Beijing Gensciences Inc. has developed a FVIIIa-mimetic bispecific antibody named SS-315 for the treatment of hemophilia A. SS-315 was developed by targeting FX with its upper Fab arms and FIXa with its down-side scFv arms, respectively; the hemostatic potential of SS-315 was investigated in vitro and in vivo.
Glycoprotein VI (GPVI) is a platelet collagen receptor involved in platelet activation and an emerging target for treating thrombotic disorders such as ischemic stroke. Data have been presented by University of Würzburg scientists regarding a humanized anti-GPVI Fab antibody, EMA-601, with unprecedented potency in vitro and in vivo.
There is growing evidence of the role of soluble endoglin in the biology of platelets, including thrombosis. French researchers have investigated the role of genetic variants in the gene encoding endoglin, ENG, and the risk of venous thrombosis development.
Transmembrane serine protease 6 (TMPRSS6) is a negative regulator of hepcidin, which is the main iron homeostasis-regulating hormone. Regeneron Pharmaceuticals Inc. has recently presented preclinical data for the monoclonal antibody targeting TMPRSS6, REGN-7999, which is being developed for the treatment of iron overload.
Researchers from Incyte Corp. and collaborators presented the preclinical profile of INCB-160058, an ATP-competitive small-molecule inhibitor of the JAK2 V617F mutant sparing its wild-type (WT) form, designed for the treatment of JAC2 V617F mutation-positive myeloproliferative neoplasms with potential as a first-in-class JAK2 V617-inhibiting drug.
Among the acquired mutations in the calreticulin (CALR) gene, both 52 bp deletion (del52) and 5 bp insertion (ins5) are among the most frequent and are linked to two different types of myeloproliferative neoplasms (MPNs).
Aberrant overactivity of platelets may lead to the formation of occlusive thrombi and consequently lead to myocardial infarction or stroke. At the recent EHA meeting, researchers from the University of Michigan and Cereno Scientific AB presented data regarding their prostacyclin (IP) receptor agonist CS-585 as a thrombolytic.
B-cell lymphoma 6 (BCL6) is up-regulated in several B-cell malignancies where it acts as a transcription factor activity mediator to induce and maintain lymphomagenesis.
The first oral session in the acute myeloid leukemia (AML) translational research track of June 15, was given by Eliza Yankova, from the University of Cambridge, who presented collaborative studies done together with Storm Therapeutics Ltd. outlining pharmacological inhibition of METTL1 as a therapeutic strategy in AML treatment.