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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...
This new approach targets disorders including cystic fibrosis, muscular dystrophy, and some cancers. In lab-grown tissue from a cystic fibrosis patient who did not respond to exist...
City Therapeutics’ lead RNA interference therapy could become an alternative to blockbuster-selling anticoagulants. The company’s technology for designing RNAi drugs and delivering...
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.
Chemical engineers found a way to rapidly produce lipid nanoparticles of varying sizes, which could make it easier to develop new vaccines and therapeutics.
RNA Variant Imaging is attracting attention because it addresses a persistent blind spot in cancer diagnostics: many clinically relevant RNA isoforms, splice events, and fusion tra...
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.
Through its entry into Lilly Gateway Labs, Rznomics officials say they plan to accelerate candidate discovery and development across its target gene database bank comprising over 2...
In a first, a subcutaneously administered RNA interference medicine crossed into the brain and plummeted levels of a protein implicated in the disease.
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...
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...
The Silence Therapeutics acquisition gives Merck KGaA a drug ready for a pivotal test in the blood disorder polycythemia vera. Silence’s RNA therapy offers a different approach and...
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 acquisition gives the German company a promising blood disorder medicine that passed a Phase 2 test and could threaten a Takeda drug.
RNA sequencing reveals how naturally occurring mRNA sequences may reduce harmful RNA aggregation, offering insights into transcriptome stability and the design of mRNA therapeutics...
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...
New financing will help Moonwalk Biosciences expand its pipeline of siRNA medicines targeting adipose tissue while building out its proprietary drug discovery platform. The post Mo...
Messenger RNA (mRNA) can be delivered into tissues and cells by encapsulating it into lipid nanoparticles (LNPs), and then injecting the nanoparticles. A target cell engulfs a LNP,...
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.
Adding a viral protein, NoV B2, helped self-amplifying RNA overcome cellular defenses and produce more target protein without diminishing immune stimulation, potentially enabling f...
Short-read RNA sequencing reporting the modifications that alter protein output is starting to make epitranscriptomics indispensable for patient selection in precision medicines. T...
Our guest today is Dr. Nerissa Kreher, Chief Medical Officer of Alltrna. Nerissa brings two decades of rare disease drug development experience. The post Rewriting the rules of gen...
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