Antisense Oligonucleotides
Ionis Heads to FDA as Antisense Therapy Aces Pivotal Study in Rare Neurological Disease
Ionis Pharmaceuticals; zilganersen; antisense oligonucleotide; Alexander disease; pivotal Phase I–III study; FDA Fast Track designation; rare neurological disease; expanded access program; walk speed improvement; Orphan Drug designation
Biogen Pledges $2 Billion Manufacturing Investment in North Carolina
Biogen; manufacturing investment; North Carolina; Research Triangle Park; biotechnology; antisense oligonucleotides; gene therapy; automation; AI; expansion
Ultragenyx’s GTX-102 Shows Promising Results in Mid-Stage Angelman Syndrome Study, Pivotal Study on the Horizon
Ultragenyx, GTX-102, Angelman Syndrome, Mid-Stage Study, Pivotal Study, Antisense Oligonucleotide, Cognitive Improvement, Behavioral Improvement, Sleep Improvement, Gross Motor Improvement
Groundbreaking Data Revealed for Genetic Epilepsy Drug STK-001 by Stoke Therapeutics, Exhilarating Investors
Stoke Therapeutics, STK-001, Genetic Epilepsy Drug, Dravet Syndrome, Antisense Oligonucleotide, Seizure Reduction, Positive Clinical Trial Data, Investor Enthusiasm