Evidence map›Paper›PMID 33209964›Full record

ReviewMolecular therapy. Methods & clinical development2020

Clinical Development of Gene Therapies: The First Three Decades and Counting.

Larissa Lapteva, Tejashri Purohit-Sheth, Mercedes Serabian, Raj K Puri

Abstract readReview
In one paragraph

Review in Molecular therapy. Methods & clinical development, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
23citing 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

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

  1. Pooled it
  2. Review
  3. Review
  4. Article
  5. Review
  6. Article
  7. Review
  8. Article
  9. New epilepsy therapies in development.Nature reviews. Drug discovery · 2024
    Review
  10. Review
  11. Human gene therapy · 2024
    Review
  12. Molecular therapy. Methods & clinical development · 2023
    Article
  13. Scaling genetic resources: New paradigms for diagnosis and treatment of rare genetic disease.American journal of medical genetics. Part C, Seminars in medical genetics · 2023
    Article
  14. Article
  15. Differential T cell immune responses to deamidated adeno-associated virus vector.Molecular therapy. Methods & clinical development · 2022
    Article
  16. Article
  17. Article
  18. Article
  19. Review
  20. 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

4 authors.

Larissa LaptevaDivision of Clinical Evaluation and Pharmacology/Toxicology, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD 20993, USA.
Tejashri Purohit-ShethDivision of Clinical Evaluation and Pharmacology/Toxicology, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD 20993, USA.
Mercedes SerabianDivision of Clinical Evaluation and Pharmacology/Toxicology, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD 20993, USA.
Raj K PuriDivision of Cellular and Gene Therapies, Center for Biologics Evaluation and Research, Office of Tissues and Advanced Therapies, U.S. Food and Drug Administration, Silver Spring, MD, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

In the past three decades the field of gene therapy has made remarkable progress, surging from mere laboratory experiments to Food and Drug Administration (FDA)-approved products that bring significant reduction in disease burden to patients who previously had no therapeutic options for their serious conditions. Herein, we review the evolution of the gene therapy clinical research landscape and describe the gene therapy product development programs evaluated by the FDA in Investigational New Drug applications received in 1988-2019. We also discuss the clinical development programs of the first six oncolytic and gene therapy products approved in the United States.

Indexed as

CBERCenter for Biologics Evaluation and ResearchFDAgene therapyINDInvestigational New Drug applicationnatural historyOffice of Tissues and Advanced TherapiesOTATproduct developmentrare diseases

Identifiers

PMID33209964
PMCPMC7658574

What OpenQuestion holds

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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.