Gray platelet syndrome is an autosomal recessive platelet disorder characterized by macrothrombocytopenia and deficiency or decreased levels of alpha granules that confer a grayish appearance to the platelets. The genetic cause is located at chromosome locus 3p21, affecting the NBEAL2 gene.
Privately held Vivacelle Bio Inc. has completed its phase IIa study of VBI-S in treating septic shock patients with severe hypovolemia, a drastic drop in blood pressure that can lead to organ failure and death. In the study, VBI-S, an intravenously injected fluid composed of phospholipid nanoparticles, increased blood pressure and improved organ function.
Previous research has suggested that factor VIII (FVIII) can regulate the osteoprotegerin (OPG)/RANKL system, which appears to play a role in hemophilic arthropathy. Investigators have now aimed to measure the OPG levels in patients with hemophilia A/B and assess their correlation with the levels of FVIII/FIX.
Glanzmann thrombasthenia (GT) is a rare bleeding disorder caused by defects in the expression of platelet surface integrins, such as integrin alpha-IIb (GPIIb, encoded by ITGA2B).
Roche Holding AG’s investigational monoclonal antibody, crovalimab, which inhibits part of the innate immune system in patients with the rare blood condition paroxysmal nocturnal haemoglobinuria, met its co-primary efficacy endpoints in a phase III trial that will form the basis of its approval submissions worldwide.
The combination of two sequencing techniques has unveiled features of a subpopulation of cells that could be producing plaques in atherosclerosis. This process is associated with an autoimmune component driven by CD4+ T cells, according to a study from researchers at Leiden University.
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.
The combination of two sequencing techniques has unveiled features of a subpopulation of cells that could be producing plaques in atherosclerosis. This process is associated with an autoimmune component driven by CD4+ T cells, according to a study from researchers at Leiden University.
“I think that we can clearly say now that atherosclerosis is a very clear autoimmune component. It is a multifactorial disease, a combination of genes, and lifestyle, but also inflammation and the immune system,” Ilze Bot and Bram Slütter, associate professors at the Division of Biotherapeutics of Leiden University, told BioWorld.
Sickle cell disease (SCD) is autosomal recessive disorder caused by mutations in the β-globin gene, and induction of fetal γ-globin is considered an established therapeutic strategy for the treatment of this disease. A research team led by scientists at Kyorin Pharmaceutical Co. Ltd. has discovered RK-701, a small-molecule inhibitor of G9a and G9a-like protein (GLP) as a potential therapeutic agent for SCD.
Coming off a tough year for raising money, Cadrenal Therapeutics Inc. is leading the charge into a new year by completing 2023’s first U.S. IPO. The company closed its IPO of 1.4 million shares at $5 each for $7 million. The stock (NASDAQ:CVKD) began trading Jan. 20 and closed 9.37% downward on Jan. 25 at $2.90 per share.
RSS
