Evidence map›Paper›PMID 39957799›Full record

ReviewLancet regional health. Americas2025

Clinical trials to gene therapy development and production in Brazil: a review.

Jonas Alex Morales Saute, Virginia Picanço-Castro, Ana Carolina de Freitas Lopes, João Batista da Silva Júnior, Johanna Henriques Nehm, Franciele Dos Santos Maciel, Ida Vanessa Doederlein Schwartz, Juliana Maria Ferraz Sallum, Margareth Castro Ozelo, Roberto Giugliani and 3 more

Abstract readReview
In one paragraph

Review in Lancet regional health. Americas, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing 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

7 citing papers in PubMed.

  1. Article
  2. Review
  3. Review
  4. Development andFrontiers in immunology · 2026
    Article
  5. Landscape ofMolecular therapy. Methods & clinical development · 2025
    Article
  6. How to democratize cell and gene therapy: A global approach.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  7. Advances and challenges for gene therapies in Brazil.Lancet regional health. Americas · 2025
    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

13 authors.

Jonas Alex Morales SauteMedical Genetics Service, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil.
Virginia Picanço-CastroCenter for Cell-based Therapy, Regional Blood Center of Ribeirão Preto, University of São Paulo, Ribeirão Preto, Brazil.
Ana Carolina de Freitas LopesExecutive Secretariat of the National Committee for Health Technology Incorporation (Conitec), Brazilian Ministry of Health, Brasilia, Brazil.
João Batista da Silva JúniorNational Health Surveillance Agency (Anvisa), Brasilia, Brazil.
Johanna Henriques NehmGraduate Program in Medicine, Medical Sciences, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil.
Franciele Dos Santos MacielGraduate Program in Medicine, Medical Sciences, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil.
Ida Vanessa Doederlein SchwartzMedical Genetics Service, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil.
Juliana Maria Ferraz SallumDepartment of Ophthalmology, Federal University of São Paulo, São Paulo, Brazil.
Margareth Castro OzeloINCT Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, SP, Brazil.
Roberto GiuglianiMedical Genetics Service, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil.
Martin BonaminoNational Network of Experts in Advanced Therapies (Reneta), Anvisa, Brasilia, Brazil.
Hilda Petrs-SilvaNational Network of Experts in Advanced Therapies (Reneta), Anvisa, Brasilia, Brazil.
Guilherme BaldoNational Network of Experts in Advanced Therapies (Reneta), Anvisa, Brasilia, Brazil.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Challenges related to the implementation of gene therapy products are daunting for low-to middle-income countries, such as Brazil, and include the creation of an appropriate technical, regulatory, and economic environment. In this manuscript, we give an overview of historical aspects, as well as the current state of clinical trials and approved gene therapy products for commercialization in Brazil. Focusing on gene replacement and CAR-T cell therapies, we discuss the main advances, limitations, and difficulties faced by the country in the production, approval, and incorporation of such products into the public health system. Finally, we highlight the potential leading role that low-to middle-income countries can have in this industry, not only by providing their own vector supply but also by addressing important issues related to the sustainability and long-term global affordability of gene therapy products.

Indexed as

CAR-T cellClinical trialsGene replacement therapyGene therapy productsLow-to middle-income countries

Identifiers

PMID39957799
PMCPMC11830283

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.