People around the globe suffer from a myriad of rare diseases, many of which have no cure or limited treatment options. Many patients have been living without hope of a significant improvement in ...

ReCode Therapeutics recently announced a research collaboration with Praxis Precision Medicines aimed at developing lipid nanoparticle formulations to improve delivery of antisense oligonucleotides to ...

“Antisense” drugs that aim to shut down disease-related genes have long been out of favor, and these days live in the shadow of a hotter and newer technology — RNA interference — that does much the ...

When the drug fomivirsen was approved by the FDA in 1998 for the treatment of cytomegalovirus retinitis in patients with HIV/AIDs, it was hailed as a milestone in drug discovery because it was the ...

- Positive Phase 2 study results demonstrate significant efficacy of donidalorsen (formerly IONIS-PKK-LRx) in the reduction of hereditary angioedema attacks - Based on the results of the Phase 2 study ...

At first, researchers thought that antisense drugs would prove so specific there would be few side effects. But toxicity-related problems did arise in initial animal studies of antisense drugs in the ...

Isis Pharmaceuticals (www.isispharm.com) has developed an antisense drug candidate that continues to show potential in treating patients with cardiovascular diseases by reducing cholesterol, LDL-C, as ...

Economic Moat Ionis has proprietary antisense oligonucleotide technology that has led to three approved RNA-based drugs and a steadily growing pipeline, which together support the firm's intangible ...

SAN DIEGO, CA — Two novel antisense oligonucleotide therapies in development significantly reduced levels of lipoprotein(a) in individuals with elevated Lp(a) in two clinical trials, according to a ...

So far, 2013 has been a good year for antisense technology. In fact, it’s been great. In January the FDA OK’d Isis Pharmaceuticals’ Kynamro™ (mipomersen sodium), the only currently marketed antisense ...

Unmodified starches present a problem to the frozen food industry as they tend to result in poor texture upon cycles of freeze-thawing. In March Nature Biotechnology, Stephen Jobling and colleagues ...