Evidence map›Paper›PMID 40682272›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025

Preclinical quality, safety, and efficacy of a CGMP iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies.

Karim Azzag, Alessandro Magli, James Kiley, Darin Sumstad, Diane Kadidlo, Sarah B Crist, Beverly Norris, Aaron Ahlquist, Laura L Hocum Stone, John Everett and 14 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 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. Progress on cell therapy for skeletal muscle disorders.Advanced drug delivery reviews · 2026
    Review
  2. Review
  3. Article
  4. Review
  5. Pluripotent stem cell-based therapy toward the clinic for muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  6. Stem/progenitor cell-based therapy for Duchenne muscular dystrophy.Frontiers in cell and developmental biology · 2025
    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

24 authors.

Karim AzzagLillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN 55455, USA.
Alessandro MagliLillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN 55455, USA; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA.
James KileyLillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN 55455, USA.
Darin SumstadMolecular and Cellular Therapeutics, University of Minnesota, St. Paul, MN 55108, USA.
Diane KadidloMolecular and Cellular Therapeutics, University of Minnesota, St. Paul, MN 55108, USA.
Sarah B CristLillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN 55455, USA.
Beverly NorrisCenter for Translational Medicine, University of Minnesota, Minneapolis, MN 55455, USA.
Aaron AhlquistLillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN 55455, USA.
Laura L Hocum StoneDepartment of Surgery, University of Minnesota, Minneapolis, MN 55455, USA.
John EverettDepartment of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Jill Shappa FaustichDepartment of Surgery, University of Minnesota, Minneapolis, MN 55455, USA.
Davis SeeligDepartment of Veterinary Clinical Sciences, University of Minnesota, St. Paul, MN 55108, USA; Department of Comparative Pathology Shared Resource, Masonic Cancer Center, University of Minnesota, Minneapolis, MN 55455, USA.
Parthasarathy RangarajanDepartment of Surgery, University of Minnesota, Minneapolis, MN 55455, USA.
Hyunkee KimLillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN 55455, USA.
Craig FloryCenter for Translational Medicine, University of Minnesota, Minneapolis, MN 55455, USA.
Frederic BushmanDepartment of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Sabarinathan RamachandranStem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA; Department of Surgery, University of Minnesota, Minneapolis, MN 55455, USA.
Peter B KangGreg Marzolf Jr. Muscular Dystrophy Center, University of Minnesota, Minneapolis, MN 55455, USA; Department of Neurology, University of Minnesota, Minneapolis, MN 55455, USA.
Robert J SchumacherCenter for Translational Medicine, University of Minnesota, Minneapolis, MN 55455, USA.
John E WagnerStem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.
Michael KybaStem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA; Greg Marzolf Jr. Muscular Dystrophy Center, University of Minnesota, Minneapolis, MN 55455, USA; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.
Melanie L GrahamDepartment of Surgery, University of Minnesota, Minneapolis, MN 55455, USA; Department of Veterinary Population Medicine, University of Minnesota, St. Paul, MN 55108, USA.
David H McKennaStem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA; Molecular and Cellular Therapeutics, University of Minnesota, St. Paul, MN 55108, USA.
Rita C R PerlingeiroLillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN 55455, USA; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA; Greg Marzolf Jr. Muscular Dystrophy Center, University of Minnesota, Minneapolis, MN 55455, USA. Electronic address: perli032@umn.edu.

Funding

Trial Design and Biostatistical Support CoreP01CA065493 · NCI · UNIVERSITY OF MINNESOTA TWIN CITIES · PI Mark J Osborn · 1995 to 2026
$48.2M
Nonhuman Primate Reagent ResourceU24AI126683 · NIAID · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Diogo Magnani · 2016 to 2026
$20.1M
Resource CoreP40OD028116 · OD · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Diogo Magnani · 2020 to 2026
$5.9M
Preclinical studies of pluripotent stem cell-derived myogenic progenitors in non-human primatesR01AR078624 · NIAMS · UNIVERSITY OF MINNESOTA · PI GRAHAM, MELANIE LYNN, PERLINGEIRO, RITA C. R. · 2021 to 2025
$2.9M
Skeletal Muscle Regeneration from Pluripotent Stem CellsR01AR078571 · NIAMS · UNIVERSITY OF MINNESOTA · PI PERLINGEIRO, RITA C. R. · 2021 to 2025
$2.1M
Targeting Dystroglycanopathies using Pluripotent-derived Myogenic ProgenitorsR01AR081882 · NIAMS · UNIVERSITY OF MINNESOTA · PI Rita C. R. Perlingeiro · 2023 to 2026
$2.1M
Foundations for a Phase 1 Clinical Trial ofCell-based Therapy for Duchenne Muscular DystrophyR34AR081536 · NIAMS · UNIVERSITY OF MINNESOTA · PI KANG, PETER B. · 2022 to 2023
$409k
NCI NIH HHS P01 CA065493NIAID NIH HHS U24 AI126683NIAMS NIH HHS R01 AR078571NIAMS NIH HHS R01 AR078624NIAMS NIH HHS R01 AR081882NIAMS NIH HHS R34 AR081536NIH HHS P40 OD028116
6 · The paper itself

Abstract

Pluripotent stem cell (PSC)-derived therapies are in clinical trials of terminally differentiated or transiently required cell types, but to date no PSC-derived trial contributing tissue-specific stem cells or any PSC-based skeletal muscle regeneration trial has been approved. We describe a process in accordance with the Current Good Manufacturing Practice (CGMP) to generate large-scale cryopreserved PAX7-induced myogenic progenitors, which reconstitute both fibers and satellite cells, from PSCs. We subjected the clinical-grade cell product MyoPAXon to biodistribution, toxicity, and tumorigenicity studies in mice under Good Laboratory Practice conditions with no adverse effects and demonstrate long-term engraftment (>1 year) and efficacy in dystrophic mice. Transplantation of 37-60 million MyoPAXon cells into immunosuppressed non-human primates showed human contribution to muscle fibers and satellite cells, with no safety concerns. The US Food and Drug Administration has recently authorized this fully characterized off-the-shelf CGMP product for a first-in-human clinical trial in Duchenne muscular dystrophy, representing the first iPSC-derived tissue-specific stem cell therapy.

Indexed as

Induced Pluripotent Stem CellsMuscle DevelopmentMuscular DystrophiesStem Cell TransplantationAnimalsCell DifferentiationDisease Models, AnimalHumansMiceMice, Inbred mdxMuscular Dystrophy, DuchennePAX7 Transcription FactorSatellite Cells, Skeletal MusclePAX7 Transcription FactorCGMP productefficacyGLP preclinical studiesiPS cellsmuscle regenerationmuscular dystrophymyogenic progenitorsMyoPAXonnon-human primatessafety

Identifiers

PMID40682272
PMCPMC12338975

What OpenQuestion holds

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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.