At the recently launched Alzheimer’s & Parkinson’s Diseases Conference held in Vienna, Lotte Bjerre Knudsen from Novo Nordisk A/S, who has extensive experience in glucagon-like peptide-1 (GLP-1) research, delivered a plenary lecture focused on the role of GLP-1 receptor agonists, such as semaglutide, in attenuating neuroinflammation and neurodegeneration. Read More
As it prepares to advance its lead RNA editing candidate, AIR-001, into a phase I/II trial for alpha-1 antitrypsin deficiency, Airna Corp. Inc. closed an oversubscribed $155 million series B financing less than a year after completing its series A round. The company, based in Cambridge, Mass., with research operations in Tübingen, Germany, focuses not only on repairing harmful genetic variants found in rare genetic disorders, but also on introducing beneficial variants that improve health in common conditions. Read More
In a study published in Nature Communications, researchers from Nanjing University present a novel nanovaccine cocktail formulated by individual conjugation of antigen p210 and adjuvant CpG ODNs onto superparamagnetic iron oxide nanoparticles (SPIONs). Read More
Multiomic Health Ltd. and Alloy Therapeutics Inc. have signed a memorandum of understanding to jointly discover and develop first-in-class renal tissue-targeting drugs. Precision medicines for chronic renal conditions targeted to the kidney tissue of specific patient subpopulations are expected to improve efficacy and safety compared to conventional systemic drugs.
Cannabinoid CB1 receptors have been a potential target for nonopioid-based pain treatment, but actually targeting the pathway has been hindered by issues with tolerance and unwanted CNS side effects. Peripherally selective CB1 agonists developed to overcome these problems have not fully resolved these issues, meaning the peripheral selectivity has to be substantially enhanced. Read More
Dyne Therapeutics Inc. presented their most recent work to advance novel therapeutics for facioscapulohumeral muscular dystrophy (FHSD). FSHD is an autosomal dominant genetic disorder characterized by muscle weakness and atrophy. Dyne Therapeutics previously developed the FORCE platform, which uses an antigen-binding fragment (Fab) specifically targeting telomeric repeat binding factor-1 (TfR1) for targeted therapeutic delivery. Read More
Syndax Pharmaceuticals Inc. has disclosed extended purine tricyclic and bicyclic nucleosides as prodrugs reported to be useful for the treatment of viral infections. Read More
In type 2 diabetes patients, inadequate hyperglycemic control can lead to insulin-secreting β-cell exhaustion, dedifferentiation and eventual loss. Since adult β cells have limited proliferative capacity, this final stage cannot be reversed. Read More
Pannexin 1 (PANX1) forms channels that may release signaling metabolites that are involved in a variety of pathophysiological processes, such as asthma, diabetes, hypertension or inflammatory bowel disease (IBD), among others. Inhibition of PANX1 when dysregulated has been proposed as a therapeutic approach in the treatment of IBD. Read More
Scientists at Beijing Wehand-Bio Pharmaceutical Co. Ltd. and Institute of Materia Medica Chinese Academy of Medical Sciences & Peking Union Medical College have synthesized phthalazinone compounds with neuroprotective effects reported to be useful for the treatment of hemorrhagic and ischemic stroke. Read More
Systemic Bio LLC has developed a 3D bioprinted human Vascularized Integrated Organ System (h-VIOS) platform that better recapitulates the native-like liver architecture and vascularization, integrating PHHs and non-parenchymal cells. Read More
Researchers at Cimplrx Co. Ltd. and Samjin Pharmaceutical Co. Ltd. have identified ectonucleotide pyrophosphatase/phosphodiesterase family member 1 (ENPP1) inhibitors reported to be useful for the treatment of cancer, hypophosphatemia, osteoarthritis, cystic fibrosis, type 2 diabetes, Alzheimer’s disease, and viral and bacterial infections. Read More
Genzyme Corp. (Sanofi Genzyme) has divulged emopamil-binding protein (EBP) inhibitors reported to be useful for the treatment of multiple sclerosis, transverse myelitis, neuromyelitis optica, optic neuritis and Guillain-Barré syndrome. Read More
Clear cell renal cell carcinoma (ccRCC) is still a clinical challenge due to its high metastasis rate at diagnosis and therapy resistance. It accounts for about 75%-80% of RCC cases. The magnesium transporter NIPA4 (NIPAL4) may influence tumor progression, but there is lack of knowledge on its prognostic value and role in ccRCC. Read More
Mainline Biosciences (Shanghai) Co. Ltd. has described peptide-drug conjugates consisting of peptide targeting somatostatin receptors covalently bound to cytotoxic agents through a linker reported to be useful for the treatment of cancer. Read More
