ABI-201 is an engineered AAV.N54 vector that expresses a soluble CD59 variant and a complement 3 (C3) inhibitor fusion protein. ABI-201 was developed by Avirmax Biopharma Inc. to confer photoreceptor protection against retinal damage. The effects of ABI-201 were tested in a sodium iodate-induced retinal damage model in nonhuman primates, a widely used model for testing therapies for age-related macular degeneration (AMD).
“I’m a pediatrician in metabolic diseases, and every day in my clinical work I’m confronted with our lack in effective therapies for our patients.” That was the sobering introduction by Sabine Fuchs in her talk at the 2025 Congress of the European Association for the Study of the Liver in Amsterdam this week. The nature of metabolic diseases makes it difficult to develop treatments for them. “There are over 1,500 diseases known by now, and it is just very difficult to develop therapies for each and every individual rare disease.”
Additional early-stage research and drug discovery news in brief, from: Akava Therapeutics, Galmed Pharmaceuticals, Kiora Pharmaceuticals, Mira Pharmaceuticals, Palatin Technologies, Thetis Pharmaceuticals.
Nuevocor Pte. Ltd. has closed a $45 million series B, enabling it to move lead gene therapy NVC-001 into the clinic in the treatment of an inherited form of cardiomyopathy.
Canwell Biotech Ltd. has described tyrosine-protein phosphatase non-receptor type 11 (PTPN11; PTP-2C; SHP-2) inhibitors reported to be useful for the treatment of cancer.
Gilead Sciences Inc. has divulged bridged tricyclic carbamoylpyridone prodrugs acting as HIV integrase inhibitors reported to be useful for the treatment of HIV infection.
Korea Research Institute of Chemical Technology has identified glutaminase inhibitors reported to be useful for the treatment of cancer and autoimmune diseases.
Researchers at Foshan Ionova Biotherapeutics Co. Ltd., Guangdong Touchstone Translational Research Institute Co. Ltd. and Shenzhen Ionova Life Science Co. Ltd. have synthesized interleukin-17A (IL-17A) inhibitors reported to be useful for the treatment of inflammatory disorders.
Scientists at Arontier Co. Ltd. and Korea Research Institute of Chemical Technology have disclosed ectonucleotide pyrophosphatase/phosphodiesterase 2 (ENPP2; autotaxin) inhibitors reported to be useful for the treatment of hepatic steatosis, hepatic fibrosis, cirrhosis, liver failure, hepatitis and liver cancer, among others.
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