The ability to make synthetic DNA and RNA by chemical or enzymatic means has revolutionized genetic research and drug discovery. Antisense technology employs short, chemically synthesized ...

Diffuse intrinsic pontine glioma (DIPG) is a lethal pediatric brain cancer that often kills within a year of diagnosis. DIPG occurs in an area of the brainstem called the pons, which controls many of ...

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 ...

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 ...

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 ...

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 ...

Ionis is a leader in RNA-based therapies, and its spinal muscular atrophy drug Spinraza, marketed by partner Biogen, is the first RNA-based therapy to achieve blockbuster status. The firm's antisense ...