A 6.5-month-old boy with the rare inherited urea cycle disorder ornithine transcarbamylase (OTC) deficiency has responded positively in a targeted in vivo gene editing trial, in which a correct copy of a defective gene was inserted at a precise locus in the genome.
Shionogi & Co. Ltd. won a $375 million project agreement from the Rapid Response Partnership Vehicle to develop its 3CL protease inhibitor, S-892216, as a long-acting injectable for COVID-19 pre-exposure prophylaxis.
Boston Scientific Corp. reported positive data for two key atrial fibrillation therapies at AF Symposium 2025 on January 17. In late-breaking data presentations, the ADVANTAGE AF trial for the Farapulse PFA system met its primary endpoints, showing a 2.3% safety event rate and 63.5% effectiveness rate in treating persistent atrial fibrillation (AF) patients, with an 85.3% symptomatic AF recurrence-free rate. Additionally, a sub-analysis of the OPTION trial demonstrated that the Watchman FLX device significantly reduced bleeding outcomes compared to oral anticoagulants.
Immunoforge Co. Ltd.’s approval of an IND by the Korea Ministry of Food and Drug Safety reminded Wall Street – not that anybody needed reminding – about the marketplace jostle among therapies for chronic myeloid leukemia (CML), where a number of drugs are cleared by the U.S. FDA but significant need remains in terms of efficacy as well as tolerability.
With positive data in hand from its phase IIb trial testing immunotherapy candidate OST-HER2 in osteosarcoma, OS Therapies Inc. anticipates a regulatory filing this year seeking accelerated approval from the U.S. FDA, putting the firm on track to receive a potentially profitable rare pediatric disease priority review voucher that could help fund further R&D work. Should OST-HER2 go on to win approval, it would mark the first new therapy in more than 40 years for osteosarcoma, an aggressive bone cancer characterized by high rates of metastases, often to the lungs, and disease recurrence.
Natera Inc., a developer of cell-free DNA testing, provided an update on its product portfolio at the J.P. Morgan Healthcare Conference on Jan. 15. The company disclosed a new version of the Signatera assay that leverages the genome. Natera said the test, now available for research and clinical use, enables bespoke assay design from a whole genome sequence of a patient’s tumor.
Esocap AG aims to transform the treatment of eosinophilic esophagitis with its drug delivery technology, ESO-101, designed to increase mucosal contact time and drug deposition in the esophagus. Data from the recent ACESO study showed that ESO-101 was safe and well tolerated and improved both histologic and endoscopic outcomes in patients with active eosinophilic esophagitis.
The 2025 edition of Clarivate’s Drugs to Watch features 11 candidates or approved therapeutics that may well revolutionize treatments or become blockbusters. The 12th annual report has a strong track record. Twelve of the 13 drugs from the 2024 Drugs to Watch report have been approved and launched.
Yuhan Corp., Oscotec Inc. and Genosco Inc. were three companies with Asian ties to reap benefit from Johnson & Johnson’s stellar top-line Mariposa study results, wherein J&J’s Rybrevant (amivantamab-vmjw) and Lazcluze (lazertinib) combo regimen beat out Astrazeneca plc’s standard of care in non-small-cell lung cancer (NSCLC).
Day two of the J.P Morgan Healthcare Conference rolled on with positive data from Sling Therapeutics Inc. that is leading the company to a phase III study in treating thyroid eye disease. The privately held company posted top-line efficacy and safety results from a phase IIb/III study of its lead candidate, linsitinib, which hits its primary endpoint with statistical significance at the twice-daily, 150-mg oral dose.
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