This session will address key challenges in the development, manufacturing, and supply chain logistics for autologous and allogeneic cell therapies. After viewing this session, participants will have a better understanding of:

• How to identify key development and manufacturing challenges
• Commercial scalability strategies for late-stage cell therapies
• Ways to drive innovation to accelerate the delivery of cell therapies to patients faster
• How, when and why to leverage state-of-the-art facilities
• The importance of end-to-end supply chain management

Speakers:

Maria Lopez
General Manager
Catalent Princeton

Trish Demko
Director, Case Management
Catalent

Viral safety and risks for cell and gene therapy, how to improve pathogen safety of cell and gene therapy.

Speaker:

Brian Buesing
Scientist, R&D
Asahi Kasei Bioprocess America, Inc.

• What are key safety concerns for genome-editing-based medicine?
• What are the gaps in measuring them?
• How we are applying long-read genomics to tackle these safety risks

Speaker:

Saumyaa Saumyaa
Senior Research Scientist
AstraZeneca

Tetracycline Enabled Self-Silencing Adenovirus (TESSA) - an adaptable solution for efficient rAAV manufacture

Efficient manufacture of recombinant adeno-associated virus (rAAV) vectors for gene delivery remains challenging. We developed a novel self-repressing helper adenovirus system entitled ‘TESSA’ for contaminant-free manufacture of rAAV. TESSA can be used for rAAV manufacture, either by using two TESSA vectors to deliver adenoviral helper functions, AAV rep and cap genes, and the rAAV genome into HEK293 cells, or wherein the rAAV genome is delivered via stable integration in the cell’s chromosome, plasmid transfection or infection with existing rAAVs. This flexibility of using TESSA enables researchers to produce cost-effective rAAVs, both scalable and rapidly, for clinic and pre-clinical use, including the generation of AAV-mediated homology-directed repair templates for gene editing.

Gene editing the B cell lineage

B cells are the major cell type mediating humoral immunity. Upon activation by cognate antigen B cells differentiate into plasma cells which produce high amounts of serum antibody. Their high protein production capacity suggest that this system could be harnessed by gene editing to express desired antibodies or other therapeutic proteins. We are developing approaches using CRISPR/Cas9 and recombinant AAV6 to efficiently edit primary B cells of various species as well as hematopoietic stem cells, the self-renewing cell type giving rise to B cells and all other blood cells. These proof of concept studies are targeted at developing treatments for HIV-1 and inherited diseases such as hemophilia.

Speakers:

Weiheng Su, DPhil
Principal Scientist
OXGENE WuXi ATU

Harald Hartweger, PhD
Senior Research Associate
Laboratory of Molecular Immunology
The Rockefeller University

Developing Technologies for Comparability To Improve Robustness

Speaker:

Maria Gianelli, PhD
Global Product Manager
Gyros Protein Technologies

Session Description Coming Soon!

Speaker:

Jared Auclair
Director Bioinnovation
Northeastern University