ASH 2024 | Robust immune reconstitution in Fanconi anemia mice following in-utero HSCT

So while we are very enthusiastic about our work in allogeneic transplants postnatally for FA patients specifically using the briquilimab antibody, as well as our work on ex vivo lentiviral gene therapy for Fanconi anemia type A patients without any genotoxic conditioning, we still recognize that there are unmet needs in FA patients, specifically continuing to reduce the toxicities and the complexities of treatment procedures, as well as to be able to expand treatment options to patients who are diagnosed at different stages of disease. So we’ve recently initiated studies exploring in utero transplantation in Fanconi anemia mice to look if that could be a therapeutic approach to correct the disease preemptively even before birth. And so we have some beautiful data showing that that’s possible without the use of any conditioning agents, given the competitive advantage of healthy hematopoietic stem progenitor cells over the failing cells in FA mice. And here we’re presenting additional work exploring further the results of that and specifically assessing immune reconstitution in that setting and showing diverse immune repertoires and really immune recovery that’s profound both in the peripheral blood as well as in tissue macrophages throughout many different systems. And so we’re very hopeful that this preclinical work will ultimately result in a clinical trial that would enable the preemptive treatment of Fanconi anemia patients who are diagnosed prenatally with any form of Fanconi anemia disease, not just Fanc A, but one of the 23 different genotypes could also be treated this way. And by having a clinical trial, it actually enables preemptive treatment in a prenatal context, we hope that also encourages more diagnosis of patients at an earlier time point, which would ultimately benefit the entire patient community.

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