Cambridge Healthtech Institute’s 2nd Annual
Cell Therapy CMC, Quality and Analytics
Part of CHI's Ninth Annual The Bioprocessing Summit
August 21-22, 2017 | Westin Copley Place | Boston, MA

Cell-based therapies are extremely complex modalities and very difficult to characterize. Cambridge Healthtech Institute’s Cell Therapy CMC, Quality and Analytics meeting focuses on the technical and regulatory requirements needed to advance the development of cell therapies, including CAR-T and TCRs, with in-depth case studies and regulatory feedback on CMC development, potency assay development and validation, flow cytometry, critical quality attributes, critical process parameters, and product release.

Final Agenda

Monday, August 21

8:00 am Short Course Registration Open

9:0011:30 Recommended
Morning Short Courses*

SC2: Comparability Strategies for Cell and Gene Therapies

* Separate registration required

11:30 Main Conference Registration Open


1:00 pm Chairperson’s Opening Remarks

Bernadette Keane, Ph.D., Consultant, Keane Consultancy

Challenges and Opportunities in Cell Therapy CMC: The Role of CQAs, In-Line Measurements, Flexible Automation and Standards

Krishnendu_RoyKrishnendu Roy, Ph.D., The Robert A. Milton Chair and Professor, Biomedical Engineering; Director, Marcus Center for Cell Therapy Characterization Manufacturing (MC3M), Georgia Tech

In this presentation, I will outline, using specific exemplars, how investment in identifying critical quality attributes (CQAs) and development of new technologies for in-line, rapid quality measurements would be critical for the field to move forward. I will also discuss how automation, especially flexible-adaptive automation and an industry-wide focus on developing pre-competitive standards will be key to our success in cell therapy CMC.


1:45 Regulatory Challenges and Strategies for Cell Therapies - US Perspective

Bernadette_KeaneBernadette Keane, Ph.D., Principal, Keane Consultancy

Cell therapies are substantially more complex than small molecule or biological approaches to medicine. This complexity poses challenges for both academic groups and companies developing cell therapies, as well as for regulators seeking to oversee this growing area of medicine. In this interactive session, we will discuss some of the common challenges and lessons learned along the way and explore how collaborations between industry and the regulators can help lead to successful translation and commercialization of cell therapies.

2:15 Regulatory Aspects of Manufacturing and Control of Genetically Modified Cells

Matthias_RennerMatthias Renner, Ph.D., Scientist, Federal Institute for Vaccines and Biomedicines, Paul Ehrlich Institute

In respect to manufacturing and quality control, genetically modified cells are considered to be most complex medicinal products. Regulatory aspects considering the fundamental steps in manufacturing and control of these medicinal products will be presented, and the regulatory framework for these products which are classified in the EU as advanced therapy medicinal products and are regulated centrally by the European Commission and the European Medicines Agency will be given.

2:45 Refreshment Break

3:15 Interview with the Regulators

Matthias_RennerMatthias Renner, Ph.D., Scientist, Federal Institute for Vaccines and Biomedicines, Paul Ehrlich Institute


Bernadette_KeaneBernadette Keane, Ph.D., Principal, Keane Consultancy


Isoplexis 3:45 Cell Therapy Product Characterization: Single Cell, Multiplexed Secreted Cytokine Analysis  

Jonathan Chen, Single Cell Evangelist, IsoPlexis

The characterization of cell therapy products and the ability to monitor product quality to support changes in the manufacturing process is challenging. This presentation introduces a new platform utilizing high parameter single cell secreted protein analysis to improve efficacy and immunotoxicity prediction.

 4:15 Key CMC Considerations for Cell Therapy Development and Approval

Allan_CallawayD. Allen Callaway II, MS, MBA, Associate Director, Global CMC Regulatory Affairs, Janssen (Pharmaceutical Companies of Johnson and Johnson)

Each cell therapy development program involves the difficult task of managing health authority expectations and evolving guidances, while also adapting to internal program challenges around manufacturing scale-up, comparability, and appropriate analytical method selection, in order to predict and control product quality for pivotal trials. In this presentation, we will focus on the key CMC considerations for early and late phase development.

4:45 Breakout Discussions

This session provides the opportunity to discuss a focused topic with peers from around the world in an open, collegial setting. Select from the list of topics available and join the moderated discussion to share ideas, gain insights, establish collaborations or commiserate about persistent challenges. Then continue the discussion as you head into the lively exhibit hall for information about the latest technologies.

Regulator Expectations for Cell and Gene Therapies

Matthias Renner, Ph.D., Scientist, Federal Institute for Vaccines and Biomedicines, Paul Ehrlich Institute

Bernadette Keane, Ph.D., Principal, Keane Consultancy

  • Cell therapy regulatory expectations throughout development
  • Common pitfalls
  • CMC Strategies for Expedited pathways
  • European vs. US expectations

The Role of CQAs, In-Line Measurements, Flexible Automation and Standards

Krishnendu Roy, Ph.D., The Robert A. Milton Chair and Professor, Biomedical Engineering; Director, Marcus Center for Cell Therapy Characterization Manufacturing (MC3M), Georgia Tech

  • Setting CQAs
  • Process analytics in cell therapy development
  • Flexible Adaptive Automation
  • Analytical Strategies

5:30 Grand Opening Reception in the Exhibit Hall with Poster Viewing

7:00 End of Day

Tuesday, August 22

7:30 am Registration Open and Morning Coffee


7:55 Chairperson’s Remarks

Christopher Bravery, Ph.D., Consulting Regulatory Scientist, Consulting on Advanced Biologicals Ltd.

8:00 Reliable Product Characterization by Flow Cytometry

Ruud_HulspasRuud Hulspas, Ph.D., Independent Consultant, Cellular Technologies Bioconsulting, LLC

As cellular therapy entered the commercial sector, reliable and accurate product characterization has received extra attention. Although flow cytometry allows for extensive characterization of millions of cells, it is difficult, expensive and can introduce significant variability. This presentation provides an overview of flow cytometry in commercial cellular therapy, and discusses a number of practices that contribute to a better balance between the advantages and disadvantages of flow cytometry.

8:30 Enumeration of CD34+ Cells by Flow Cytometry: USP’s Perspective

Huiping_TuHuiping Tu, Ph.D., Director, Global Biologics, Science & Standard, United States Pharmacopeia

What is the perspective of the enumeration of CD34+ cells from USP? USP Chapter <127> Flow Cytometric Enumeration of CD34+ Cells provides a single platform, standardized flow cytometric method for CD34+ cell enumeration based on ISHAGE protocol. The USP CD34+ Cell Enumeration System Suitability Reference Standard has been developed to assess the reagents and ensure the correct gating during data acquisition and analysis.


9:00 FEATURED PRESENTATION: Development of an Automated 28-Day Assay for T Cell Proliferation

Geoff_HodgeGeoffrey Hodge, Ph.D., CTO, Unum

Unlike traditional drugs, cell therapies like Unum’s antibody-coupled T cell receptor (ACTR) platform technology actively expand and change after administration. Since traditional process development tools geared toward characterizing a static product are insufficient to fully understand the potential of cell therapies, Unum has designed a high-throughput 28-day “stress test,” used to assess potential product and process changes, which reveals differences in performance not detected in short term in vitro assays.

9:30 Cell Therapy Meets Drug Delivery

Biju Parekkadan, Ph.D., Co-Founder, Director, and Office of Chief Scientific Officer, Sentien Biotechnologies, Inc.

9:45 Coffee Break in the Exhibit Hall with Poster Viewing

10:30 Regulatory challenges for analytical methods in cell therapy products

Xin Du, PhD., Executive Director, Regulatory Affairs, Advaxis

Meeting the regulatory requirements for cell therapy products is very important in the cell therapy drug development. Understanding the uniqueness of cell therapy manufacturing and the associated analytical methods and the relevant regulatory guidance could help establishing a successful drug development strategy. This presentation will review the relevant regulatory guidance and provide regulatory considerations for analytical methods in cell therapy products.

11:00 Development and Implementation of a Cell-Based Potency Assay for a Dehydrated Human Tissue Product

Rebeccah_BrownRebeccah Brown, Ph.D., Vice President, Global Regulatory Affairs, MiMedx Group, Inc.

Numerous research studies on dehydrated human amnion/chorion membrane have demonstrated that it recruits and modulates the activity of cells to regrow, remodel and revascularize wounds. To determine that each lot of tissue maintains biological activity, a cell migration assay was selected for development into a Quality Control assay. The automation and validation activities required for successful implementation of a potency assay for an intrinsically variable product will be discussed.

11:30 Potency Assay Development and Validation for Processed Human Nerve Allograft

Mark_FriedmanMark L. Friedman, Ph.D., Vice President, Regulatory Affairs and Quality Assurance, AxoGen® Corporation

The regenerative potential of peripheral nerve allograft is due, in part, to the laminin coating of intact endoneurial tubes. A three-dimensional organotypic assay has been developed and validated which has shown to be able to assess the inherent regenerative potential of peripheral nerve allografts. This methodology can readily assess the potential efficacy of peripheral nerve allografts and provide a method to measure adjunctive regenerative therapies with peripheral nerve scaffolds.

12:00 pm Cell and Gene Therapies: The Intricacies of Logistics on a Global Scale

James Connolly, Business Development Manager, Sales, World Courier, Inc.

An in depth look at the challenges and unique transportation needs when conducting clinical trials in the cell and gene arena. From early stage, Phase I trials through to commercialization, we will review how early planning and foresight can greatly increase the chances of success as scale up is needed through the life of the trial stages and into the commercial market.

12:30 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

1:15 Dessert Refreshment Break in the Exhibit Hall with Poster Viewing

1:55 Chairperson’s Remarks

Damian Marshall, Ph.D., Head, Analytical Development, Non-Clinical Operations, Cell & Gene Therapy Catapult UK

2:00 Advanced Techniques for Immunotherapy Product Characterization

Damian_MarshallDamian Marshall, Ph.D., Head, Analytical Development, Non-Clinical Operations, Cell & Gene Therapy Catapult UK

Autologous products such as gene-modified T-cell immunotherapies can have high levels of in-process variability which results in lower consistency of product manufacture. This presentation will demonstrate how advanced product characterisation and real-time in-process analytics can be applied to model variability and increase overall process control.


2:30 Autolus’ Approach for Early-Stage CAR-T Cell Production

Emma_ChanEmma Chan, Ph.D., Senior Scientist, Process Development, Autolus Ltd.

Chimeric Antigen Receptor (CAR) T-cell therapies have shown great promise in hematological malignancies and are being developed using new technologies to target solid tumors, with the potential to offer a cure. Autolus, a private company spun-out by University College London in 2014, is developing cutting edge T-cell programming and manufacturing technology. Our aim is to optimize T-cell production processes to enable widespread distribution and commercialization of CAR T-cell technology.

3:00 Manufacturing Control Strategies for Cell Therapies

Christopher_BraveryChristopher Bravery, Ph.D., Consulting Regulatory Scientist, Consulting on Advanced Biologicals Ltd.

For approval, it is necessary to demonstrate the manufacturing process is under control such that the product can be made at a consistent quality. To achieve this, the CQA need to be controlled through understanding and control of the critical process parameters and suitable in-process controls. The relative importance of these along with regulatory expectations will be discussed.

3:30 Refreshment Break in the Exhibit Hall with Poster Viewing

4:15 US Approval of Three Rapid Microbiological Methods for MACI Product Release

John_DuguidJohn Duguid, Ph.D., Senior Director, Research & Development, Vericel Corporation

Rapid detection of contaminants is essential for cell therapy products with short shelf lives. Integrating quality into the process through lot segregation, raw material qualification, environmental control, personnel training, and detailed procedures is critical because final results for conventional microbiological tests may not be available prior to product release or patient administration. US FDA approval of the MACI BLA in 2016 included three RMM product release assays for sterility, endotoxin, and mycoplasma.

4:45 A Practical Guide to Process Development – An Academic Perspective

Patrick_HanleyPatrick J. Hanley, Ph.D., Laboratory Facility Director, Cellular Therapy and Stem Cell Processing, Program for Cell Enhancement and Technologies for Immunotherapy, Division of Blood and Marrow Transplantation, Children’s National Health System

This presentation will focus on our experience translating cellular therapies from a basic science discovery at the bench into a Phase I clinical trial. Included in this presentation will be how to engage all members of the team, from clinicians to the manufacturing team, and design a system to best meet the needs of the process. These items include reagent qualification, cell selection, staffing, and product testing.

5:15 Close of Conference

6:008:30 Recommended
Dinner Short Course*

SC5: Potency Assay Development for Cell and Gene Therapy Products

* Separate registration required