Newsletter | April 29, 2021

04.29.21 -- An Analysis Of The Gene Therapy Viral Vector Landscape

 
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How Single-Use Technology Will Be Critical To The Commercialization Of Cell And Gene Therapies

Scaling-up cell and gene therapies from R&D to commercialization creates challenges for manufacturers. Single-use solutions for in-process sampling can have a tremendous effect on workflow efficiency.

Cut It Out: The Role Of Gene-Editing Technologies In Combating HIV

Despite the public attention and large population impacted by HIV, developing a cure has been particularly difficult. Will gene-editing techniques, such as the CRISPR-Cas9 system, change the paradigm?

Krabbe Disease Successfully Treated With Gene Therapy In Preclinical Animal Model

Krabbe disease is an aggressive, incurable pediatric neurodegenerative disease caused by mutations in the galactosylceramidase (GALC) gene. The standard of care for presymptomatic babies is hematopoietic stem cell transplantation (HSCT); however, the morbidity and mortality of HSCT is high. A recent study published in The Journal of Clinical Investigation reports the successful treatment of Krabbe disease in a preclinical animal model using gene therapy.

Navigating The Adoption Of Continuous Pharma Manufacturing Amid Unprecedented Global Challenges

Driving a path forward for continuous manufacturing calls for a closer look at current global challenges, any existing barriers, and the ideas and initiatives necessary to fulfill its potential.

Closed And Automated CAR T-Cell Lentiviral Transduction By Spinoculation

Although equipment is available for most workflow steps of CAR T-cell therapy manufacturing, other steps, such as lentiviral transduction for gene transfer, need improved methods and technologies. Here, we present two user cases as examples of using SpinOculation C-Pro protocol software for lentiviral vector transduction in different CAR T-cell workflows.

Optimizing Successful Development Of Viral Vector Gene Therapies, Gene Therapy Trials, And Companion Diagnostics

Despite risks associated with gene therapy, the field is growing as a promising treatment for various diseases. In this paper we provide a general background of the gene therapy process and discuss hurdles to the development of viral vector gene therapies, with specific considerations for adeno-associated virus (AAV)-based gene therapies, challenges to initiating a clinical trial with a gene therapy, and how Precision for Medicine can help develop gene therapy programs from bench to bedside.

Allogeneic T-Cell Therapies: Efficient Commercial Manufacturing Readiness Using “Manufacturing By Design” Methodology

Contract development and manufacturing organizations need to play a critical role in the scale-up and industrialization of cellular therapies by providing customized tools and processes. The “Manufacturing by Design” approach for addressing the industrialization of cell therapies allows for optimal production conditions.

Advancing Cell And Gene Therapies

Cells used for cell and gene therapies pass through several process steps and must be managed as well as transported between these stakeholders in a fast, sterile, and seamless manner.

The Benefits Of Choosing A Suspension System

With more gene therapy manufacturers needing to increase their batch sizes and efficiency, it’s necessary to invest in a viral vector manufacturing system that can be scaled up to meet commercial demand. In this infographic, learn how suspension systems offer many benefits that will help viral gene therapy developers enhance their processes and capabilities to better meet patient demands.

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