The non-viral delivery era has begun, with challenges ahead

“Ultimately, the long‑term success of in vivo LNP delivery will depend on the ability to transform these platforms into robust, manufacturable, and globally accessible therapeutic modalities.” In recent years, the development of lipid nanoparticles (LNPs) including targeted LNPs (tLNPs) has positioned non‑viral delivery as one of the most important aspects of the evolving genetic medicines space for the delivery of nucleic acid‑based moieties. The success of COVID‑19 mRNA vaccines marked the first time the industry recognized the clinical relevance of non‑viral platforms and that these could be scaled for global industrial manufacturing. At the same time, the emergence of in vivo CAR‑T and in vivo gene editing has further strengthened market enthusiasm around LNP‑based technologies. Despite continued progress and beneath the current excitement, there are still several major challenges to overcome with this technology.

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Publication Details

Journal
Cell and Gene Therapy Insights
Published
2026-06-17
DOI
https://doi.org/10.18609/cgti.2026.061
Primary Topic
RNA Interference and Gene Delivery
Type
article
Field-Weighted Citation Impact
0.00
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article

The non-viral delivery era has begun, with challenges ahead

Pete Smith, Yu Cao
Cell and Gene Therapy Insights
RNA Interference and Gene Delivery
article

The non-viral delivery era has begun, with challenges ahead

Pete Smith, Yu Cao
article en

Abstract

“Ultimately, the long‑term success of in vivo LNP delivery will depend on the ability to transform these platforms into robust, manufacturable, and globally accessible therapeutic modalities.” In recent years, the development of lipid nanoparticles (LNPs) including targeted LNPs (tLNPs) has positioned non‑viral delivery as one of the most important aspects of the evolving genetic medicines space for the delivery of nucleic acid‑based moieties. The success of COVID‑19 mRNA vaccines marked the first time the industry recognized the clinical relevance of non‑viral platforms and that these could be scaled for global industrial manufacturing. At the same time, the emergence of in vivo CAR‑T and in vivo gene editing has further strengthened market enthusiasm around LNP‑based technologies. Despite continued progress and beneath the current excitement, there are still several major challenges to overcome with this technology.

Cell and Gene Therapy InsightsVol. 12(5)
Intarcia Therapeutics (United States) (US), Mandala Biosciences (United States) (US)
Industry, innovation and infrastructure
Openalex Percentile: Top 12%
RNA Interference and Gene Delivery
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