Spirits were high at the 2023 Annual Meeting of the American Society of Hematology (ASH), buoyed by U.S. FDA approval of the first two gene therapies for sickle cell disease (SCD) the day before the conference kicked off in San Diego.
Cereno Scientific AB has completed the preclinical safety program for CS-014, a histone deacetylase (HDAC) inhibitor in development for arterial and venous thrombosis prevention.
Sernova Corp. has announced that its hemophilia A program, combining the Sernova Cell Pouch with a patient’s own cells corrected for the production of factor VIII (FVIII), has been awarded U.S. orphan drug and rare pediatric disease designations.
Sirius Therapeutics Inc. has submitted an application in Australia to begin a first-in-human trial of SRSD-107 for the prevention and treatment of thromboembolic disorders.
Generation Bio Co. has announced a breakthrough in its nonviral genetic medicine platform with the development of a proprietary, novel DNA called immune-quiet DNA (iqDNA).
Research at Purdue Research Foundation has led to the identification of SYK kinase inhibitors reported to be useful for the treatment of glucose-6-phosphate dehydrogenase deficiency, sickle cell disease, α- and β-thalassemia.
Recently, researchers at Cincinnati Children’s Hospital, in collaboration with colleagues in Japan, have developed a human vascular organoid model that accurately mimics the vascular damage caused by SARS-CoV-2.
Baudax Bio Inc.’s lead clinical candidate, TI-168, has been awarded U.S. orphan drug designation by the FDA for the treatment of hemophilia A with inhibitors. TI-168 is a next-generation, factor VIII (FVIII)-specific regulatory T-cell (Treg) therapy designed to address hemophilia A in patients with FVIII inhibitors.
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