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Dermatologic

Biocryst outlines preclinical pipeline development

Nov. 6, 2023

Biocryst Pharmaceuticals Inc. has highlighted progress in its pipeline, with five programs set to enter the clinic in the next 24 months.

Drug Design, Drug Delivery & Technologies

Generation Bio develops immune-quiet DNA to be used as cargo in all programs

Oct. 19, 2023

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).

Hematologic

Purdue Research Foundation patents SYK inhibitors

Oct. 13, 2023

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.

Hematologic

GC Biopharma’s GC-1126A awarded US orphan drug designation for thrombotic thrombocytopenic purpura

Oct. 6, 2023

The FDA has awarded orphan drug designation to GC Biopharma Corp.’s GC-1126A for thrombotic thrombocytopenic purpura (TTP).

Infection

Human blood vessels transplanted in mice reveal COVID-19 coagulation molecule

Oct. 6, 2023

By Subhasree Nag

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.

Illustration of blood vessel that isn't clotting due to hemophilia

Hematologic

FDA awards orphan drug designation to Baudax’s TI-168 for hemophilia A with inhibitors

Sep. 29, 2023

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.

Drug Design, Drug Delivery & Technologies

Precision Biosciences outlines pipeline progress

Sep. 12, 2023

Precision Biosciences Inc. uses its proprietary Arcus platform to develop in vivo gene editing therapies and has outlined new data from its wholly owned and partnered pipeline.

Thrombus in bloodstream with platelets and fibrin

Hematologic

RBD-4059, the first siRNA-based strategy targeting FXI, is ready for human development

Sep. 4, 2023

Current anticoagulant strategies include small-molecule inhibitors and biological entities targeting factor XI (FXI) which, although effective, still have bleeding as a major risk.

Fluorescence microscopy image of mitochondria

Hematologic

Mitochondrial modulator rejuvenates blood stem cells

Sep. 4, 2023

By Nuala Moran

Researchers have demonstrated that inhibiting mitophagy in ‘old’ hematopoietic stem cells (HSCs) completely restored their blood reconstitution capabilities, raising the prospect of addressing the age-related weakening of the immune system that stems from HSCs deteriorating over time.

Hematologic

CS-585 prevents platelet clot formation without increasing the risk for bleeding

Sep. 1, 2023

Researchers from University of Michigan presented preclinical data for the novel oxylipin analogue CS-585 being developed as an antithrombotic agent. CS-585 was synthesized as an orally available analogue of 12(S)-hydroxy-eicosatrienoic (12-HETrE), with potent and selective prostacyclin (IP) receptor agonist activity and ability.

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