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BioWorld - Wednesday, May 27, 2026
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3d illustration of human body muscle tissue anatomy
Musculoskeletal

FDA grants orphan drug designation to BLR Bio candidate for systemic sclerosis

April 12, 2024
BLR Bio LLC announced that the U.S. FDA has granted orphan drug designation to BLR-200, the company’s investigational therapy for the treatment of systemic sclerosis (SSc).
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C. auris illustration
Infection

Discovery of potent fungistatic agent with efficacy in mouse model of candidiasis

April 12, 2024
Researchers from Second Military Medical University PLA (Shanghai) and affiliated organizations announced the discovery and preclinical characterization of novel antifungal small-molecule candidates.
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Illustration of tumor
Cancer

TLR7 agonist SMU-L-11 demonstrates antitumoral activity in melanoma

April 12, 2024
Recent work at Southern Medical University, Guangzhou, focused on the discovery and synthesis of imidazoquinoline-based Toll-like receptor 7 (TLR7) agonists as potential candidates for the treatment of melanoma.
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Cancer

FDA clears IND for Lamassu Biotech’s SA53-OS

April 12, 2024
Lamassu Biotech Inc. has announced that its IND for SA53-OS has been cleared by the FDA in the U.S. The planned phase I/IIa trials will investigate the genetically targeted therapy that blocks the MDM2 protein, a key regulator of the tumor suppressor p53 gene.
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Microscope
Cancer

VB-15010, a potent and selective PARP-1 inhibitor and durable trapper, described

April 12, 2024
Previous studies have shown that targeting poly(ADP-ribose) polymerase 1 (PARP-1) demonstrated promising activity for cancer treatment.
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Vaccine target
Infection

Soligenix’s vaccine for Sudan ebolavirus granted orphan drug designation in US

April 12, 2024
The FDA has granted orphan drug designation to the active ingredient in Soligenix Inc.’s Suvax, a subunit protein vaccine of recombinantly expressed Sudan ebolavirus (SUDV) glycoprotein, for the prevention and post-exposure prophylaxis against SUDV infection. SUDV is a type of ebolavirus for which there is no current treatment or vaccine.
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Cancer

Betta Pharmaceuticals reports a highly potent and selective KRAS G12D inhibitor

April 12, 2024
Results of preclinical studies with a highly potent KRAS (G12D mutant) inhibitor for the treatment of cancer were recently reported by researchers from Betta Pharmaceuticals Co. Ltd. BPI-501836 displayed higher inhibitory activity at KRAS G12D, when compared to the reference compound (IC50=0.25 and 2.4 nM, respectively) in NanoBRET assays.
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Inflammatory

The Scripps Research Institute patents new Rab-27A/JFC1 interaction inhibitors

April 12, 2024
The Scripps Research Institute has disclosed Ras-related protein Rab-27A/synaptotagmin-like protein 1 (SYTL1; JFC1) interaction inhibitors reported to be useful for the treatment of endotoxemia, sepsis, systemic inflammatory response syndrome and cryopyrin-associated periodic syndromes (CAPS).
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Other news to note for April 11, 2024

April 11, 2024
Additional early-stage research and drug discovery news in brief, from: Cantargia, Compugen, Moleculin, Revolution, Vesicure.
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Infection

Schrodinger and Takeda scientists discover new 3CLpro inhibitors

April 11, 2024
Researchers at Schrodinger Inc. and Takeda Pharmaceutical Co. Ltd. have described 3C-like proteinase (3CLpro; Mpro; nsp5) inhibitors reported to be useful for the treatment of coronavirus acute respiratory syndrome.
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