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 ...

The small interfering RNA (siRNA) therapy, designed to reduce triglyceride levels by suppressing production of apolipoprotein ...

Researchers have devised a molecular structural modification that boosts the efficacy of antisense oligonucleotide-based drugs by replacing the RNA strand of a heteroduplex oligonucleotide with DNA.

Antisense oligonucleotide (ASO) therapy has the potential to ameliorate many neurodegenerative diseases at the genetic level to suppress the production of harmful proteins or non-coding RNAs.

Ionis is planning a supplemental submission by the end of the year to expand Tryngolza into severe hypertriglyceridemia. If ...

Akcea Therapeutics and Ionis Pharmaceuticals announced that a phase 2 study of AKCEA-ANGPTL3-L Rx for the treatment of patients with hypertriglyceridemia, type 2 diabetes and nonalcoholic fatty liver ...

genetic code Antisense oligonucleotide drug HTTRx reduced the concentration of mutant huntingtin protein in the cerebrospinal fluid of persons with Huntington disease. Primary end point was the safety ...

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...

A Penn Medicine patient with a genetic form of childhood blindness gained vision, which lasted more than a year, after receiving a single injection of an experimental RNA therapy into the eye, ...

Two complementary studies led by researchers from the Yong Loo Lin School of Medicine, National University of Singapore (NUS ...