Vega Therapeutics Inc. emerged from stealth in advance of a podium presentation at this year’s American Society of Hematology meeting in New Orleans. The company, a spin-out from Star Therapeutics Inc., will disclose preclinical data on its lead drug candidate VGA-039, a first-in-class antibody in development for von Willebrand disease.
Researchers from BioCryst Pharmaceuticals Inc. have developed a series of plasma kallikrein inhibitors with potential for the treatment of hereditary angioedema (HAE).
Does cancer cause autoimmune disease or is it the other way around? In looking at the question of which comes first, the chicken or the egg, researchers at the Garvan Institute of Medical Research in Australia found that a genetic mutation that alters immune cells in leukemia is behind certain autoimmune disorders.
Enzyre BV reported closing a $12.5 million series A round to ramp development of its micro diagnostics platform used for monitoring blood coagulation status at home.
The U.S. FDA gave its go-ahead for Hemgenix (etranacogene dezaparvovec-drlb), Uniqure NV’s one-time gene therapy – the first for the treatment of adults 18 and older living with hemophilia B. Patients have been waiting “maybe beyond two decades” for a new therapy, Uniqure CEO Matthew Kapusta said. Hemgenix emerged from pioneering work by St. Jude Children’s Research Hospital and the University College London.
Merck & Co. Inc. is adding to its hematology assets in the takeover of Imago Biosciences Inc. for $36 per share, an arrangement with an equity value of about $1.35 billion. Shares of Imago (NASDAQ:IMGO) took flight as Wall Street learned of the deal, ending the day at $35.59, up $18.19, or 104%. Imago is working on new drugs for myeloproliferative neoplasms and other bone marrow diseases. Lead candidate bomedemstat (IMG-7289), an orally available inhibitor of the epigenetic enzyme lysine-specific demethylase 1 (LSD1), is undergoing phase II trials for the treatment of essential thrombocythemia, myelofibrosis and polycythemia vera, along with other indications.
Shanghai Meiyue Biotech Development Co. Ltd. has identified macrocyclic compounds acting as coagulation factor XIa (FXIa) inhibitors reported to be useful for the treatment of thrombosis.
Protagonist Therapeutics Inc. has disclosed hepcidin mimetic conjugates acting as solute carrier family 40 member 1 (SLC40A1, ferroportin) ligands with extended half-life and stability.
Galecto Inc.’s late-September news from its ongoing phase IIa trial with GB-2064 in myelofibrosis hiked shares of the Boston-based firm and piqued interest in the lysyl oxidase-like 2 (LOXL2) target, where such names as Gilead Sciences Inc. and Pharmaxis Ltd. also have been busy.
X4 Pharmaceuticals Inc.’s recent stock-boosting phase Ib news with lead candidate mavorixafor fueled already-strong interest in finding an alternative therapy for neutropenia and deepened esteem for the drug’s long-known mechanism of action.
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