Just in time for the Chinese New Year, Mabwell Bioscience Co. Ltd. announced that its U.S. subsidiary, Mabwell Therapeutics Ltd., closed a licensing deal with Disc Medicine Inc. worth up to $412 million for preclinical candidate 9MW-3011, a humanized monoclonal antibody (MAb) that regulates iron metabolism.
Mabwell Therapeutics Inc., a wholly owned subsidiary of Mabwell (Shanghai) Bioscience Co. Ltd., has entered into a license agreement with Disc Medicine Inc. for 9MW3011 (MWTX-001, MWTX-002 & MWTX-003) for hematologic diseases.
By combining drug sensitivity with genomic profiling of tumor cells, a study from St. Jude Children's Research Hospital with more than 800 patients has shown a wide diversity in drug sensitivity for pediatric acute lymphoblastic leukemia (ALL) and defined six patterns of response to treatment. “This work provides a framework for ‘functional precision medicine’,” corresponding author Jun Yang, vice chair of the Department of Pharmacy and Pharmaceutical Sciences at St. Jude Children's Research Hospital, told BioWorld.
Scientists based at Beijing Institute of Radiation Medicine have reported preclinical evaluation of a novel recombinant activated human factor VII (rFVIIa), GEN-0828, being developed as potential candidate for the treatment of hemophilia and trauma hemorrhage. GEN-0828 is rFVIIa that was mainly produced from Chinese hamster ovary (CHO) cells.
Researchers from Beam Therapeutics Inc. presented the discovery and preclinical evaluation of an engineered stem cell antibody paired evasion (ESCAPE) strategy for antibody (Ab)-mediated autologous hematopoietic stem cell (HSC) therapy conditioning for the treatment of hemoglobinopathies.
The serious adverse event in the first phase I/II patient dosed with nulabeglogene autogedtemcel (nula-cel) – a gene editing, autologous hematopoietic stem cell therapy for sickle cell disease (SCD) – may have involved the quality of the stem cells and not the conditioning regimen or characteristics of the patient at baseline. But researchers won’t know until more work is done.
Jasper Therapeutics Inc. may have found a way around toxicity with current approaches in sickle cell disease (SCD) conditioning approaches, if phase I/II data with briquilimab stay consistent – and the drug already has proved itself across a range of indications. Wall Street liked the prospect, sending the Redwood City, Calif.-based firm’s shares (NASDAQ:JSPR) on a wild ride to close at $2.74, up $2.26, or 476%.
The most common cause of anemia in chronically ill hospitalized patients is due to inflammatory anemia (IA) that is caused indirectly by diseases such as autoimmune, cancer, chronic kidney disease, congestive heart failure, or pulmonary disease. The precise and common etiology of these diseases involves hypercytokinemia that leads to excessive increases in hepcidin, a master regulator of iron homeostasis that blocks intestinal iron absorption when levels are too persistently high.
The primary reason for hospitalization of patients with sickle cell disease (SCD) is an acute systemic painful vaso-occlusive episode (VOE), which serves as an antecedent to acute chest syndrome (ACS). It has been previously demonstrated that P-selectin-dependent neutrophil-platelet aggregation and the complement pathway activation contribute to the vaso-occlusive pathophysiology in SCD.
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.