop its in-house development and manufacturing capability. This presentation will share Amicus Therapeutics Inc.’s experience in building its in-house analytical development capability, including the key requirements, strategy, priority, and examples of success and lessons.

11:05 Analytical Ultracentrifugation to Assess Critical Quality Attributes of Viral Vectors for Gene Therapy

Richter_KlausKlaus Richter, PhD, Group Leader, Analytical Ultracentrifugation (AUC) Group, Coriolis Pharma Research

Viral vectors are far more complex than other biopharmaceuticals. As part of drug development, it is required to monitor infectious virus titers, total number of virus particles, filled and empty virus particles, the functional envelope surface proteins (if applicable), virus aggregation and viral particle morphology. The talk focuses on how analytical ultracentrifugation (AUC) can be integrated as a powerful method for assessing critical quality attributes of viral vectors.

11:35 Manufacturing Lentiviral Vector for Gene Therapy Against RAG1-SCID

Luitjens_AlfredAlfred Luitjens, Director Cell Technology, Operations, Batavia Biosciences

We will present a case study on the process development and subsequent GMP production of lentivirus-based gene therapy against severe combined immunodeficiency syndrome. Our partner, Leiden University Medical Center transferred a lab-scale process to Batavia Biosciences, which was subsequently implemented and further developed to a GMP production process to be able to deliver material for clinical trials.

12:05 pm Session Break

12:15 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

1:15 Session Break

Aqua Salon




The PepTalk Plenary Keynote Panel convenes a group of leading scientists working across novel therapeutic modalities and R&D technologies to explore the many challenges associated with discovering, developing, and advancing today’s novel biotherapeutics. The Panel, via a highly interactive format, encourages discussion among both the panelists and the audience members. Please come prepared with your questions and ideas for this spirited discussion.

  • Advances and challenges in expression and production for novel modalities
  • Implementing next-generation informatics: data collection, standardization, analysis, ML/AI, and considerations for IP landscape and protection
  • Implementing R&D and production capacity for gene and cell therapies – where are we heading?
  • Modality-specific challenges: multi-specifics for cancer, improving the ADC therapeutic window, improved safety and pharmacology, novel delivery/targeting
  • Preclinical and clinical development of drug combinations with focus in IO: How do we select the right combination dose so we can accelerate clinical development?


Mohammad Tabrizi, PhD, Senior Director, Pharmacology, Ascendis Pharma A/S




Edward Kraft, PhD, Senior Scientific Manager, Biomolecular Resources, Genentech



Ilya Shestopalov, PhD, Associate Director, Cell Analytics, bluebird bio



David E. Szymkowski, PhD, Vice President, Cell Biology, Xencor, Inc.



Alayna George Thompson, PhD, Senior Scientist I, Drug Discovery Science & Technology, AbbVie



3:05 Refreshment Break in the Exhibit Hall with Poster Viewing (Sapphire Ballroom)


4:00 Chairperson’s Remarks

Lisa Lundberg, Lead, QC Bioassay & Cell Culture, Spark Therapeutics

4:05 Monitoring and Control of Aggregates and Impurities in Large Scale AAV Production

Lisa Lundberg, Lead, QC Bioassay & Cell Culture, Spark Therapeutics

Monitoring and control of aggregates and impurities are important for successful large-scale production of gene therapies. In this presentation, we will discuss various aggregates and different types of impurities that can be encountered in a large-scale AAV production, what tools and techniques can be used to monitor and characterize them, consideration for setting specifications, etc.

4:35 Characterization and Quantification of Empty and Full AAV Capsids

Qin Zou, PhD, Associate Research Fellow and Group Leader, Analytical Research and Development, Pfizer, Inc.

This presentation can highlight various analytical methods for AAV empty and full capsids and emphasize the proper use of analytical ultracentrifugation for this purpose. AAV empty and full capsids is a product-related impurity and a critical quality attribute. Careful consideration of using appropriate analytical techniques to control that is important for a successful product.

5:05 Predicting Viral Clearance: DOE, HTS and AAV Case Studies Utilizing a Non-Infectious MVM Surrogate during Downstream Development

Cetlin_DavidDavid Cetlin, Founder & CEO, MockV Solutions LLC

Viral clearance studies are expensive and logistically challenging. This presentation will highlight data from the use of a non-infectious MVM surrogate in a variety of downstream applications and processes.


5:35 Selected Poster Presentation: Detection of AAV Capsid Proteins by CE-SDS as an Alternative to Silver Stain SDS-PAGE

April Blodgett, MS, LAT, Biotherapeutics Senior Sales Specialist, PerkinElmer, Inc.


SDS-PAGE followed by silver staining has typically been used to visualize the VP1, VP2, VP3 ratio.  This approach is labor and time-intensive but yields only qualitative data with poor reproducibility.  Here, we describe the use of microfluidic CE-SDS for the characterization of capsid proteins from AAV serotype 8 as the rapid, quantitative, reproducible alternative to SDS-PAGE with silver stain. 

6:05 - 7:00 Networking Reception in the Exhibit Hall with Poster Viewing (Sapphire Ballroom)

7:00 Close of Gene Therapy Analytics and Manufacturing Conference