Ahead Therapeutics SL has been awarded an Industry Discovery and Development Partnership (IDDP) grant from Breakthrough T1D to support progression of the company’s proprietary Mimi-Top (mimicking tolerogenic particles) platform.
DFNB16, a recessive form of mild-to-moderate human deafness, is caused by mutations in the STRC gene, which encodes stereocilin, a protein essential for the effective function of outer hair cells as cochlear amplifiers. Researchers from the Institut Pasteur and Sorbonne Université in Paris, France, have developed and evaluated the efficacy of an Strc gene therapy in a mouse model of DFNB16.
Formation Bio Inc. and Jiangsu Chia Tai Feng Hai Pharmaceutical Co. Ltd. have announced Formation Bio’s acquisition of worldwide rights, excluding Greater China, to FHND-5032, an oral, small-molecule miR-124 activator being developed for the treatment of autoimmune diseases.
Insilico Medicine Cayman Topco has nominated ISM-0676 as a preclinical candidate targeting the glucose-dependent insulinotropic polypeptide receptor (GIPR).
Bone metastasis represents a challenge in cancer therapy because of the independency of immunosuppression, neuropathic pain and osteolytic destruction. Recent evidence has suggested the tumor microenvironment can be reshaped through the activation of stimulator of interferon genes protein (STING) and pyroptosis induction.
How much have AI applications learned, and how can one know their capabilities if they are being evaluated with an exam that is far too easy? In 2024, with the publication of the previous benchmark to measure AI performance, the field debated whether existing assessments would keep pace with the rapid progress of AI.
Seamless Therapeutics has received big pharma endorsement of its proprietary recombinase gene editing platform, sealing a potential $1.12 billion deal with Eli Lilly and Co. to apply the technology in hearing loss.
Beijing Avistone Pharmaceuticals Biotechnology Co. Ltd. has patented HER2 (erbB2) Ex20Ins mutant inhibitors. They are reported to be useful for the treatment of cancer, atherosclerosis and pulmonary fibrosis.
Shanghai Haiyan Pharmaceutical Technology Co. Ltd. and Yangtze River Pharmaceutical Group have divulged new isoindoline proteolysis targeting chimeric (PROTAC) compounds comprising cereblon (CRBN) ligands covalently linked to a eukaryotic peptide chain release factor GTP-binding subunit ERF3A (GSPT1)-targeting moiety. They are reported to be useful for the treatment of cancer.
Jiangsu Hengrui Pharmaceuticals Co. Ltd. and Shanghai Hengrui Pharmaceutical Co. Ltd. have identified oxyheteroaryl compounds acting as sodium channel protein type 10 subunit α (Nav1.8) blockers. They are reported to be useful for the treatment of pain, urinary incontinence, multiple sclerosis, cough, Charcot-Marie-Tooth disease and arrhythmia.