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Zanvastro is now the first approved medicine for Alexander disease. Elsewhere, an immune system specialist posted positive results and a cell therapy developer turned to layoffs.
Ionis Pharmaceuticals’ zilganersen, brand name Zanvastro, is the first disease-modifying therapy approved for the neurological disorder Alexander disease. While Ionis has experienc...
Hibsago is viewed as a potential “functional cure” for chronic infections and an important part of the British drugmaker’s goal of reaching £40 billion in annual revenue by 2031.
The offering will support a trio of medicines in or nearing clinical testing, as well as earlier research into RNAi treatments that get into tissues outside the liver.
Results published in NEJM raised questions about the benefits “silencer” medications provide on top of standard therapies for TTR cardiomyopathy and amplified investor pressure on...
The results position Roche to bring a new kind of medicine to market for IgA nephropathy, a disease that’s drawn considerable attention of late from drugmakers.
Pelacarsen didn’t protect heart health despite lowering levels of a protein particle, “Lp(a),” in a large clinical trial — a surprise result with important implications for cardiov...
City Therapeutics’ lead RNA interference therapy could become an alternative to blockbuster-selling anticoagulants. The company’s technology for designing RNAi drugs and delivering...
A Lutathera copycat has become the first “radioligand equivalent” cleared by regulators. Elsewhere, Novo dropped the “Nordisk” from its name and a novel RNAi drug showed early prom...
ADARx is the only maker of RNA-based drugs to go public since 2024 and the first RNAi biotech to price an IPO in the U.S. in more than a decade.
One Failed Heart-Drug Trial Hits Ionis, Novartis and Amgen: What Happened and Why It Matters Biotechnology stocks are being punished today after a closely watched cardiovascular tr...
ADARx Pharmaceuticals upsized its IPO and raised additional cash through a private placement with partner AbbVie. Lead ADARx program onvuzosiran is in Phase 3 testing for the rare...
The acquisition gives the German company a promising blood disorder medicine that passed a Phase 2 test and could threaten a Takeda drug.
Ultragenyx Pharmaceuticals’ apazunersen did not meet the goals of its Phase 3 test in Angelman syndrome, a rare genetic neurological disorder with no FDA-approved therapies. The di...
In a first, a subcutaneously administered RNA interference medicine crossed into the brain and plummeted levels of a protein implicated in the disease.
Karyopharm bought an extra month to negotiate with creditors or pursue a deal. Elsewhere, BridgeBio Pharma’s oncology spinout shook up its development plans and a brain-focused gen...
Part of a wave of young biotechs employing AI to accelerate research, the company claimed in regulatory filings that its approach has the potential to “disrupt legacy drug discover...
Electra Therapeutics’ IPO cash will support a potential first-in-class drug whose lead indication is a rare hyperinflammatory disorder with no FDA-approved treatments. Meanwhile, c...
The failure of “del-desiran,” which Novartis got through a $12 billion acquisition, could raise questions about the company’s business development acumen, according to one analyst.
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