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 ...
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 ...
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 ...
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 ...
PALO ALTO, Calif. & SAN DIEGO--(BUSINESS WIRE)--n-Lorem, a nonprofit foundation, and GondolaBio, a clinical-stage biotechnology company founded in 2024 and dedicated to developing novel medicines for ...
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 ...
The initial clinical trial of a novel approach to treating amyotrophic lateral sclerosis (ALS) - blocking production of a mutant protein that causes an inherited form of the progressive ...
When LINE-1 RNA was depleted in human progeroid cells using antisense oligonucleotides the senescent phenotypes were ameliorated, while in the premature aging mouse model this therapy restored tissue ...
Where is a scientist to begin studying a newly cloned gene that has no apparent function or phenotype? A common approach for obtaining functional clues is to inactivate the gene either in vitro or in ...
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