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 ...

Discover how a newly developed platform technology from The University of North Carolina at Chapel Hill can enhance RNAi ...

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 ...

BOSTON--(BUSINESS WIRE)--Alloy Therapeutics Inc. (“Alloy”), a biotechnology ecosystem company dedicated to democratizing access to cutting edge drug discovery technologies, announced today a target ...

Ionis is a promising biotech with a strong late-stage pipeline and lucrative Big Pharma partnerships, despite current weak commercial revenues and heavy losses. The company has five FDA-approved drugs ...

Assessing the distribution of a medication in the brain is critical for the treatment of a vast range of neurological disorders, especially conditions such as Parkinson's and Alzheimer's diseases. To ...

WASHINGTON, Jan. 23, 2024 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug ...

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 ...

Patients treated with high-dose BIIB080 saw positive trends on the global Clinical Dementia Rating Sum of Boxes, Mini-Mental State Exam cognitive scales and Functional Activities Questionnaire at week ...

“Together, these findings support a beneficial role for CD47 knockdown in alleviating age-associated metabolic dysfunction through coordinated effects across multiple organs” “Together, these findings ...