Cambridge Healthtech Institute’s 3rd Annual

Cell Therapy Analytics & Manufacturing

CMC and Characterization Strategies, Analytical Tools, Process Development, and Scale-Up

January 17 - 18, 2022 ALL TIMES PST

The rapid boom in cell therapy medicines must be met with the fast development of robust, scalable, and well-characterized processes and products. Cambridge Healthtech Institute’s 3rd Annual Cell Therapy Analytics & Manufacturing conference will bring together leading scientists from the biopharmaceutical industry, academia, and government to discuss unpublished, in-depth case studies, new technologies, assay on CMC strategies, characterization, critical quality attributes, analytical toolbox, process development and scale-up, and role of CDMOs in manufacturing of cell-based therapies. This conference is followed by a sister conference, the 3rd Annual Gene Therapy Analytics & Manufacturing.

Sunday, January 16

4:00 pm Conference Registration Open (Sapphire West Foyer)

Monday, January 17

7:00 am Registration and Morning Coffee (Sapphire West Foyer)


Session Room: Sapphire D

9:00 am Organizer's Welcome Remarks

Nandini Kashyap, MPharm, Conference Director, Cambridge Healthtech Institute

Bernardo Cordovez, PhD, Chief Science Officer and Founder, Halo Labs

Non-Radioactive Assays for Cell Therapies

Preet M. Chaudhary, MD, PhD, Professor & Chief Hematology & Director, Blood & Marrow Transplant, University of Southern California

Despite the success of CAR-T in blood cancers, there are several limitations of this approach, including toxicities, disease relapse in blood cancers, and lack of efficacy in solid tumors. Dr. Chaudhary’s talk will focus on the challenges facing the cell therapy field and describe a variety of luciferase-based assays that his laboratory has developed to engineer the next generation of cell therapies.

10:10 am

Characterization of CARs from the Cell Surface Combining Immunoprecipitation and Mass Spectrometry to Look at Post-Translational Modifications

Jenifer Kaplan, PhD, Principal Scientist I, Novartis Institutes for Biomedical Research

CAR T cells are engineered T cells expressing a chimeric antigen receptor (CAR) on the cell surface. CARs allow the T cell to engage an antigen on tumor cells and activate downstream signaling that leads to destruction of the tumor cell. Surface expression of the CAR is critical for therapeutic efficacy, but often differences in efficacy are observed between constructs that show similar surface expression. We developed a workflow using immunoprecipitation of the CAR from the cell surface to characterize post-translational modifications by mass spectrometry (IP-MS) to gain insights on differences in efficacy.

10:40 am Networking Coffee Break (Sapphire West Foyer)


11:00 am

GMP Release Tests  and Non-GMP Characterization for Multiple Phase I/II and III Clinical Trial

Riccardo Biavasco, PhD, Scientist, Analytical Development, Bluebird Bio

Cell-based gene therapy drug products require comprehensive analytical assays to characterize their safety and efficacy. This talk will focus on the GMP release tests implemented across multiple phase I/II and III clinical trials and the additional non-GMP characterization assays performed to address the heterogeneity of drug products. Correlative analyses between the results of different analytical assays will be discussed.

11:30 am

Starting Material Characterization Impact to Process Development and Manufacturing

Dominic Clarke, PhD, ISCT Process & Product Committee Co-Chair & CTO, Cell and Gene Therapy, Discovery Life Sciences

Cell and gene therapy processes and products are fundamentally complex. As cell-based therapies continue to be translated and advance through clinical trials, long-term success may hinge on our ability to gain greater process and product understanding through enhanced analytics. For cell-based therapies, the starting source material is a vital component to ensuring therapeutic success. The starting material is inherently variable which directly impacts downstream process development and manufacturing. More and more in-process analytical techniques are being developed and implemented to gain better process and product understanding to reduce risks of potential delays or translational success. This presentation will focus on donor-derived starting materials and the impact of characterization on process analytics, consistency, and overall product quality. 

Bernardo Cordovez, PhD, Chief Science Officer and Founder, Halo Labs

In all biological products, distinguishing aggregated API from other particle types matters for understanding the root cause of instability. Until now, subvisible particle characterization methods have been unreliable, slow, and difficult to use across many workflows. Introducing the Aura, a 96-well, low-volume, high throughput aggregate and particle imaging system can rapidly size, count, and characterize biological particles and identify them as proteins, non-proteins, cellular aggregates, or other types of molecules.

12:30 pm Session Break
James McDonald, PhD, Field Application Scientist, Nexcelom Bioscience

 The increased utilization of cells in biomanufacturing and as therapeutic products over the last decade has prompted the development and publication of two ISO Cell Counting Standards, ISO 20391 – 1:2018 and ISO 20391 – 2:2019. This talk will discuss 6 success factors for obtaining high-quality cell counting results based on ISO standards, with specific recommendations for the cell therapy manufacturing environment. 

1:10 pm Session Break


2:50 pm Find Your Table and Meet the BuzZ Sessions Moderator
3:00 pm BuzZ Sessions with Refreshments (Sapphire Foyer)

PepTalk BuzZ Sessions are focused, stimulating discussions in which delegates discuss important and interesting topics related to upstream protein expression and production through downstream scale-up and manufacturing. This is a moderated discussion with brainstorming and interactive problem-solving between scientists from diverse areas who share a common interest in the discussion topic. Continue to check the event website for detailed discussion topics and moderators.

BuzZ Table 5: Cell Therapy Drug Product Development

Bharathi Vellalore, PhD, Scientist, Biotherapeutics Drug Product Development, Janssen
  • Process considerations for manufacturing autologous and allogeneic cell therapy products
  • Drug product considerations for hematological malignancies and solid tumor indications
  • Clinical vs commercial supply chain needs: Integrated drug product design​

BuzZ Table 6: Topic to be Announced

Matthew B. Seefeldt, PhD, Executive Director & Research Instructor, Cell Therapy & Manufacturing, University of Colorado


4:00 pm

Challenges and Opportunities in Cell Therapy Formulation and Delivery

Bharathi Vellalore, PhD, Scientist, Biotherapeutics Drug Product Development, Janssen

CAR T drug products generally use commercial formulation media with complex, undisclosed stoichiometry of ingredients. The proprietary nature of the commercial formulation limits complete understanding of the impact of formulation excipients on product quality, safety and efficacy. In this talk, we present our formulation efforts to develop defined formulations for T and NK cells and also discuss the ability of formulation excipients to increase the product quality and recovery administration.

4:30 pm

Forced Degradation of Cell-Based Medicinal Products Guided by Flow Imaging Microscopy with Machine Learning: Explorative Studies with Jurkat Cells

Tim Menzen, PhD, CTO & Pharmacist, Coriolis Pharma Research GmbH

Analytical characterization and stability assessment of cell-based medicinal products (CBMPs) is challenging because of their intrinsic complexity. We submitted differently formulated Jurkat cell suspensions to thawing and shaking stress mimicking conditions to which CBMPs might be exposed from cell procurement to administration to the patient. Cell viability and concentrations of cells and debris particles were determined by flow-imaging microscopy and machine learning.

5:00 pm

Reprogramming Natural Killer Cells for Immunotherapy of Solid Tumors

Sandro Matosevic, PhD, Assistant Professor, Department of Industrial and Physical Pharmacy, Purdue University

Natural killer (NK) cell infiltration into and anti-tumor immunity against solid tumors is often low. Functional and metabolic impairment of NK cells is induced by the suppressive microenvironment of solid tumors due to, among others, hypoxia, metabolites, such as adenosine, and the expression of inhibitory NK checkpoints. Here, we discuss our work in redirecting NK cells to overcome immunosuppressive solid tumor by genetically rewiring their functional and immunometabolic responses.

Jiangbo Zou, Manager of Cell Engineering Product Department, Cell Engineering Product Department, GenScript Biotech

Lentivirus is a very common tool in cell biology, especially in immunology and cell therapy, acting as an effective strategy for cell editing. However, for some challenging cells (ex: T & B), there are still some struggles to achieve high transduction efficiency and insert the targets into genome stably. At GenScript, our proprietary lentivirus system provides intact and functional viruses customized according to research needs and accelerate drug discovery process.

6:00 pm Welcome Reception in the Exhibit Hall with Poster Viewing (Sapphire Ballroom)
7:30 pm Close of Day

Tuesday, January 18

8:30 am Registration and Morning Coffee (Sapphire West Foyer)


Session Room: Sapphire D

9:00 am

Chairperson's Remarks

Vincenzo Di Cerbo, PhD, Lead Scientist, Cell & Gene Therapy Catapult
9:05 am

Considerations for Plasmid DNA Used in Cell Therapy Manufacturing

Basak Clements, PhD, Associate Director, Material Science, Janssen Pharmaceuticals, Inc.

Plasmids are starting materials consisting of double-stranded, circular DNA and used to produce viral vectors in cell and gene therapy modalities. While the regulatory classification of plasmids for cell therapy has evolved in the recent years, necessary controls for plasmids remain to be an area of continued debate and definition. This talk will cover the key considerations and elements of control strategy for plasmids used in cell therapy manufacturing.  

9:35 am

Novel Analytical Assays for Characterisation of Lentiviral Vector Therapies

Vincenzo Di Cerbo, PhD, Lead Scientist, Cell & Gene Therapy Catapult

Lentiviral vectors are largely used as gene delivery vehicles for the generation of gene-modified cell and gene therapies for ex vivo treatment of cancers and rare diseases and for some in vivo applications. Manufacturing of lentiviral vectors and derived lentiviral-based cell therapies is complex and requires rapid and reliable analytical assays to measure product’s critical quality attributes and demonstrate consistent manufacturing, safety, and efficacy. Cell and Gene Therapy Catapult has developed a portfolio of assays for LV product characterisation. This presentation will showcase some examples of novel analytical solutions for the characterisation of LV-based products, which aim to reduce variability and turnaround time.

10:05 am Coffee Break in the Exhibit Hall with Poster Viewing (Sapphire Ballroom)
11:00 am

Gates Biomanufacturing Facility: The Experience of Building a Multi-Product Cell Therapy/Biologics Phase I cGMP Facility

Matthew B. Seefeldt, PhD, Executive Director & Research Instructor, Cell Therapy & Manufacturing, University of Colorado

The purpose of this talk will be to walk through the design and build-out of a multi-product GMP manufacturing facility in an academic setting. The talk would review both the technical design considerations as well as business components to ensure staffing and Phase I compliance.

11:30 am

AI and Laser Processing for Scalable Generation of Autologous iPSC-Based Cell Therapies

Marinna Madrid, PhD, Co-Founder, Cellino Biotech

Cellino’s platform combines label-free imaging and high-speed laser editing with machine learning to automate cell reprogramming, expansion, and differentiation in a closed cassette format, enabling thousands of patient samples to be processed in parallel in a single facility.

12:00 pm Sponsored Presentation (Opportunity Available)
12:30 pm Session Break
Chao Yan Liu, Senior Manager, Cell Biology, Thermo Fisher Scientific

Adeno-associated virus (AAV) has emerged as the leading platform for gene therapy; however, challenges exist in scaling up production to treat larger patient populations. The AAV-MAX System addresses key challenges by enabling scalable, high titer, and cost-effective production of AAV.  Here, we will discuss data on the use of AAV-MAX for production of AAV across multiple scales in shake flasks and bioreactors, as well as downstream purification strategies and analytics.

1:10 pm Close of Cell Therapy Analytics & Manufacturing