SLN-140 (Silence Therapeutics plc) is a novel small interfering RNA (siRNA) targeting protein S for the treatment of hemophilia A (HA). Researchers from the University of Bern recently presented data from studies conducted in animal models of HA, performed to evaluate the safety and efficacy of SLN-140.
New and updated clinical data presented by biopharma firms at the American Society of Hematology annual meeting and exposition, including: Amgen, Beigene, Novartis, Pharming.
Vega Therapeutics Inc. has reported promising preclinical data on VGA-039, a first-in-class monoclonal antibody directed against human protein S (ProS) that inhibits ProS cofactor activity for tissue factor pathway inhibitor α (TFPIα) and activated protein C (aPC), thus enhancing thrombin generation by acting on both the initiation (TFPIα) and propagation (aPC) phases of coagulation for potential activity against various bleeding disorders.
After gaining U.S. FDA priority approval for the first gene therapy to treat hemophilia B, CSL Ltd. reported long-term data from the pivotal HOPE-B trial that showed a single infusion of Hemgenix (etranacogene dezaparvovec-drlb) generated elevated and sustained mean factor IX levels and reduced the rate of annual bleeding.
New and updated clinical data presented by biopharma firms at the American Society of Hematology annual meeting and exposition, including: Abbvie, Actinium, Adicet, Affimed, Agios, Alphamab, Aptose, Argenx, Ascentage, Bellicum, Blueprint, BMS, Cellcentric, Cogent, CSL, CTI, Ellipses, Excellthera, Fate, Genentech.
Novelmed Therapeutics Inc. has reported topline results with its lead anti-properdin antibody NM-3086 in a rabbit model of paroxysmal nocturnal hemoglobinuria (PNH). NM-3086 is a potent, first-in-class, humanized monoclonal antibody that is highly selective for properdin of the alternative pathway (AP) without blocking the classical pathway (CP), which is critical for maintaining host defense against infections.
Doctors at Great Ormond Street Hospital London have reported the first successful use of base-edited donor T cells, in the treatment of refractory T-cell lymphoblastic lymphoma. The case of a 13-year-old girl named Alyssa, whose disease had not responded to chemotherapy and a bone marrow transplant, was presented at the American Society of Hematology meeting in New Orleans on Dec. 11, 2022.
After gaining U.S. FDA priority approval for the first gene therapy to treat hemophilia B, CSL Ltd. reported long-term data from the pivotal HOPE-B trial that showed a single infusion of Hemgenix (etranacogene dezaparvovec-drlb) generated elevated and sustained mean factor IX levels and reduced the rate of annual bleeding. Presented at the American Society of Hematology (ASH) annual meeting on Dec. 10, data showed 24-month results reinforced the safety of treatment, with no serious treatment-related adverse effects.
Affimed NV said the latest data with its lead innate cell engager (ICE) known as AFM-13, disclosed at the American Society of Hematology (ASH) meeting, have caused the focus to shift from monotherapy to combination regimens.
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