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Cambridge Healthtech Institute’s Inaugural

Cell Therapy Analytics and Manufacturing

Analysis, Process Development, Scale-Up, and Commercialization of Cell Therapies

January 23-24, 2020

Part of the Cell & Gene Therapies pipeline

Cambridge Healthtech Institute’s Inaugural Cell Therapy Analytics and Manufacturing conference will discuss opportunities, advances, and challenges facing the analysis and manufacture of autologous and allogenic cell therapies. The conference will discuss analytical, CMC, and quality control challenges of cell therapies. The conference will also discuss commercialization aspects, such as manufacturing, scale-up, bioreactors, next-generation production technologies, automation, supply chain, and other manufacturing-related issues.

Final Agenda

 

THURSDAY, JANUARY 23

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

Strategies, Regulations, and Guidance
Aqua d

8:10 Organizer’s Welcome Remarks

Nandini Kashyap, Conference Director, Cambridge Healthtech Institute

8:15 Chairperson’s Opening Remarks

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

 

KEYNOTE PRESENTATION

8:20 Manufacturing Challenges for the Commercialization of Cell and Gene Therapy Products

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

Achieving and meeting the requirements of manufacturing control for Cell and Gene Therapy product at commercial scale prior to licensure necessitates intensive focus and resources throughout the product development lifecycle. In my talk, I will attempt to highlight key challenges in consistent manufacturing of Cell and Gene Therapy products at commercial scale and provide possible solutions to some of these challenges, particularly as it is related to the collection of starting materials, establishing manufacturing control, and dealing with manufacturing changes during the product development lifecycle.

9:00 USP Standards for Cell Therapy

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

USP Standards Development for Cell Therapies - This presentation will provide updated information on existing USP Standards relevant to developers of Cell Therapies, such as Chapter <1046> Cell and 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 Cell Therapies.

9:30 Novel Luciferase Based Assays for Determining the Expression of CAR-T Cells and Cytotoxicity of Adoptive Cell Therapies

Chaudhary_PreetPreet Chaudhary, MD, PhD, Professor of Medicine and Chief Hematology, Blood & Marrow Transplant, Keck School of Medicine, Univ of Southern California

The talk will describe novel non-radioactive marine luciferase based assays (Topanga and Matador Assays) for detection of CAR-T cells and for measuring the cytotoxicity of CAR-T cells and other forms of adoptive cell therapies.

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

SOLID TUMORS
AQUA D

11:00 Reprogramming Natural Killer Cells for Immunotherapy of Solid Tumors

matosevic_sandroSandro 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.

11:30 PANEL DISCUSSION: Next-Generation Production Technologies and Process Development

Discussion Points:

  • Next-Gen Cell Therapy Pipeline
  • Next-Gen Processes for Better and Faster Optimization
  • Next-Gen Scale-Up and Production Technologies

Moderator:

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


Panelists:

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

Sra_KuldipKuldip Sra, PhD, Senior Director, Crispr Therapeutics


Kemp_KellyKelly Kemp, PhD, Director, Process Development, ViaCyte


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


Chaudhary_PreetPreet Chaudhary, MD, PhD, Professor of Medicine and Chief Hematology, Blood & Marrow Transplant, Keck School of Medicine, Univ of Southern California

12:00  Enjoy Lunch on Your Own

1:10 Ice Cream Break in the Exhibit Hall with Poster Viewing (Sapphire Ballroom)

Analytical Toolbox and CMC Strategies
AQUA D

2:15 Chairperson’s Remarks

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

2:20 Characterization and Qualification of Cell Banks and Other Critical Reagents Used in Potency Assays

Bowen_KellyKelly Bowen, M.S., Sr. Associate Scientist, Analytical Development, bluebird bio

Cells are highly critical and complex reagents that are the foundation for any cell-based assay. To ensure operational consistency and limit the variability in cell-based assays, cell banks and cell lines require stringent characterization and control strategies. This presentation will focus on strategies to characterize, qualify, and manage cell banks used in potency assays.  

2: 50 Analytical Methods and CMC for Cell Therapies

Sra_KuldipKuldip Sra, PhD, Senior Director, Crispr Therapeutics

For autologous cell therapy products, each patient is a product batch. Manufacturing is a very tedious and manual process. The urgency to release the product quickly to the patient is very high. The presentation will cover the implementation of rapid analytical methods to release the final product in the desired timeframe to patients.

3:20 Networking Refreshment Break (Sapphire West & Aqua West Foyer)

3:45 Outcomes of Characterization of CD34-Enriched Gene Therapy Drug Products

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

Cell-based drug products manufactured with lentiviral vectors (LVVs) require advances in analytical methods to characterize their safety and efficacy. This seminar will cover implementation of novel technologies to address unique challenges posed by gene therapy drug products. Approaches to identify drug product CQAs correlating with in vivo function will be discussed.

 

4:15 Exosomes as an Alternative for Cell Therapies: Novel Tools for Manufacturing Process and Characterization of Exosomes

AlAzzam_WasfiWasfi AlAzzam, PhD, Chief Scientific Officer, TechnoPharmaSphere LLC 

Cell therapy provides immense hope for the treatment of diseases and regenerating pathological organs, yet has been marred by issues surrounding the effectiveness, unclear mechanisms, and survival of the donated cells. As exosomes fulfill a critical role in cellular communication with targeted message content, they become obvious candidates for an extensive range of diagnostic and therapeutic applications. However, the development of such large molecules need fast, robust and scalable purification procedure with effective analytical characterization. 

4:45 CMC study of Viral Vector in CAR-T

Ben Xu, PhD, BD Director, Biologics Development, Genscript


5:15 Close of Day

 

FRIDAY, JANUARY 24

8:00 am Registration (Sapphire West Foyer)

8:00 BuzZ Sessions with Continental Breakfast

Protein therapeutics is a fast-growing global market. As the science improves, so does the complexity of the R&D organization. Ensuring product quality plus speed to market requires insights from stakeholders working across the stages of protein science R&D. Join experts representing this PepTalk pipeline, peers, and colleagues for an interactive roundtable discussion. Topics include highlights from the week’s presentations, new technologies and strategies, challenges, and future trends.

Click here for more details

Process Development, Scale-Up, and Manufacturing (Cont.)
AQUA D

9:00 Chairperson’s Remarks

Kelly Kemp, PhD, Director, Process Development, ViaCyte

9:05 Challenges of Scaling-Up Cell-Based Processes to Meet Pivotal Trial Requirements

Kemp_KellyKelly Kemp, PhD, Director, Process Development, ViaCyte

Product characterization and process understanding is critical when scaling a manufacturing process; for example, moving from a 2D to a 3D cell culture platform, to ensure a comparable, reliable, and robust manufacturing process. A review of the challenges, including flexibility of batch sizes to support increasing trial commercial demands, and separation of high-quality cells for cryopreservation, will be presented along with proposed solutions.

9:35 Cryopreservation Optimization of CAR T Drug Product by Utilizing a Stochastic Global Optimization Approach

Zhao_JohnJohn Zhao, MSc, Senior Associate Scientist II, Cellular Process Development and Gene Editing, bluebird bio

Cryopreservation is a complex process with multiple variables that can interact and impact cell viability and proliferation potential. The work at bluebird bio used a stochastic global optimization tool as a successful means to efficiently test a small number of conditions to improve the cryopreservation process (50 out of >500 combinations). 


10:05 Starting and Raw Material Quality for Successful Scale-up of Clinical and Commercial Manufacturing

clarke_dominicDominic Clarke, PhD, ISCT Process & Product Committee Co-Chair and Global Head, Cell Therapy, HemaCare

Consistency of the starting source material is a vital component to ensuring the success of cell and gene therapies. As allogeneic therapies transition to multiregional clinical trials, navigating the potential regulatory barriers for access and use of the starting material presents its own set of challenges. It is important to understand as early as possible the quality and level of compliance (e.g. cGMP) required globally.

10:35 Networking Coffee Break (Sapphire West & Aqua West Foyer)

11:00 Evaluating Cell Settling Velocity

Danika Rodrigues, MS, Senior Associate Scientist, Research & Development, Janssen Pharmaceutical, LLC

Cell settling can occur during dose preparation and administration of cell therapies, potentially compromising homogeneity of product suspension. A model that considered cell and fluid properties was used to calculate settling velocities for cell product suspensions under varied administration conditions. Model calculations show agreement with experimental velocities and can be expanded to serve as a predictive simulation tool. We intend to optimize this model for the evaluation of clinical preparation/administration procedures.

11:30 Interactive Buzz Session Report Out 

Cell and Gene therapies are a fast-growing field. As science improves, so does the complexities. Join experts representing this PepTalk pipeline, peers, and colleagues for an interactive roundtable discussion report out. Topics include highlights from the week’s presentations, new technologies and strategies, challenges, and future trends, which talks, and speakers motivated you, learnings during the week that you can apply in your work.

11:45 Conference Wrap-Up

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

12:00 Close of Conference


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