Completed Heart, Stroke & Blood Infection & Immunity

Large scale lentiviral vector production

In plain English

AI plain-English summary

Lentiviral vectors—harmless viruses engineered to carry therapeutic genes—can permanently rewrite the genetic code of a target cell, but no one can yet manufacture them at the scale needed for treating thousands of patients. These vectors are the most efficient, safe, and non-toxic tools available for permanently modifying both dividing and non-dividing cells, making them essential for cell and gene therapies. The problem is that current production methods work only for small batches—enough for a lab experiment or a handful of patients. This manufacturing bottleneck now blocks clinical trials and commercial use of dozens of therapies in development. If this project succeeds, it will create a large-scale manufacturing process for lentiviral vectors. That would remove a critical barrier to bringing gene and cell therapies—for conditions such as inherited immune disorders, certain cancers, and blood diseases—from the lab bench into routine clinical use. The impact would be felt not in daily life directly, but in the supply chains and production pipelines that quietly underpin modern medicine.

View original technical description
Lentiviral vectors (LV) are remarkable in their ability to insert their genetic payload into a target cell's genome. This affects a permanent genetic change in the target which is propagated through to its progeny. While there are several means of genetic modification, few are capable of permanent modification of target cells. Amongst all vectors for gene delivery, LV are unique in unparalleled efficiency, safety, lack of toxicity and ability to modify non-dividing target cells. They have therefore come to be recognised as a key reagent required for the efficient development of the burgeoning cell therapy, as well as gene therapy industries. However, although LV represent a well understood and robust technology, there is no manufacturing methodology for very large-scale LV production. This is now an acknowledged bottle-neck both for clinical trials and for commercial exploitation of many cell and gene therapy products in current development. We propose to address this unmet need

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Researchers

Adrian Thrasher (Co-Investigator)Martin Pule (Principal Investigator)Pamela Tranter (Co-Investigator)Waseem Qasim (Co-Investigator)Yasuhiro Takeuchi (Co-Investigator)

Related Research

Grants with similar aims, by meaning.

21EBTA Novel Engineering Biology Solutions to the Production of Lentiviral Vectors by In vitro Assembly for Gene Therapy
Development of a stable, continuous lentiviral (LV) production system for improved gene delivery and cell function
Establishment of high-titre, stable LV producer cells with high-level genome RNA
Continuous Manufacture of Lentiviral Vectors for Therapeutic Vaccine Applications
Towards rapid, high-throughput and cost-effective evaluation of viral vector efficacy: Rapid image pattern analysis from microfluidic cell cultures us

Original classification

Research Grant

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