Evidence map›Paper›PMID 36579950›Full record

ReviewNucleic acid therapeutics2023

Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides.

Aurélie Goyenvalle, Cecilia Jimenez-Mallebrera, Willeke van Roon, Sabine Sewing, Arthur M Krieg, Virginia Arechavala-Gomeza, Patrik Andersson

Abstract readReview
In one paragraph

Review in Nucleic acid therapeutics, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 44 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
44citing papers in PubMed, 1 pooled it
–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

44 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Review
  4. Article
  5. Review
  6. Article
  7. Article
  8. Article
  9. Article
  10. Article
  11. Article
  12. Review
  13. Review
  14. Genetic therapies for neurological diseases.Pharmacological reviews · 2026
    Review
  15. Review
  16. Article
  17. Article
  18. Article
  19. Article
  20. Article
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

7 authors.

Aurélie GoyenvalleUniversité Paris-Saclay, UVSQ, Inserm, END-ICAP, Versailles, France.ORCID 0000-0003-3938-1165
Cecilia Jimenez-MallebreraLaboratorio de Investigación Aplicada en Enfermedades Neuromusculares, Unidad de Patología Neuromuscular, Servicio de Neuropediatría, Institut de Recerca Sant Joan de Déu, Esplugues de Llobregat, Spain.
Willeke van RoonDepartment of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands.ORCID 0000-0002-3035-0533
Sabine SewingPharma Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland.
Arthur M KriegRNA Therapeutics Institute, University of Massachusetts, Worcester, Massachusetts, USA.
Virginia Arechavala-GomezaNeuromuscular Disorders, Biocruces Bizkaia Health Research Institute, Barakaldo, Spain.ORCID 0000-0001-7703-3255
Patrik AnderssonSafety Innovation, Safety Sciences, Clinical Pharmacology and Safety Sciences, R&D, AstraZeneca, Gothenburg, Sweden.ORCID 0000-0002-0585-4756

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The nucleic acid therapeutics field has made tremendous progress in the past decades. Continuous advances in chemistry and design have led to many successful clinical applications, eliciting even more interest from researchers including both academic groups and drug development companies. Many preclinical studies in the field focus on improving the delivery of antisense oligonucleotide drugs (ONDs) and/or assessing their efficacy in target tissues, often neglecting the evaluation of toxicity, at least in early phases of development. A series of consensus recommendations regarding regulatory considerations and expectations have been generated by the Oligonucleotide Safety Working Group and the Japanese Research Working Group for the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use S6 and Related Issues (WGS6) in several white papers. However, safety aspects should also be kept in sight in earlier phases while screening and designing OND to avoid subsequent failure in the development phase. Experts and members of the network "DARTER," a COST Action funded by the Cooperation in Science and Technology of the EU, have utilized their collective experience working with OND, as well as their insights into OND-mediated toxicities, to generate a series of consensus recommendations to assess OND toxicity in early stages of preclinical research. In the past few years, several publications have described predictive assays, which can be used to assess OND-mediated toxicity

Indexed as

OligonucleotidesOligonucleotides, AntisenseAnimalsDrug Evaluation, PreclinicalHumansPharmaceutical PreparationsOligonucleotidesOligonucleotides, AntisensePharmaceutical Preparationsantisense oligonucleotidesearly preclinical safety assessmentpredictive assaystoxicity

Identifiers

PMID36579950
PMCPMC9940817

What OpenQuestion 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 OpenQuestion graph.