Nephronophthisis (NPH) is a recessive cystic kidney disease responsible for 10% to 20% of pediatric end-stage renal disease cases. NPHP1 (nephrocystin 1) is the most frequently implicated among over 20 known causative genes. However, the pathogenesis of NPH remains unclear, and no effective therapies are available.
Smsbiotech Inc. has gained clearance in Australia to begin a phase I trial of its small mobile stem (SMS) cell therapy for chronic obstructive pulmonary disease (COPD).
Sarcopenia is an age-related condition in which muscle mass and strength decrease, leading to reduced overall physical performance. As fast-twitch muscle fibers are more impacted by age-related decline, strategies aimed at enhancing the regeneration and functionality of these fibers are essential to prevent the progression of sarcopenia.
Atherosclerosis is a chronic inflammatory condition and a known risk for the development of cardiovascular diseases. In animal models of atherosclerosis, macrophage pyroptosis is linked to the development and instability of atherosclerotic plaques, where gasdermin-D (GSDMD) plays a crucial role.
Hoth Therapeutics Inc. has released preclinical findings demonstrating the efficacy of HT-KIT, a novel targeted therapy for gastrointestinal stromal tumors (GIST).
Prime Medicine Inc. has announced its new preclinical program for the treatment of α1-antitrypsin deficiency (AATD). The program is advancing through its final stages of lead optimization, with an IND and/or clinical trial application (CTA) filing anticipated around the middle of next year.
Researchers from Senju Pharmaceutical Co. Ltd. presented results from preclinical studies that assessed the hyperthermic effects of SJP-0132, a selective antagonist of transient receptor potential vanilloid 1 (TRPV1), which is currently awaiting approval in Japan as an ophthalmic treatment to alleviate the signs and symptoms of dry eye disease.
Researchers at the German Cancer Research Center Deutsches Krebsforschungszentrum (DKFZ) and their collaborators have cast new light on the mechanisms by which hepatic stellate cells control liver metabolism and regeneration. The work builds on the concept of angiocrine signaling, established 15 years ago.
Researchers at Concept to Medicine Biotech Co. Ltd., Lepu Biopharma Co. Ltd. and Shanghai Miracogen Inc. have described antibody-drug conjugates consisting of trophoblast glycoprotein (TPBG, 5T4)-targeting antibodies covalently linked to cytotoxic drugs through linkers reported to be useful for the treatment of cancer.
Miracure Biotechnology Ltd. has divulged Toll-like receptor 7 (TLR7) and/or TLR8 agonists reported to be useful for the treatment of cancer, infections, respiratory disorders and immunological disorders.
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