{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Agarwal R"],"funding":["NCI NIH HHS","California Institute for Regenerative Medicine (CIRM)"],"pagination":["3183-3190"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC12443608"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["31(9)"],"pubmed_abstract":["Current hematopoietic stem cell transplantation (HSCT) conditioning strategies cause widespread tissue damage and systemic toxicities, especially in patients with DNA-repair deficiencies such as Fanconi anemia (FA). We have developed an alternative conditioning approach that incorporates the anti-CD117 antibody, briquilimab, which targets host hematopoietic stem and progenitor cells in place of genotoxic irradiation- and busulfan-based chemotherapy. Here we report a phase 1b clinical trial in patients with FA and bone marrow failure, evaluating safety and efficacy of briquilimab-based conditioning in combination with rabbit anti-thymocyte globulin, cyclophosphamide, fludarabine and rituximab immunosuppression and T cell receptor (TCR)αβ<sup>+</sup> T cell-depleted and CD19<sup>+</sup> B ce"],"journal":["Nature medicine"],"pubmed_title":["Irradiation- and busulfan-free stem cell transplantation in Fanconi anemia using an anti-CD117 antibody: a phase 1b trial."],"pmcid":["PMC12443608"],"funding_grant_id":["P30 CA008748","CLIN2-14315"],"pubmed_authors":["Pang WW","Bouge A","Wlodarski MW","Agarwal R","Weinberg K","Chan YY","Cheng JC","Van Hentenryck M","Krampf MR","Willner H","Shizuru JA","Nofal R","Perriman RJ","Kunte N","Din HN","Klinger EF","Boelens JJ","Soco C","Czechowicz A","Bertaina A","Walck E","Porteus M","Sen S","Barbarito G","Long-Boyle JR","Scheck A","Roncarolo MG","Hiroshima L","Saini G","Parkman R","Istomina E"],"additional_accession":[]},"is_claimable":false,"name":"Irradiation- and busulfan-free stem cell transplantation in Fanconi anemia using an anti-CD117 antibody: a phase 1b trial.","description":"Current hematopoietic stem cell transplantation (HSCT) conditioning strategies cause widespread tissue damage and systemic toxicities, especially in patients with DNA-repair deficiencies such as Fanconi anemia (FA). We have developed an alternative conditioning approach that incorporates the anti-CD117 antibody, briquilimab, which targets host hematopoietic stem and progenitor cells in place of genotoxic irradiation- and busulfan-based chemotherapy. Here we report a phase 1b clinical trial in patients with FA and bone marrow failure, evaluating safety and efficacy of briquilimab-based conditioning in combination with rabbit anti-thymocyte globulin, cyclophosphamide, fludarabine and rituximab immunosuppression and T cell receptor (TCR)αβ<sup>+</sup> T cell-depleted and CD19<sup>+</sup> B ce","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Sep","modification":"2026-06-03T11:25:59.888Z","creation":"2026-04-27T03:10:28.716Z"},"accession":"S-EPMC12443608","cross_references":{"pubmed":["40696207"],"doi":["10.1038/s41591-025-03817-1"]}}