Haematology studies the blood and the haematopoietic organs, as well as the conditions that affect them — from coagulation disorders to haematological malignancies. Often chronic and disabling, these diseases now benefit from major innovations driven by gene therapies, next-generation antibodies and cellular models.
The work to which Inovarion has contributed falls into two groups. The first concerns haemostasis disorders and bleeding diseases. The laboratory took part in developing a nanobody directed against the A3 domain of von Willebrand factor, able to detect ADAMTS13 proteolysis in von Willebrand disease[7], as well as in creating a humanised mouse model of this disease[2]. This kind of model, carrying the human proteins, offers a particularly useful platform for evaluating new therapeutic options. In haemophilia, other work has focused on single-domain antibodies targeting antithrombin to rebalance haemostasis — including through hepatotropic AAV-vector gene therapy[8] — and on the equivalence of non-factor therapies, such as emicizumab, to factor VIII[4]. Bernard-Soulier syndrome, a rare platelet disorder, was studied via the RhoA pathway and megakaryocytes derived from induced pluripotent stem cells, combined with a three-dimensional bone-marrow model reproducing thrombopoiesis[3].
The second group concerns myeloid malignancies, in particular chronic myelomonocytic leukaemia: the role of the CXCL8 chemokine secreted by immature granulocytes in inhibiting normal haematopoiesis[5], and the targeting of heterochromatin (the H3K9me2 mark) to eliminate malignant stem cells[6]. This work relies on single-cell sequencing and spectral cytometry to dissect clonal dominance and the role of myeloid-derived suppressor cells. Recent work also addresses erythrophagocytosis in sickle cell disease, via the role of protein S and phosphatidylserine exposure[1].
This research mobilises single-domain (nanobodies) and bispecific antibodies, humanised and haemophilic mouse models, iPSCs combined with three-dimensional bone-marrow models, single-cell sequencing, spectral cytometry, AAV-vector gene therapy and chromatin analysis (CUT&Tag). This work has appeared in Blood, Haematologica, the Journal of Clinical Investigation and EMBO Molecular Medicine.
From correcting a coagulation defect to eliminating leukaemic cells, this work spans a broad range of haematology.
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Representative publications
- Auditeau et al. Protein S Enhances the Phagocytosis of Phosphatidylserine-Exposing Erythrocytes: Implications in Sickle Cell Disease. Am J Hematol, 2026. PubMed
- McCluskey et al. A fully humanized von Willebrand disease type 1 mouse model as unique platform to investigate novel therapeutic options. Haematologica, 2025. Record → · PubMed
- Lordier et al. Increased RhoA pathway activation downstream of αIIbβ3/SRC contributes to heterozygous Bernard Soulier syndrome. Haematologica, 2025. Record → · PubMed
- Sefiane et al. Consistent clinical factor VIII equivalency is unlikely for non-factor therapies in hemophilic mice. Haematologica, 2025. Record → · PubMed
- Deschamps et al. CXCL8 secreted by immature granulocytes inhibits WT hematopoiesis in chronic myelomonocytic leukemia. J Clin Invest, 2024. Record → · PubMed
- Hidaoui et al. Targeting heterochromatin eliminates chronic myelomonocytic leukemia malignant stem cells through reactivation of retroelements and immune pathways. Commun Biol, 2024. Record → · PubMed
- Kizlik-Masson et al. A nanobody against the VWF A3 domain detects ADAMTS13-induced proteolysis in congenital and acquired VWD. Blood, 2023. Record → · PubMed
- Barbon et al. Single-domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors. EMBO Mol Med, 2020. Record → · PubMed