Evidence map›Paper›PMID 40507949›Full record

ArticleInternational journal of molecular sciences2025

A Complete Approach for circRNA Therapeutics from Purification to Lyophilized Delivery Using Novel Ionizable Lipids.

Esther Broset, Ana Larraga, Verónica Lampaya, Víctor Navarro, Alexandre López-Gavín, Diego de Miguel, Álvaro Peña, Juan Martínez-Oliván, Diego Casabona

Abstract read
In one paragraph

Article in International journal of molecular sciences, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

6 citing papers in PubMed.

  1. Review
  2. Review
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  6. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors.

Esther BrosetCertest Pharma, Certest Biotec Ltd., Polígono Industrial Río Gallego II, Calle J, 1, 50840 San Mateo de Gállego, Spain.ORCID 0000-0003-4155-749X
Ana LarragaCertest Pharma, Certest Biotec Ltd., Polígono Industrial Río Gallego II, Calle J, 1, 50840 San Mateo de Gállego, Spain.
Verónica LampayaCertest Pharma, Certest Biotec Ltd., Polígono Industrial Río Gallego II, Calle J, 1, 50840 San Mateo de Gállego, Spain.
Víctor NavarroCertest Pharma, Certest Biotec Ltd., Polígono Industrial Río Gallego II, Calle J, 1, 50840 San Mateo de Gállego, Spain.
Alexandre López-GavínCertest Pharma, Certest Biotec Ltd., Polígono Industrial Río Gallego II, Calle J, 1, 50840 San Mateo de Gállego, Spain.
Diego de MiguelCertest Pharma, Certest Biotec Ltd., Polígono Industrial Río Gallego II, Calle J, 1, 50840 San Mateo de Gállego, Spain.ORCID 0000-0002-8486-8514
Álvaro PeñaCertest Pharma, Certest Biotec Ltd., Polígono Industrial Río Gallego II, Calle J, 1, 50840 San Mateo de Gállego, Spain.ORCID 0000-0002-3517-4102
Juan Martínez-OlivánCertest Pharma, Certest Biotec Ltd., Polígono Industrial Río Gallego II, Calle J, 1, 50840 San Mateo de Gállego, Spain.
Diego CasabonaCertest Pharma, Certest Biotec Ltd., Polígono Industrial Río Gallego II, Calle J, 1, 50840 San Mateo de Gállego, Spain.ORCID 0009-0000-9722-9124

Funding

Agencia Estatal de Investigación 10.13039/501100011033Gobierno de Aragón IDMF/2023/0003Ministerio de Ciencia, Innovación y Universidades DIN2021-011799
6 · The paper itself

Abstract

Circular RNA (circRNA) has gained significant attention as a potential therapeutic tool due to its remarkable stability and resistance to degradation by exonucleases. However, scalable and efficient methods for purification and delivery remain critical challenges that must be addressed. In this study, we developed and evaluated an optimized affinity chromatography method using Oligo (dT) columns for the purification of circRNA, achieving high yield and purity compared with high-performance liquid chromatography. Additionally, we investigated the in vivo efficacy of circRNA-Oligo (dT) encapsulated in lipid nanoparticles (LNPs) formulated with emerging ionizable lipids, including CP-LC-0867 and CP-LC-0729. Our results showed that LNPs formulated with CP-LC-0867 consistently produced higher protein expression compared to SM-102, with sustained luciferase activity observed over a 14-day period. Furthermore, we assessed the lyophilization potential of LNP-circRNA-Oligo (dT) using CP-LC-0729 to extend the shelf life and eliminate the need for ultra-low-temperature storage. Remarkably, the lyophilized LNPs exhibited no significant differences in protein expression compared to their non-lyophilized counterparts, demonstrating that lyophilization is a viable strategy for extending the storage and transport of circRNA therapies. These findings underscore the potential of optimized new ionizable lipids, improved purification strategies, and lyophilization techniques to enhance the scalability, stability, and practical application of circRNA therapies.

Indexed as

LipidsRNA, CircularAnimalsChromatography, AffinityFreeze DryingHumansMiceNanoparticlesLipidsRNA, CircularcircRNAionizable lipidsLNPLyophilizationscalable purification

Identifiers

PMID40507949
PMCPMC12154124

What Socratic holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.