Cited by 35Pharmaceutical Economics and PolicyVirus-based gene therapy researchCAR-T cell therapy research

The DOI matches its Crossref record; 1 of the 1 references it cites were found in a registry or an index.

Cost and availability of novel cell and gene therapies: Can we avoid a catastrophic second valley of death?

Open access
De Luca, Michele, Cossu, Giulio·2023·EMBO Reports

Abstract

Advanced gene and cellular therapies risk a second "valley of death" due to their high costs and low patient population. As these are life-saving therapies, measures are urgently needed to prevent their withdrawal from the market.

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Crossref · 10.15252/embr.202256661

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  • High costs involved in the production of cell-based therapies currently limit their use in the clinic.

    “This, coupled with the high costs involved in the production of such cell-based therapies (192), is currently limiting their use in the clinic.”

    From Targeting macrophages in liver fibrosis (2026) · cites this paper as [192]

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Citations

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Research works printed but not linked: 13

  1. Abbot A (2013) Italian stem‐cell trial based on flawed data. Nature https://doi.org/10.1038/nature.2013.13329 · DOI
  2. Aiuti A, Pasinelli F, Naldini L (2022) Ensuring a future for gene therapy for rare diseases. Nat Med 28: 1985–1988 · DOI
  3. Cossu G, Birchall M, Brown T, De Coppi P, Culme‐Seymour E, Gibbon S, Hitchcock J, Mason C, Montgomery J, Morris S et al (2018) Lancet commission: stem cells and regenerative medicine. Lancet 391: 883–910 · DOI
  4. Cossu G, Fears G, Griffin G, ter Meulen V (2020) Regenerative medicine: a long and winding road. Lancet 395: 1746–1747 · DOI
  5. Dal‐Ré R, Kesselheim AS, Bourgeois FT (2020) Increasing access to FDA inspection reports on irregularities and misconduct in clinical trials. JAMA 323: 1903–1904 · DOI
  6. De Luca M, Aiuti A, Cossu G, Palmer M, Pellegrini G, Robey P (2019) Stem cells find their way to clinics. Nat Cell Biol 21: 801–811 · DOI
  7. Ferrua F, Aiuti A (2017) Twenty‐five years of gene therapy for ADA‐SCID: from bubble babies to an approved drug. Hum Gene Ther 28: 972–981 · DOI
  8. Halley MC, Smith HS, Ashley EU, Goldenberg AJ, Tabor HK (2022) A call for an integrated approach to improve efficiency, equity and sustainability in rare disease research in the United States. Nat Genet 54: 219–222 · DOI
  9. Kueckelhaus M, Rothoeft T, De Rosa L, Yeni B, Ohmann T, Maier C, Eitner L, Metze D, Losi L, Seconetti AS et al (2021) Transgenic epidermal cultures for junctional epidermolysis bullosa – 5 – year outcomes. N Engl J Med 385: 2264–2270 · DOI
  10. McDonald CM, Campbell C, Torricelli RE, Finkel RS, Flanigan KM, Goemans N, Heydemann P, Kaminska A, Kirschner J, Muntoni F et al (2017) Ataluren clinical evaluator training group; ACT DMD study group. 2017. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD):a multicentre, randomised, double‐blind, placebo‐controlled, phase 3 trial. Lancet 390: 1489–1498 · DOI
  11. Palamenghi M, De Luca M, De Rosa L (2022) The steep uphill path leading to ex vivo gene therapy for genodermatoses. Am J Physiol Cell Physiol 323: C896–C906 · DOI
  12. Rama P, Matuska S, Paganoni G, Spinelli A, De Luca M, Pellegrini G (2010) Limbal stem‐cell therapy and long‐term corneal regeneration. N Engl J Med 363: 147–155 · DOI
  13. Sipp D, Caulfield T, Kaye J, Barfoot J, Blackburn C, Chan S, De Luca M, Kent A, McCabe C, Munsie M et al (2017) Marketing of unproven stem cell–based interventions: a call to action. Sci Transl Med 9: eaag0426 · DOI

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Metadata

Authors
De Luca, M., & Cossu, G.
Affiliations
University of Manchester (GB)
Corresponding author
Cossu, Giulio
Venue
EMBO Reports
Published
2023-01-02
Volume
24
Issue
2
Pages
e56661-e56661
ISSN
  • 1469-221X (ISSN-L)
  • 1469-3178
OpenAlex
W4313406630
Cited by
35
References
1
Citations by year
2026: 7 · 2025: 10 · 2024: 11 · 2023: 7
FWCI
7.13
Type
journal article
Language
en
Open access
Locations
  • EMBO Reports (publishedVersion, other-oa)
  • PubMed Central (publishedVersion, cc-by-nc-nd)
  • PubMed (publishedVersion)
  • Research Explorer (The University of Manchester) (publishedVersion, cc-by-nc-nd)
Funders
Medical Research Council (MR/S015116/1)
Topics
  • Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Pharmaceutical Economics and Policy
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
  • Health Sciences › Medicine › Oncology › CAR-T cell therapy research
Keywords
cell and gene therapy, treatment costs, drug availability, market access, valley of death, rare diseases
MeSH terms
Environment, Humans, Genetic Therapy

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