This webinar is intended to provide the audience with an understanding on the following topics:

• Existing regulatory landscape, guidance and regulations applicable to cell and gene therapy products
• Hot topics related to manufacturing facilities to be considered during regulatory filings
• Segregation measures and controls pivotal for a multiproduct facility
• Lonza's regulatory capability and offering such as master files that sponsors can utilize to accelerate their regulatory timeline

Speakers:

Karen Magers
Head of Regualtory Affairs, Cell and Gene Technologies
Lonza Pharma & Biotech

Rajesh Thangapazham
Head of Regualtory Strategy, Cell and Gene Technologies
Cell and Gene Technologies Lonza Pharma & Biotech

This session will cover:

• Introduction to Immatics
• Development of compliant GMP manufacturing of TCR based T cell therapies in open systems
• Application and implementation of off the shelf, commercially available closed systems fit for every purpose for Immatics manufacturing including downstream processing technologies
• Other available off the shelf manufacturing closed systems that are ready to use for various purposes

Speaker:

Ali Mohamed
VP, CPC
Immatics

Session Description TBD

Speaker:

Ricardo Jorge Sousa da Silva
Senior Scientist
iBET, Portugal

Chimeric antigen receptor (CAR) -T cell therapy is a promising immunotherapy due to the ability to rapidly deliver a population of tumor specific T cells. Despite the impressive response rates in patients treated with FDA approved CAR-T cells, not all patients respond to CAR-T therapy. Studies have shown a correlation between central memory T cells and clinical response, whereas T cells from non-responders upregulate genes associated with effector differentiation, glycolysis, and apoptosis.

Therefore, elucidating the metabolic requirements that shift T cells toward higher efficacy will allow us to formulate T cell media that foster the production of therapies capable of converting non-responders into responders. One potential strategy to bolster in vivo persistence and efficacy is to “re-program” T cells during ex vivo manufacturing. Tools that will allow us to execute this strategy include our advanced metabolic and proteomic capabilities, a robust T cell expansion medium, and a deep understanding of how the workflow affects the cellular product. We have applied this approach in the development of a new T cell expansion medium that retains a more favorable phenotype, while increasing T cell growth. By combining optimally engineered cells and a “smarter” T cell medium with a fine-tuned workflow, we aim to improve T cell function, even in T cells from patients previously categorized as non-responders.

Speaker:

Evan Zynda
Research and Development, Bioproduction
Thermo Fisher Scientific

For many therapeutic cellular products, the development of a scalable cell expansion process optimized for quality and costs is a crucial step in their manufacturing process. In this case study, we optimized T cell expansion under stirred conditions using the automated, micro-scale ambr® 15 platform. In a first step, parallel experiments testing performance of various T cell media in the ambr® 15 microbioreactors and static well plates or T flasks revealed differences in media performance under agitated and static conditions. Performing a DoE approach using the MODDE software, further process parameters (pH, DO, stirring speed, Il-2 concentration, seeding density) were optimized. Based on optimal parameter settings determined in the ambr® 15 DoE studies, we successfully transferred agitated T cell expansion from ambr® 15 microbioreactors to larger scale, ambr® 250 modular bioreactors.

Participants of this webcast will learn how high-throughput tools such as the ambr® 15 system can be used as an early stage process optimization platform to define process parameter settings in a time- and cost-efficient manner. Furthermore, participants will gain knowledge about the benefits of DoE studies in process development and for further process transfer to larger scale bioreactors.

Speaker:

Dr. Julia Hupfeld
Scientist Regenerative Medicine PD
Sartorius Stedim Biotech

The success of gene therapies has increased the number of companies that are entering this arena and seeking advice on how they should produce their product candidates. FUJIFILM is developing a platform for AAV production.

Our platform will contain

1. A suspension HEK293 cell line
2. GMP grade Adenovirus helper
3. GMP rep/cap plasmid representing several AAV serotypes
4. A GOI plasmid for insertion of genes and
5. Tools for in-process and release testing.

Speaker:

Steve Pincus, PhD
Head of Science and Innovation
FUJIFILM Diosynth Biotechnologies

PLX (PLacental eXpanded) cells are placenta-derived mesenchymal cells that are designed to be administered intramuscularly to patients without the need for tissue or genetic matching. PLX are currently administered to patients in two Phase III clinical trials and have been safely administered to hundreds of patients resulting in an excellent safety profile across all ethnics. PLX and have shown positive clinical data in response to inflammation, ischemia/reperfusion injury, muscle trauma, irradiation and hematological disorders and shown the ability to modulate and support recovery of various organ systems by direct regeneration capabilities and modulating undesired inflammation, which plays a central role in the body’s response to injuries.

PLX showed significant therapeutic effect in animal studies of pulmonary hypertension, lung fibrosis and acute kidney injury, and gastrointestinal injury some of these are potential complications of the severe COVID-19 infection. PLX cells have immunomodulatory properties which include generation of regulatory T cells, and M2 macrophages, both might reduce the deadly symptoms of pneumonia and pneumonitis, caused by the COVID-19. On the other hand, PLX as an allogeneic cell, can activate Natural killer (NK) cells, and possibly CD8+ T cells, two cell populations which are important for the killing of viral-infected cells.

Due to the cells capabilities, we believe it could play a meaningful role in mitigating the tissue-damaging effects of COVID-19 infectious disease by boosting the immunological intratissue virus control. Pluristem is currently treating Covid-19 patients in Israel via “compassionate use” protocol and exploring regulatory pathways for a multinational clinical trial.

Speaker:

Racheli Ofir, PhD.
Vice President Research & Intellectual Property
Pluristem

The talk will give an overview of opportunities and challenges related to the Cell and Gene Medicinal product, main focus will be the manufacturing side of advance therapies including but not limited to:

• Sustainability and Manufacturability of Cell and Gene therapy product
• The critical impact of raw materials and related bottlenecks
• Challenges and opportunities for logistics and analytics
• Opportunity of “contaminations” from other industries

Speaker:

Stefano Baila
Director of Operations
Anemocyte

Rapid developments in cell & gene therapy have resulted in recent product approvals, bringing these transformative medicines to market to improve global health and well-being. Commercializing these products, however, have introduced a unique new set of challenges incl. consistent quality, continuity of supply, reducing contamination risks and open processing.

Learn how Avantor is enabling our customers scale therapies from R&D to patient delivery, faster and safer. Our experts will discuss why using cGMP chemicals matters in these applications, how to improve product sampling, and other developments in this innovative area in 2020.

Speakers:

Timothy Korwan
Director, New Product Developments
Avantor, Inc

Wayne Lynch
Product Manager
Avantor, Inc

Biopharmaceutical products, such as gene therapy products, therapeutic monoclonal antibodies and vaccines, are synthesised in bacterial or eukaryotic cells. Current purification techniques are not completely effective in eliminating host residual DNA (rDNA); hence these processes are a source of a range of potentially unsafe contaminates. Concerns exist surrounding the potential of these impurities to induce undesirable immunogenic or oncogenic host responses. The regulatory authorities including WHO have set a residual host cell DNA limit ranging between 100pg/dose to 10ng/dose depending on the cell line used as well as the mode and frequency of dosing. Given the requirements by the authorities for rDNA testing of biotherapeutic manufactured products, a sensitive, fast, and cost-efficient method of quantification is essential. The industry standard for detecting and quantifying rDNA in bio-manufactured products is quantitative PCR (qPCR). While the traditional qPCR offers broad dynamic range, the advent of the digital droplet PCR (ddPCR) technology offers higher sensitivity testing, with increased robustness and reproducible results, which are critical parameters for detecting low abundance DNA.

Intertek Pharmaceutical Services Manchester has a state-of-the-art Cell and Gene therapy lab which offers bespoke assay development and off the shelf validated qPCR and ddPCR testing in a GMP environment. In this presentation we will discuss our off the shelf HEK293 and E.Coli rDNA quantification assays, comparing the qPCR and ddPCR technologies in terms of sensitivity and reproducibility along with technical challenges

Speaker:

Sumaya Dauleh
Senior Analyst
Intertek Pharmaceutical Services