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Comment
. 2025 Dec 21;404(10471):2490-2491.
doi: 10.1016/S0140-6736(24)02301-8. Epub 2024 Dec 3.

Non-toxic curative option for patients with genetic disorders

Affiliations
Comment

Non-toxic curative option for patients with genetic disorders

Denis Claude Roy. Lancet. .
No abstract available

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Conflict of interest statement

I report grants paid to my institution from TheCell: Cell, Tissue and Gene Therapy Network, Fonds de recherche Quebec-Sante, BioCanRx, Cancer Research Society, Medicament-Quebec, Canadian Institutes of Health Research, HMR Foundation, Infilise Foundation, Quebec Ministry of Economy, Innovation and Energy and some of this funding is for clinical trials mostly in the area of cancer immunotherapy; speakers fees from CEL for health care; and advisory board fees from VOR. I am the Chief Scientific Officer and Board member of C3i, a not-for-profit organisation that develops and manufactures cost-effective state-of-the-art integrated cell and gene therapies for clinical implementation. I am CEO, and Board member (unpaid) of the CellCAN Network, a non-profit organisation created under the Government of Canada's Networks of Centers of Excellence. I am Director of the University Institute for Hematology-Oncology and Cell Therapy. As a full Professor of Medicine at the Université de Montréal, I have a research laboratory developing novel cell therapy approaches.

Comment on

  • Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials.
    Río P, Zubicaray J, Navarro S, Gálvez E, Sánchez-Domínguez R, Nicoletti E, Sebastián E, Rothe M, Pujol R, Bogliolo M, John-Neek P, Bastone AL, Schambach A, Wang W, Schmidt M, Larcher L, Segovia JC, Yáñez RM, Alberquilla O, Díez B, Fernández-García M, García-García L, Ramírez M, Galy A, Lefrere F, Cavazzana M, Leblanc T, García de Andoin N, López-Almaraz R, Catalá A, Barquinero J, Rodríguez-Perales S, Rao G, Surrallés J, Soulier J, Díaz-de-Heredia C, Schwartz JD, Sevilla J, Bueren JA; FANCOLEN-1 gene therapy investigators. Río P, et al. Lancet. 2025 Dec 21;404(10471):2584-2592. doi: 10.1016/S0140-6736(24)01880-4. Epub 2024 Dec 3. Lancet. 2025. PMID: 39642902 Clinical Trial.

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