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Pharmaceutics
Special Issues
Nanotechnology and RNA-Based Technologies Applied to Prophylaxis and Advanced Therapies
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Nanotechnology and RNA-Based Technologies Applied to Prophylaxis and Advanced Therapies

A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Gene and Cell Therapy".

Deadline for manuscript submissions: 20 November 2025 | Viewed by 1277

Special Issue Editors

Prof. Dr. Bruna Aparecida Souza Machado

E-Mail Website
Guest Editor
SENAI Institute of Innovation (ISI) in Health Advanced Systems (CIMATEC ISI SAS), SENAI CIMATEC University Center, Salvador 41650-010, Bahia State, Brazil
Interests: biotechnology; mRNA; adjuvants; vaccines; therapies
Dr. Vinicius Pinto Costa Rocha

E-Mail Website
Guest Editor
1. Institute of Health Technology, National Industrial Learning Service—Integrated Manufacturing and Technology Campus, SENAI CIMATEC, Salvador 41650-010, Bahia, Brazil
2. Laboratory of Tissue Engineering and Immunopharmacology, Oswaldo Cruz Foundation, Gonçalo Moniz Institute—Fiocruz, Salvador 40296-710, Bahia, Brazil
Interests: biotechnology; mRNA vaccines; mRNA therapeutics; interference RNA; lipid nanoparticles; cancer; infectious diseases; neglected diseases

Special Issue Information

Dear Colleagues,

Various RNA technologies have revolutionized biotechnology by enabling the precise manipulation and understanding of genetic information. Techniques such as RNA interference (RNAi) and CRISPR-Cas9 utilize RNA molecules to target and modify specific genes, offering potential treatments for genetic disorders and cancers. mRNA vaccines, like those developed for COVID-19, showcase the power of RNA in rapidly producing effective vaccines. Overall, RNA technologies are paving the way for innovative therapies, diagnostics, and research tools in biotechnology. This Special Issue aims to cover the aspects of nanotechnology and RNA-based technologies applied to vaccines and treatments, including recent original research or reviews, based on any of the following themes:

  • Nanotechnology for RNA applications;
  • Lipid nanoparticles for mRNA vaccines and therapies;
  • mRNA vaccines and therapies;
  • RNA-based advanced therapies;
  • RNAi for therapeutics.

We look forward to receiving your contributions.

Prof. Dr. Bruna Aparecida Souza Machado
Dr. Vinicius Pinto Costa Rocha
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Pharmaceutics is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2900 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • RNA-technologies
  • nanotechnology
  • messenger RNA (mRNA)
  • interference RNA (RNAi)
  • vaccines
  • treatment

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Published Papers (1 paper)

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Research

18 pages, 2776 KiB  
Article
Predictive Lung- and Spleen-Targeted mRNA Delivery with Biodegradable Ionizable Lipids in Four-Component LNPs
by Juan Heredero, Álvaro Peña, Esther Broset, Beatriz Blandín, Diego de Miguel, Teresa Alejo, Alfonso Toro, Elena Mata, Alexandre López-Gavín, Ana Gallego-Lleyda, Diego Casabona, Verónica Lampaya, Ana Larraga, Esther Pérez-Herrán, David Luna, Irene Orera, Eduardo Romanos, Alba García, Juan Martínez-Oliván and Javier Giménez-Warren
Pharmaceutics 2025, 17(4), 459; https://doi.org/10.3390/pharmaceutics17040459 - 2 Apr 2025
Viewed by 977
Abstract
Background/Objectives: Lipid nanoparticles (LNPs) are leading mRNA delivery vehicles, with ionizable lipids (ILs) as their key component. However, the relationship between the IL structure and LNP endogenous organ-targeting is not well understood. In this study, we developed a novel library of biodegradable [...] Read more.
Background/Objectives: Lipid nanoparticles (LNPs) are leading mRNA delivery vehicles, with ionizable lipids (ILs) as their key component. However, the relationship between the IL structure and LNP endogenous organ-targeting is not well understood. In this study, we developed a novel library of biodegradable ILs featuring beta-propionate linkers, which, when incorporated into a four-component LNP formulation, show excellent extrahepatic selectivity and high protein expression. Methods: We explored the impact of structural modifications in the hydrophobic chains and polar-head groups in the ILs while keeping the linkers unchanged. In vivo results were evaluated to examine how structural changes influence the biodistribution to spleen or lungs. LNP formulations were assessed for their protein expression levels and organ-specific targeting. Additionally, protein corona formation by the best-performing LNPs was examined to provide further mechanistic insights. Results: Organ targeting was significantly influenced by structural changes in the ILs, allowing for precise control of the biodistribution between the spleen and lungs. Branched hydrophobic chains demonstrated a higher propensity for spleen targeting, while modifications in the polar-head group could drastically shift biodistribution from the lung to the spleen. This led to the identification of LNPs’ zeta potential as a key determinant of their extrahepatic targeting properties. Notably, ionizable lipid A3T2C7, also known as CP-LC-1495, displayed strong lung selectivity (97%) and high protein expression in lung tissue (1.21 × 108 p/s). Similarly, several promising candidates for spleen-targeting LNPs displayed protein expression levels exceeding 1 × 107 p/s (selectivity >80%). Conclusions: This study elucidates the structure–function relationships of ILs in passive organ-specific mRNA delivery, highlighting how the fine-tuning of hydrophobic chains, polar-head groups, and surface charge (zeta potential) allows for the precise control of LNP endogenous biodistribution, a mechanism influenced by protein corona formation. These findings enable the rational design of targeted LNP systems, enhancing their therapeutic potential for specific organs, such as the spleen and lungs. Full article
(This article belongs to the Special Issue Nanotechnology and RNA-Based Technologies Applied to Prophylaxis and Advanced Therapies)
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