“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.” Read More
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). Read More
Viverita Therapeutics Inc. has established a strategic research collaboration with Boehringer Ingelheim Pharma GmbH & Co. KG aimed at accelerating the discovery and validation of novel therapeutic targets for cancer. Read More
Interleukin-10 (IL-10) is an immunoregulatory cytokine produced by a variety of immune cells, including type 1 regulatory T cells and macrophages, as well as nonimmune cells such as epithelial cells and fibroblasts. Read More
Genprex Inc. and University of Pittsburgh have entered into a new sponsored research agreement (SRA) to study GPX-002, Genprex’s gene therapy for diabetes in animal models of type 1 and type 2 diabetes. Read More
Metis Pharmaceuticals Inc. recently presented a novel mRNA-encoded human papillomavirus (HPV) 16/18 therapeutic vaccine for HPV-driven malignancies. Read More
Malaria continues to exact heavy financial and socioeconomic burdens in several parts of the world, with 249 million cases and more than 630,000 deaths reported in 2022. Two-thirds of malaria-related deaths occur among children younger than 5 years. Read More
Scientists at ETH Zürich, F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have synthesized fluorescent probes acting as monoglyceride lipase (MGLL; MAGL) inhibitors reported to be useful for diagnostic imaging of MAGL. Read More
Bristol Myers Squibb Co. has identified molecular glue degraders comprising cereblon ligands and a eukaryotic peptide chain release factor GTP-binding subunit ERF3A (GSPT1)-targeting moiety acting as GSPT1 degradation inducers reported to be useful for the treatment of cancer. Read More
Stroke accounts for nearly 12% of all deaths worldwide, making it the second leading cause of death. In acute ischemic stroke, the ischemia and subsequent return of blood flow create oxidative and nitrosative stress that can overwhelm neurons and disable important circuitry. Read More
The expression of tissue factor (TF) is limited in normal tissues to the perivascular compartment where it acts as the receptor for the serin protease factor VIIa. This signaling axis triggers the initiation of extrinsic coagulation protease cascade. Read More
Solute carrier family 25 member 19 (SLC25A19) is a transporter protein of thiamine pyrophosphate across cellular membranes, which is needed as a cofactor for multiple metabolic enzymes and is important for homeostasis regulation. It was hypothesized that SLC25A19 may be a pan-cancer marker and a therapeutic target, and more concretely in hepatocellular carcinoma (HCC). Read More
Tenvie Therapeutics Inc. has described endosomal/lysosomal proton channel TMEM175 modulators reported to be useful for the treatment of cancer, neurological disorders, lysosomal storage disorders and inflammatory disorders. Read More
