Evidence map›Paper›PMID 39197837›Full record

ArticleJournal of medicinal chemistry2024

Structure-Activity Relationship of Antibody-Oligonucleotide Conjugates: Evaluating Bioconjugation Strategies for Antibody-Phosphorodiamidate Morpholino Oligomer Conjugates for Drug Development.

Michael Cochran, Isaac Marks, Tyler Albin, Danny Arias, Philip Kovach, Beatrice Darimont, Hanhua Huang, Usue Etxaniz, Hae Won Kwon, Yunyu Shi and 4 more

Abstract read
In one paragraph

Article in Journal of medicinal chemistry, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing 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

11 citing papers in PubMed.

  1. Article
  2. Review
  3. Review
  4. Antisense RNA therapies for muscular dystrophies.Journal of neuromuscular diseases · 2026
    Review
  5. Review
  6. TheInternational journal of molecular sciences · 2025
    Review
  7. Article
  8. Review
  9. Review
  10. Article
  11. 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

14 authors.

Michael CochranAvidity Biosciences, Inc., 10578 Science Center Drive Suite 125, San Diego, California 92121, United States.
Isaac MarksAvidity Biosciences, Inc., 10578 Science Center Drive Suite 125, San Diego, California 92121, United States.
Tyler AlbinAvidity Biosciences, Inc., 10578 Science Center Drive Suite 125, San Diego, California 92121, United States.
Danny AriasAvidity Biosciences, Inc., 10578 Science Center Drive Suite 125, San Diego, California 92121, United States.
Philip KovachAvidity Biosciences, Inc., 10578 Science Center Drive Suite 125, San Diego, California 92121, United States.
Beatrice DarimontCYTOO, 7 Parv. Louis Néel CS 20050, 38040 Grenoble, France.
Hanhua HuangAvidity Biosciences, Inc., 10578 Science Center Drive Suite 125, San Diego, California 92121, United States.
Usue EtxanizAvidity Biosciences, Inc., 10578 Science Center Drive Suite 125, San Diego, California 92121, United States.
Hae Won KwonAvidity Biosciences, Inc., 10578 Science Center Drive Suite 125, San Diego, California 92121, United States.
Yunyu ShiAvidity Biosciences, Inc., 10578 Science Center Drive Suite 125, San Diego, California 92121, United States.
Matthew DiazAvidity Biosciences, Inc., 10578 Science Center Drive Suite 125, San Diego, California 92121, United States.
Olecya TyagloAvidity Biosciences, Inc., 10578 Science Center Drive Suite 125, San Diego, California 92121, United States.
Arthur LevinAvidity Biosciences, Inc., 10578 Science Center Drive Suite 125, San Diego, California 92121, United States.
Venkata Ramana DoppalapudiAvidity Biosciences, Inc., 10578 Science Center Drive Suite 125, San Diego, California 92121, United States.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Antibody-oligonucleotide conjugates (AOCs) are promising treatments for Duchenne muscular dystrophy (DMD). They work via induction of exon skipping and restoration of dystrophin protein in skeletal and heart muscles. The structure-activity relationships (SARs) of AOCs comprising antibody-phosphorodiamidate morpholino oligomers (PMOs) depend on several aspects of their component parts. We evaluate the SAR of antimouse transferrin receptor 1 antibody (αmTfR1)-PMO conjugates: cleavable and noncleavable linkers, linker location on the PMO, and the impact of drug-to-antibody ratios (DARs) on plasma pharmacokinetics (PK), oligonucleotide delivery to tissues, and exon skipping. AOCs containing a stable linker with a DAR9.7 were the most effective PMO delivery vehicles in preclinical studies. We demonstrate that αmTfR1-PMO conjugates induce dystrophin protein restoration in the skeletal and heart muscles of

Indexed as

Mice, Inbred mdxMorpholinosMuscular Dystrophy, DuchenneAnimalsDrug DevelopmentDystrophinHumansImmunoconjugatesMaleMiceMice, Inbred C57BLMuscle, SkeletalOligonucleotidesReceptors, TransferrinStructure-Activity RelationshipDystrophinImmunoconjugatesMorpholinosOligonucleotidesReceptors, Transferrin

Identifiers

PMID39197837
PMCPMC11403617

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.