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The FDA approved bepirovirsen as the first antisense oligonucleotide for chronic hepatitis B virus infection, alongside existing antiviral therapy for eligible adults without cirrh...
Cancer researchers are turning a deceptively simple piece of DNA into a molecular “decoy” that could disrupt the genetic commands driving tumors. The strategy, reviewed in a new ar...
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...
A preclinical study showed how chemically enhanced suppressor tRNAs combined with a lung-targeted delivery system can restore production of a critical protein in models of cystic f...
In a first, a subcutaneously administered RNA interference medicine crossed into the brain and plummeted levels of a protein implicated in the disease.
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 PROPEL program aims to control gene expression through RNA structure, building logic-gated RNA therapies that switch on only where and when intended, without altering the genom...
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 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.
Ionis Pharmaceuticals’ zilganersen, brand name Zanvastro, is the first disease-modifying therapy approved for the neurological disorder Alexander disease. While Ionis has experienc...
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.
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