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How synthetic DNA and RNA are becoming the discovery engines behind a new generation of precision therapies. The post Rewriting Disease: Oligonucleotides Take Aim at the Untreatabl...
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...
As AstraZeneca appears to close the book on a combo approach following the phase 3 failure of its Ionis-partnered Wainua, several analysts contend it’s too early to count out Alnyl...
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...
New research coming out of the University of Toronto has shown that transfer RNA may offer a promising therapeutic avenue for treating genetic diseases.
Ionis Pharmaceuticals’ zilganersen, brand name Zanvastro, is the first disease-modifying therapy approved for the neurological disorder Alexander disease. While Ionis has experienc...
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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