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.
Sanegenebio has obtained clinical trial clearance in China for SGB-7342, an siRNA medicine targeting inhibin β E (INHBE) for the treatment of obesity. A phase I trial is scheduled to begin early next year.
Immusoft of CA Inc., a wholly owned subsidiary of Immusoft Corp., has announced that the FDA has granted orphan drug designation to ISP-002, the company’s investigational engineered B-cell therapy for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.
Fat tissue balances energy by storing lipids during times of abundance and mobilizing them when needed, yet sustained metabolic stress demands mechanisms that limit excessive lipid loss. Researchers at the University of California San Diego (UCSD) report that stress-induced fat breakdown triggers a neutrophil response in visceral adipose tissue that feeds back to restrain lipolysis and preserve lipid reserves.
Gene editing can repair mutations that prematurely halt protein synthesis, resulting in incomplete peptides that cause various diseases. However, other approaches achieve the same effect without altering the genome. Startup Alltrna Inc. has developed a strategy based on transfer RNA (tRNA) to bypass the premature stop codons that end early protein translation. The company already has a first clinical candidate that could treat metabolic diseases such as methylmalonemia (MMA) or phenylketonuria (PKU).
RSS
