The Hitchhiker’s Guide to Human Therapeutic Nanoparticle Development
Nanomedicine plays an essential role in developing new therapies through novel drug delivery systems, diagnostic and imaging systems, vaccine development, antibacterial tools, and high-throughput screening. One of the most promising drug delivery systems are nanoparticles, which can be designed with...
| Main Authors: | , , , , , , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2022-01-01
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| Series: | Pharmaceutics |
| Subjects: | |
| Online Access: | https://www.mdpi.com/1999-4923/14/2/247 |
| _version_ | 1797477229501874176 |
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| author | Thelvia I. Ramos Carlos A. Villacis-Aguirre Katherine V. López-Aguilar Leandro Santiago Padilla Claudia Altamirano Jorge R. Toledo Nelson Santiago Vispo |
| author_facet | Thelvia I. Ramos Carlos A. Villacis-Aguirre Katherine V. López-Aguilar Leandro Santiago Padilla Claudia Altamirano Jorge R. Toledo Nelson Santiago Vispo |
| author_sort | Thelvia I. Ramos |
| collection | DOAJ |
| description | Nanomedicine plays an essential role in developing new therapies through novel drug delivery systems, diagnostic and imaging systems, vaccine development, antibacterial tools, and high-throughput screening. One of the most promising drug delivery systems are nanoparticles, which can be designed with various compositions, sizes, shapes, and surface modifications. These nanosystems have improved therapeutic profiles, increased bioavailability, and reduced the toxicity of the product they carry. However, the clinical translation of nanomedicines requires a thorough understanding of their properties to avoid problems with the most questioned aspect of nanosystems: safety. The particular physicochemical properties of nano-drugs lead to the need for additional safety, quality, and efficacy testing. Consequently, challenges arise during the physicochemical characterization, the production process, in vitro characterization, in vivo characterization, and the clinical stages of development of these biopharmaceuticals. The lack of a specific regulatory framework for nanoformulations has caused significant gaps in the requirements needed to be successful during their approval, especially with tests that demonstrate their safety and efficacy. Researchers face many difficulties in establishing evidence to extrapolate results from one level of development to another, for example, from an in vitro demonstration phase to an in vivo demonstration phase. Additional guidance is required to cover the particularities of this type of product, as some challenges in the regulatory framework do not allow for an accurate assessment of NPs with sufficient evidence of clinical success. This work aims to identify current regulatory issues during the implementation of nanoparticle assays and describe the major challenges that researchers have faced when exposing a new formulation. We further reflect on the current regulatory standards required for the approval of these biopharmaceuticals and the requirements demanded by the regulatory agencies. Our work will provide helpful information to improve the success of nanomedicines by compiling the challenges described in the literature that support the development of this novel encapsulation system. We propose a step-by-step approach through the different stages of the development of nanoformulations, from their design to the clinical stage, exemplifying the different challenges and the measures taken by the regulatory agencies to respond to these challenges. |
| first_indexed | 2024-03-09T21:14:41Z |
| format | Article |
| id | doaj.art-1b3e2be4913240709d3e519337d436a0 |
| institution | Directory Open Access Journal |
| issn | 1999-4923 |
| language | English |
| last_indexed | 2024-03-09T21:14:41Z |
| publishDate | 2022-01-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Pharmaceutics |
| spelling | doaj.art-1b3e2be4913240709d3e519337d436a02023-11-23T21:36:05ZengMDPI AGPharmaceutics1999-49232022-01-0114224710.3390/pharmaceutics14020247The Hitchhiker’s Guide to Human Therapeutic Nanoparticle DevelopmentThelvia I. Ramos0Carlos A. Villacis-Aguirre1Katherine V. López-Aguilar2Leandro Santiago Padilla3Claudia Altamirano4Jorge R. Toledo5Nelson Santiago Vispo6Laboratorio de Biotecnología y Biofármacos, Departamento de Fisiopatología, Facultad de Ciencias Biológicas, Universidad de Concepción, Víctor Lamas 1290, Concepción 4070386, ChileLaboratorio de Biotecnología y Biofármacos, Departamento de Fisiopatología, Facultad de Ciencias Biológicas, Universidad de Concepción, Víctor Lamas 1290, Concepción 4070386, ChileCarrera Ingeniería en Biotecnología, Departamento de Ciencias de la Vida y la Agricultura, Universidad de las Fuerzas Armadas—ESPE, Sangolquí 171103, EcuadorFaculty of Biological Sciences, Friedrich-Schiller-Universität, 07743 Jena, GermanyEscuela de Ingeniería Bioquímica, Facultad de Ingeniería, Pontificia Universidad Católica de Valparaíso, Av. Brasil 2085, Valparaíso 2362803, ChileLaboratorio de Biotecnología y Biofármacos, Departamento de Fisiopatología, Facultad de Ciencias Biológicas, Universidad de Concepción, Víctor Lamas 1290, Concepción 4070386, ChileSchool of Biological Sciences and Engineering, Yachay Tech University, Hda. San José s/n y Proyecto Yachay, Urcuquí 100119, EcuadorNanomedicine plays an essential role in developing new therapies through novel drug delivery systems, diagnostic and imaging systems, vaccine development, antibacterial tools, and high-throughput screening. One of the most promising drug delivery systems are nanoparticles, which can be designed with various compositions, sizes, shapes, and surface modifications. These nanosystems have improved therapeutic profiles, increased bioavailability, and reduced the toxicity of the product they carry. However, the clinical translation of nanomedicines requires a thorough understanding of their properties to avoid problems with the most questioned aspect of nanosystems: safety. The particular physicochemical properties of nano-drugs lead to the need for additional safety, quality, and efficacy testing. Consequently, challenges arise during the physicochemical characterization, the production process, in vitro characterization, in vivo characterization, and the clinical stages of development of these biopharmaceuticals. The lack of a specific regulatory framework for nanoformulations has caused significant gaps in the requirements needed to be successful during their approval, especially with tests that demonstrate their safety and efficacy. Researchers face many difficulties in establishing evidence to extrapolate results from one level of development to another, for example, from an in vitro demonstration phase to an in vivo demonstration phase. Additional guidance is required to cover the particularities of this type of product, as some challenges in the regulatory framework do not allow for an accurate assessment of NPs with sufficient evidence of clinical success. This work aims to identify current regulatory issues during the implementation of nanoparticle assays and describe the major challenges that researchers have faced when exposing a new formulation. We further reflect on the current regulatory standards required for the approval of these biopharmaceuticals and the requirements demanded by the regulatory agencies. Our work will provide helpful information to improve the success of nanomedicines by compiling the challenges described in the literature that support the development of this novel encapsulation system. We propose a step-by-step approach through the different stages of the development of nanoformulations, from their design to the clinical stage, exemplifying the different challenges and the measures taken by the regulatory agencies to respond to these challenges.https://www.mdpi.com/1999-4923/14/2/247nanoparticlesnanomedicineregulatory aspectspharmacokineticspreclinicalimmunotoxicity |
| spellingShingle | Thelvia I. Ramos Carlos A. Villacis-Aguirre Katherine V. López-Aguilar Leandro Santiago Padilla Claudia Altamirano Jorge R. Toledo Nelson Santiago Vispo The Hitchhiker’s Guide to Human Therapeutic Nanoparticle Development Pharmaceutics nanoparticles nanomedicine regulatory aspects pharmacokinetics preclinical immunotoxicity |
| title | The Hitchhiker’s Guide to Human Therapeutic Nanoparticle Development |
| title_full | The Hitchhiker’s Guide to Human Therapeutic Nanoparticle Development |
| title_fullStr | The Hitchhiker’s Guide to Human Therapeutic Nanoparticle Development |
| title_full_unstemmed | The Hitchhiker’s Guide to Human Therapeutic Nanoparticle Development |
| title_short | The Hitchhiker’s Guide to Human Therapeutic Nanoparticle Development |
| title_sort | hitchhiker s guide to human therapeutic nanoparticle development |
| topic | nanoparticles nanomedicine regulatory aspects pharmacokinetics preclinical immunotoxicity |
| url | https://www.mdpi.com/1999-4923/14/2/247 |
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