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.
Tuberous sclerosis complex (TSC) is a multisystemic disorder caused by loss-of-function mutations in the TSC1 or TSC2 genes. Its clinical phenotype is heterogenous, but most patients with TSC have epilepsy.
New and updated preclinical data presented at the American Society of Hematology Annual Meeting in New Orleans, by: Affimed, Asher Biotherapeutics, Disc Medicine, Keros Therapeutics, Kymera Therapeutics, Neoleukin Therapeutics, NGM Biopharmaceuticals, Oric Pharmaceuticals, Salarius Pharmaceuticals, Vincerx Pharma.
Researchers from Tubulis GmbH presented preclinical data for a CD30-targeting antibody-drug conjugate (ADC), TUB-010, being developed as a potential anticancer immunotherapy candidate.
Researchers from Salarius Pharmaceuticals Inc. have presented preclinical data on SP-3164, a novel cereblon (CRBN)-binding protein degrader intended for the treatment of lymphoma. SP-3164 bound to cereblon and consequently induced the degradation of hematological transcription factors Ikaros and Aiolos. The aim of their studies was to investigate the drug’s antitumor efficacy in preclinical models of diffuse large B-cell lymphoma (DLBCL).
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.
Treatment with anti-CD19 bispecific T-cell engager and CAR T therapies can lead to T-cell exhaustion and treatment failure. Novartis AG’s first-in-class anti-CD19, anti-CD3 and anti-CD2 IgG-like trispecific antibody PIT-565, which engages CD19+ on tumor cells, and CD3 (TCR signaling component) and CD2 (a costimulatory receptor) on T cells simultaneously to redirect T-cell cytotoxicity toward CD19-positive malignant B cells, has been designed to avoid T-cell exhaustion.
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.
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