Gene therapy has emerged as a transformative technology, offering life-changing treatments for conditions with few options. Supported by recent FDA-approvals and a robust industry-wide development pipeline, global investment in the gene therapy field could surge from $18 billion in 2023 to more than $97 billion by 2033 (1). But in large part due to manufacturing limitations, development stage gene therapies suffer from a disproportionately high number of regulatory holds, and some marketed therapies are priced at more than $4 million per dose (2). As more gene therapies progress toward commercialization, manufacturers face mounting pressure to improve scalability, lower production costs and accelerate affordable patient access.
For the field to reach its full market and clinical potential, developers must overcome persistent obstacles in the production and manufacturing processes that contribute to unfavorable development outcomes including regulatory holds, failures due to safety, and scalability challenges, all of which drive up development costs.
Limits of transient transfection
Transient transfection dominates gene therapy manufacturing processes, but this method—while effective in early-stage research—is challenged to meet the demands of large-scale commercial production. Requiring multiple plasmids and reagents, it is prone to batch failures and presents reproducibility challenges.
The drawbacks of transient transfection become even more evident at scale. Yield inconsistencies limit production volume and increase regulatory hurdles, increasing cost and time to reach patients.
New technologies such as automation and novel cell lines aim to improve consistency and reduce manufacturing timelines. But these efforts are limited by their reliance on transient transfection methods. Alternative vector production platforms—such as those using insect cells—show promise but have encountered challenges related to regulatory approval and product consistency.
Innovation in gene therapy manufacturing
Developers are exploring alternative approaches to transient transfection gene therapy production and manufacturing. Non-viral gene therapy vectors could help reduce manufacturing costs and address immunogenicity, however, the technology is in very early stages. Moreover, gene therapies primarily rely on recombinant adeno-associated virus (rAAV) viral vector delivery and these approaches will remain the superior approach for many years to come.
To enable large-scale manufacturing of rAAVs and usher in the next decade of gene therapy development, stable, high-yield production platforms are needed to replace transient transfection methods. Many companies have attempted to develop more cost-effective and higher-yield rAAV production systems, but they’ve encountered substantial hurdles: dealing with rep/helper genes toxic to cells, achieving optimal rAAV viral titers and ensuring system stability.
NewBiologix is pioneering a stable inducible rAAV producer cell line platform capable of addressing core limitations of transient transfection systems. By creating stable cell lines that produce viral vectors efficiently and at scale, NewBiologix’s platform is designed to eliminate the need for repeated transient plasmid transfections, significantly reducing production timelines and enhancing reliability during manufacturing. With this platform, manufacturers can expect faster production cycles, reduced costs and improved batch consistency—outcomes that are critical for achieving regulatory approval and scaling patient access. For pharmaceutical companies, predictable manufacturing processes lead to higher-quality product, more efficient trials and streamlined commercialization. For patients, this can translate to faster, more affordable access to life-changing therapies.
In its commitment to developing a stable HEK293 cell line that consistently produces high-quality, high-purity rAAVs, NewBiologix is laying the groundwork for a safe, reliable, and robust rAAV production system. The newly launched NewBiologix Xcell™ Portfolio features a comprehensive suite of advanced technologies aimed at achieving excellence in gene and cell therapy production. This includes a high-performance engineered HEK293 cell line specifically designed for rAAV production, an advanced platform that streamlines the production and testing of rAAV candidates, and customized sequencing and bioinformatics tools to analyze rAAV quality. By integrating these components, NewBiologix is accelerating rAAV development at the preclinical stage, which will ultimately reduce the risk of failure in drug development.
Looking ahead: Affordable, effective and safe gene therapy at scale
Estimates suggest that as many as 1 million people could benefit from gene therapies in the next 10 years (3). Innovations in production will ensure the field can keep pace with its potential and deliver high volumes of gene therapy products at an affordable cost.
NewBiologix launched its Xcell Portfolio October 31. The platform includes a suite of advanced technologies designed for excellence in gene & cell therapy production:
- Xcell™ Eng-HEK293 Cell Line: High-performance engineered HEK293 cell line for transient rAAV production.
- Xcell™ rAAV Production & Testing Platform: Cutting-edge platform designed to streamline the production and testing of rAAV candidates.
- Xcell™ Genomic Analytical Platform: Tailored sequencing and bioinformatics solutions to address a wide range of scientific inquiries.
“The Xcell Portfolio draws on our leadership’s decades of experience in cell line development and our focus on precision, reliability, and scalability,” said Igor Fisch, Ph.D., CEO of NewBiologix. “We are committed to catalyzing rAAV development for research institutions, CDMOs, and biotechnology and pharmaceutical companies. Our innovative suite of services and cell line solutions tackles complex challenges in gene therapy production, ensuring that safe and effective treatments are accessible to patients.”
Learn more about NewBiologix here.
References:
(1) Cell and Gene Therapy Market Size Report, 2024-2033. (2024, April). Retrieved from Nova One Advisor website: https://www.novaoneadvisor.com/report/cell-and-gene-therapy-market
(2) Drug Discovery & Development,With prices topping $4 million, high stakes define cell and gene therapy landscape (2024, April). Retrieved from Drug Discovery & Development website: https://www.drugdiscoverytrends.com/how-price-safety-and-efficacy-shape-the-cell-and-gene-therapy-landscape/
(3) National Bureau of Economic Research, Estimating the financial impact of gene therapy in the U.S. (2021, April). Retrieved from: https://nber.org/system/files/working_papers/w28628/w28628.pdf
Article originally published on Endpoints
AUTHOR
Igor Fisch
Ph.D., CEO of NewBiologix
NewBiologix on LinkedIn
https://www.newbiologix.com/
