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.
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.
Epidermal growth factor receptor (EGFR), when overactive or overexpressed, may lead to tumor growth and spread, and is thus a robust target for therapy.
Inhibiting the sodium-hydrogen exchanger 1 (NHE1), which simultaneously imports Na+ and exports H+ in cardiac cells, shows therapeutic potential against heart failure.
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.
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.
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.
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.
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