Genprex Inc. has released preliminary in vivo proof-of-concept data for GPX-002, the company’s diabetes gene therapy drug candidate, in a nonhuman primate (NHP) model of type 2 diabetes and in a mouse model of type 2 diabetes. GPX-002 is being developed for the potential long-term control of both type 1 and type 2 diabetes.
Hyloris Pharmaceuticals SA has entered into a license agreement with Arbormed Co. Ltd. for exclusive rights in Europe and Turkey to Arbormed’s novel injectable product candidate for Wilson’s disease.
A review of 2025's noteworthy advances in medical research, including GLP-1 receptor agonists as anti-aging drugs, tumor-agnostic therapies and xenotransplants.
CSPC Pharmaceutical Group Ltd. has obtained approval from the National Medical Products Administration (NMPA) in China to conduct clinical trials of SYH-2072 (tablets), a highly selective and potent aldosterone synthase inhibitor. It is being investigated as a potential treatment for uncontrolled hypertension and primary aldosteronism.
Fortvita Biologics Inc. has described glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of autoimmune diseases, cardiovascular disorders, inflammatory disorders, diabetes, lung diseases, neurological disorders, obesity and renal disorders, among others.
Gene editing technologies are moving forward in preclinical development with innovative strategies designed to treat diseases at their root and even reverse them. However, many approaches still struggle to reach target cells or tissues – either they fail to arrive, or their efficacy is low. In vivo therapies face numerous challenges, but despite these hurdles, 2025 has marked a year of remarkable progress.
Aché Laboratórios Farmacêuticos SA has identified compounds acting as sodium/glucose cotransporter 1 (SGLT-1), SGLT-2 and dipeptidyl peptidase 4 (DPP4; CD26) inhibitors reported to be useful for the treatment of cardiovascular, renal and metabolic diseases.
Enveda has obtained IND clearance from the FDA and commenced dosing in a phase I study of ENV-308, a once-daily oral therapy designed for chronic weight management.
Rona Therapeutics Co. Ltd. has announced the submission of RN-5681 to the Australian Human Research Ethics Committee (HREC), and anticipates dosing to begin in a phase I trial in the first quarter of next year.
Confo Therapeutics NV has nominated CFTX-2034 as a development candidate to treat conditions linked to hyperinsulinemic hypoglycemia, specifically post-bariatric hypoglycemia (PBH). The company is now advancing CFTX-2034 through IND-enabling studies.
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