Ribomic’s umedaptanib pegol (RBM-007) looks to have some advantages compared to competitors in the achondroplasia space, and the company plans to progress the oligonucleotide-based aptamer that targets anti fibroblast growth factor 2 to phase III trials, Ribomic’s business development head, Kihei Yamashita, told BioWorld.
Partly focused on delivery challenges that have limited the reach of RNA medicines, new biotech company Axelyf Inc. closed a $2.6 million seed round to support development of its AXL technology and to advance lead autoimmune candidate AXL-003.
Worrisome new signals caused the U.S. FDA – which earlier this month OK’d revised labeling for Valneva SE’s chikungunya virus vaccine Ixchiq – to suspend the product’s license altogether. Regulators pointed to four added reports of serious adverse events consistent with chikungunya-like illness, and told Valneva, of Saint Herblain, France, that the company must stop U.S. shipping and sales of the product. Shares (NASDAQ:VALN) closed Aug. 25 at $9.43, down $2.21, or 19%.
The frontiers of xenotransplantation have been further extended with a pig-to-human lung transplant, the first time an organ that is directly exposed to the external environment – with the associated risk of respiratory pathogens – has been transplanted. The genetically modified pig lung remained viable and functional for nine days, after it was transplanted into a 39-year-old man who was declared brain dead following a hemorrhagic stroke.
In another deal between the two companies that could be worth more than $1 billion, Abbvie Inc. is buying Gilgamesh Pharmaceuticals Inc.’s lead candidate bretisilocin (GM-2505) for up to $1.2 billion, including an undisclosed up-front payment and development milestones. Privately held Gilgamesh has the psychedelic compound for treating moderate to severe major depressive disorder in a phase II study.
Deficiencies in interferon-stimulated gene 15 (ISG15), a protein that normally regulates the immune response, causes mild but persistent inflammation. However, its absence also provides an unexpected advantage by increasing resistance to viral infections. Inspired by this condition and using mRNA technology, scientists at Columbia University and the Icahn School of Medicine at Mount Sinai have developed a broad-spectrum antiviral platform.