Little more than a month after the U.S. FDA approved the first gene therapy for adults with hemophilia B, Uniqure NV’s Hemgenix, strong phase III data have come from Pfizer Inc. The Pfizer results show fidanacogene elaparvovec, a vector containing an AAV capsid and a high-activity human coagulation factor IX (FIX) gene for treating adult men with moderately severe to severe hemophilia B, hit the primary endpoint in the phase III Benegene-2 study. The one-time therapy is designed to allow those living with hemophilia B to be able to produce FIX instead of receiving regular, ongoing doses of exogenous FIX.
Lunac Therapeutics Ltd. has synthesized coagulation factor XII (FXIIa) inhibitors reported to be useful for the treatment of arthritis, Alzheimer's disease, disseminated intravascular coagulation, pulmonary embolism, myocardial infarction, diabetic retinopathy, stroke and deep venous thrombosis, among others.
CRISPR gene editing has been one of the important advances of the last decade, in biotechnology and increasingly in medicine. First applied to human cells in 2013, and honored with the 2020 Nobel Prize in Physiology or Medicine, its meteoric rise can make CRISPR look like the molecular equivalent of a miracle healer. But in the research and clinical trenches, CRISPR-based approaches, like any others, need to find applications where their desired effects outweigh their side effects. And finding those applications necessitates ways to identify off-target effects.
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.
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