Last week we celebrated cell and gene therapy with an entire week of cell and gene therapy related articles. In case you missed it, I’ve highlighted the articles here along with some of our favorite cell and gene therapy pieces from this year.

Article Highlights

In-Process Analytical Methods are Critical in Gene Therapy Development

In gene therapy manufacturing, evaluating viral vector stability and integrity is critical in process development prior to production scale up. Process changes can affect critical quality attributes and as a result any process changes need to be carefully evaluated to determine the impact on viral particle size and intactness to ensure final product quality…

Semi-automated Closed CAR-T Manufacturing Process

CAR-T cell-based gene therapy technology is one of the most exciting new medical developments, with two of the most popular examples being the cancer immunotherapies Kymriah^® and Yescarta^®. These cell-based gene therapies have been able to provide unprecedented remission rates and have demonstrated success where other therapies have failed. Even with impressive clinical success, there are challenges with the costly and complex manufacturing of these patient specific therapies. While much of the biotherapeutic industry is moving toward strategies to reduced cost such as single-use equipment, increased automation, and closed processes, most cell therapy manufacturing still contains open workflows and manual steps…

Mustang Bio’s gene therapy has tremendous potential to cure X-SCID or Bubble Boy Disease based on study results from St. Jude

In this podcast, we interviewed Dr. Manny Litchman, President and CEO, Mustang Bio and Dr. Knut Niss, Chief Technology Officer, Mustang Bio about the exciting study results for their gene therapy candidate to treat X-SCID, why this disease is a good fit for gene therapy and next steps…

There’s a New CAR in Town: CAR-Expressing Natural Killer Cells

Discovered in the early 1970s², NK cells are large granular lymphocytes and potent cytolytic effectors of the innate immune system. Descended from the common lymphoid progenitor, which also gives rise to B and T lymphocytes and dendritic cells, NK cells are the body’s first line of defense against infections and tumor formation. Unlike T (and B) cells, NK cells are able to mount a rapid immune response against cancer and virus-infected cells without prior sensitization, which is how they became known as “natural” killers. NK cells achieve this by releasing cytotoxic proteins, primarily granzymes and perforin, stored within secretory lysosomes, which induce target cell lysis. They can also release a host of cytokines that recruit other immune cells to the site of infection³. Their ability to target cells that are missing the “self” MHC (major histocompatibility complex)-1 markers is unique because harmful cells that lack or downregulate their MHC1 markers go undetected by other immune cells (like T cells) ^3.4. This HLA (human leukocyte antigen)-independent mechanism makes NK cells very attractive for cancer immunotherapy…

Novel Acoustic Platform Provides Gentler, More Efficient Solution for Cell Processing

Cell and gene therapies are in need of evolving manufacturing technologies to move products from research, through development and into, commercial manufacturing. As more cell and gene therapy products achieve clinical success, more investments are being made in creating manufacturing technologies to support the industry. There are several advancements in this area including the closing and automating of many manufacturing processes and increasing efficiencies with the creation of fit for purpose equipment and consumables. There are still, however, many needed improvements to further increase productivity and reduce cost…

End-to-End Integrated Manufacturing Solutions Enable Gene Therapy Commercialization

With clinical success and increased investment from the market, many gene therapy companies are looking toward manufacturing and commercialization of their lead therapies. As gene therapy commercialization is relatively new, there are still several challenges that must be considered when looking at how these products will be manufactured consistently, at appropriate scale, and cost effectively…

Transfection Reagent Portfolio Enables Seamless Scalability from R&D to Commercial Therapeutic Viral Vector Manufacturing

Cell and gene therapies are achieving greater success in the clinic and as a result there is an increasing need to transition manufacturing from research scale to clinical and ultimately to commercialization. Critical to the manufacture of many gene-based therapies is the production of large quantities of recombinant viral vectors. Two commonly used recombinant viral vectors, Adeno-Associated Virus (AAV) and lentivirus, are mainly produced using PEI-mediated transient transfection in adherent and suspension HEK-293 or HEK-293T cells…

Viral Vector Manufacturing for Gene Therapy – Developing a Platform Process

In this podcast and accompanying article, we interviewed Dr. Mats Lundgren, PhD Customer Applications Director, Life Sciences, GE Healthcare, Sweden about developing platform processes for viral vector manufacturing and Mats describes a recently developed platform process for adenovirus production…

Expediting Cell and Gene Therapy Workflows

In this podcast and accompanying article, we interviewed Mr. Dave Eansor President of the Protein Sciences Segment at Bio-Techne and Dr. Sean Kevlahan, Senior Director of Cell and Gene Therapy at Bio-Techne about new technologies that can be implemented to expedite cell therapy and gene therapy workflows and facilitate the path from bench to clinic…

Scalable Viral Vector Upstream Process for AAV Gene Therapy Manufacturing

Production of viral vectors, particularly the efficient scale up of manufacturing systems, is one of the biggest challenges that the industry faces today. Généthon, a gene therapy company looking to move into clinical trials with a scalable high-capacity and cost efficient process for viral vector manufacturing, partnered with Pall on a project to assess the iCELLis^® single-use, fixed bed bioreactor system for both viral vector production and scalability…

Key Considerations for Gene Therapy Manufacturing for Commercialization

Gene therapy is the use of a gene-modifying technology to repair, replace or correct damage in the body. The first approved gene therapy study was conducted by the National Institutes of Health (NIH) in 1989 and provided evidence for the first time that human cells could be genetically modified and returned to the patient without harm. To date, approximately 2,600 clinical trials and six gene therapy products have been approved in various countries…