Researchers from Trinity College Dublin, Princeton University and collaborators have discovered that blocking the uptake of lipids by immune cells within the ascites microenvironment could reshape treatment for advanced ovarian cancer. Read More
Among the most significant genetic risk factors for age-related macular degeneration is the Y402H variant of complement factor H, which, along with its splice isoform FHL-1, impairs binding to the retinal surface. Read More
Capsida Biotherapeutics Inc. has gained IND clearance from the FDA for CAP-002, its first-in-class, intravenously administered gene therapy for syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE). Dosing in the phase I/IIa SYNRGY trial will begin in the third quarter of this year. Read More
Current therapies for chronic hepatitis B virus (HBV) infection can effectively suppress viral replication but do not achieve a functional or complete cure. Capsid assembly modulators inhibit the assembly of viral capsids, prevent the encapsidation of pregenomic RNA, and interfere with both the formation and amplification of covalently closed circular DNA, which is essential for viral persistence. Read More
Work at Scorpion Therapeutics Inc. has led to the identification of cellular tumor antigen p53 (TP53) (Y220C mutant) activators reported to be useful for the treatment of cancer. Read More
Inhibiting the sodium-hydrogen exchanger 1 (NHE1), which simultaneously imports Na+ and exports H+ in cardiac cells, shows therapeutic potential against heart failure. Read More
Epidermal growth factor receptor (EGFR), when overactive or overexpressed, may lead to tumor growth and spread, and is thus a robust target for therapy. Read More
Hitgen Ltd. has prepared and tested interleukin-17 receptor A (IL17RA; CD217) antagonists reported to be useful for the treatment of autoimmune disease, cancer, infections, asthma, multiple sclerosis, psoriasis, rheumatoid arthritis and inflammatory disorders. Read More
Radiopharmaceuticals can offer a targeted approach for cancers that have limited therapeutical options. Abdera Therapeutics Inc. recently presented results of their novel 5T4-targeted radiopharmaceutical. Read More
Mavrix Bio has received IND clearance from the FDA for MVX-220, an investigational AAV gene therapy for the treatment of Angelman syndrome. The company expects to initiate its first-in-human study, ASCEND-AS, in the second half of this year. Read More
Histone acetyltransferase p300 (EP300) inhibitors have been reported in a Kronos Bio Inc. patent and are described as potentially useful for the treatment of cancer and inflammatory disorders. Read More
Some deubiquitinating enzymes may help protect against cancer, but others appear to promote it. Researchers from the First Affiliated Hospital of Zhejiang University have discovered that the deubiquitinator PSMD14 helps drive bladder cancer, and they have defined at least one signaling pathway through which it works, opening up possibilities for development of new treatments. Read More
A study has demonstrated the potential of a novel ligand-based CAR T-cell therapy for targeting CD7-positive T-cell malignancies, including T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphomas. The receptor CD7 is a prominent target antigen, being expressed in around 95% of T-ALL, 50% of peripheral T-cell lymphomas and 10% of acute myeloid leukemias. Read More
