Clinical progress of nanomedicine-based RNA therapies

Lim, Siyoung A.; Cox, Alysia; Tung, Madelynn; Chung, Eun Ji

doi:10.1016/j.bioactmat.2021.10.018

Cited by 29 publications

(34 citation statements)

References 97 publications

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Section: Applications Of Trehalose Materialsmentioning

confidence: 99%

“…Gene therapy recently witnessed a surge in translation to the clinic with the approval and widespread use of mRNA-based COVID-19 vaccines, and many more nanoparticle gene formulations for a range of diseases are currently in clinical trials. 108 Polymeric materials play an important role in these nonviral gene delivery strategies, including trehalose materials. 109 Backbone poly(trehalose) was selected early on by the Reineke group to prepare cationic copolymers carrying amidines 38 or quaternary amines 39 for plasmid DNA (pDNA) stabilization and delivery.…”

Section: Gene Deliverymentioning

confidence: 99%

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Synthesis and Application of Trehalose Materials

Vinciguerra

Gelb

Maynard

2022

JACS Au

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Trehalose is a naturally occurring, nonreducing disaccharide that is widely used in the biopharmaceutical, food, and cosmetic industries due to its stabilizing and cryoprotective properties. Over the years, scientists have developed methodologies to synthesize linear polymers with trehalose units either in the polymer backbone or as pendant groups. These macromolecules provide unique properties and characteristics, which often outperform trehalose itself. Additionally, numerous reports have focused on the synthesis and formulation of materials based on trehalose, such as nanoparticles, hydrogels, and thermoset networks. Among many applications, these polymers and materials have been used as protein stabilizers, as gene delivery systems, and to prevent amyloid aggregate formation. In this Perspective, recent developments in the synthesis and application of trehalose-based linear polymers, hydrogels, and nanomaterials are discussed, with a focus on utilization in the biomedical field.

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Section: Applications Of Trehalose Materialsmentioning

confidence: 99%

Section: Gene Deliverymentioning

confidence: 99%

Synthesis and Application of Trehalose Materials

Vinciguerra

Gelb

Maynard

2022

JACS Au

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“…120 Notably, some of the approved COVID-19 vaccines are based on mRNA encapsulated lipid nanoparticles. 121,122 Recently we have reviewed the causes that delay the effective implementation of nanomaterials in clinical applications 98,115,123 and some similarities with TCM can be drawn. For instance, the accurate description of the substances employed.…”

Section: Regulation and Safetymentioning

confidence: 99%

“…After this, few others (<100 liposomal formulations, organic, and polymer particles of nanometric size) have been approved − and fewer in the case of inorganic nanocrystals, e.g., the mentioned case of iron oxide nanoparticles) . Notably, some of the approved COVID-19 vaccines are based on mRNA encapsulated lipid nanoparticles. , …”

Section: Mutual Benefits and Current Limitations Of The Integration O...mentioning

confidence: 99%

The Integration of Nanomedicine with Traditional Chinese Medicine: Drug Delivery of Natural Products and Other Opportunities

et al. 2022

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The integration of progressive technologies such as nanomedicine with the use of natural products from traditional medicine (TM) provides a unique opportunity for the longed-for harmonization between traditional and modern medicine. Although several actions have been initiated decades ago, a disparity of reasons including some misunderstandings between each other limits the possibilities of a truly complementation. Herein, we analyze some common challenges between nanomedicine and traditional Chinese medicine (TCM). These challenges, if solved in a consensual way, can give a boost to such harmonization. Nanomedicine is a recently born technology, while TCM has been used by the Chinese people for thousands of years. However, for these disciplines, the regulation and standardization of many of the protocols, especially related to the toxicity and safety, regulatory aspects, and manufacturing procedures, are under discussion. Besides, both TCM and nanomedicine still need to achieve a wider social acceptance. Herein, we first briefly discuss the strengths and weaknesses of TCM. This analysis serves to focus afterward on the aspects where TCM and nanomedicine can mutually help to bridge the existing gaps between TCM and Western modern medicine. As discussed, many of these challenges can be applied to TM in general. Finally, recent successful cases in scientific literature that merge TCM and nanomedicine are reviewed as examples of the benefits of this harmonization.

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“…1,2 RNAs are unstable and easily degraded in the body and blood; 3,4 thus, RNA-based medicines must be combined with drug carriers for delivery to target organs and cells. 5,6 Among the numerous nanoparticle technologies available, lipid nanoparticles (LNPs) are the most promising RNA carriers, demonstrating great efficacy and biocompatibility. 7−11 LNPs are composed of several functional lipids.…”

Section: Introductionmentioning

confidence: 99%

Microfluidic Platform Enabling Efficient On-Device Preparation of Lipid Nanoparticles for Formulation Screening

Matsuura-Sawada

Uno

Maeki

et al. 2022

ACS Appl. Eng. Mater.

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To obtain an optimal lipid nanoparticle (LNP) formulation, it is necessary to consider various formulation and process parameters related to the quality of LNPs and efficacy of active ingredients. Microfluidic technology has become the most widely used method of LNP production. Although this technology facilitates size control and improves production output, there is a need for a screening platform to optimize LNP formulations. This study describes the development and application of a microfluidic platform equipped with multiple microchannels that allows the tuning of LNP compositions by adjusting the flow rate of the ethanol phases. We found that the LNPs prepared using our platform were almost equivalent in size to those prepared by the conventional off-device mixing process. We prepared siRNA-loaded LNPs with different cationic lipid ratios and amounts of PEGylated lipid modifications and confirmed the corresponding variation in particle size and polydispersity. siRNA-loaded LNPs with different amounts of PEGylated lipid modification were evaluated in vitro, and the optimal amount of PEGylated lipid with high siRNA knockdown activity was determined. The microfluidic platform has the potential to save time and effort in LNP-formulation screening as well as reduce the waste of expensive RNAs and other bioactive ingredients. We anticipate that this microfluidic platform will promote the rapid formulation of clinically effective LNPs.

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Clinical progress of nanomedicine-based RNA therapies

Cited by 29 publications

References 97 publications

Synthesis and Application of Trehalose Materials

Synthesis and Application of Trehalose Materials

The Integration of Nanomedicine with Traditional Chinese Medicine: Drug Delivery of Natural Products and Other Opportunities

Microfluidic Platform Enabling Efficient On-Device Preparation of Lipid Nanoparticles for Formulation Screening

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