Web26 jul. 2024 · This commitment extends across multiple modalities, including antisense oligonucleotides, as with BIIB080,” said Alfred Sandrock, Jr., M.D., Ph.D., Head of Research and Development at Biogen. “Biogen is encouraged by the results of this trial, and we look forward to our continued research in future clinical studies with this promising … Web6 jun. 2024 · The improved performance will be driven by advances made by the Ionis core antisense program. Today, we have medicines that can be administered …
Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema ...
Web10 jul. 2024 · Angelman syndrome (AS) is a rare neurodevelopmental disorder caused by loss of function of the maternally inherited UBE3A allele. In neurons, the paternal allele of UBE3A is silenced in cis by the long noncoding RNA, UBE3A-ATS . Unsilencing paternal UBE3A by reducing UBE3A-ATS is a promising therapeutic approach for the treatment of … Web2 dec. 2024 · Antisense oligonucleotides (ASOs) regulate gene expression by binding to complementary target RNA, and ASOs can be designed to take advantage of a growing array of post RNA binding molecular mechanisms. Intracellular trafficking of ASOs influences their efficacy. We have identified a number of membrane-less structures in the … lithograph framing
Ionis
WebThis is the approach we have taken at Ionis. Obviously, to create antisense technology, we have had to address a wide array of strategic questions, for example, the medicinal … Web10 nov. 2024 · About Ionis Pharmaceuticals, Inc. As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug … Web10 okt. 2024 · - Ionis scientists, including CEO Stanley Crooke, M.D., Ph.D., recognized with prestigious Paper of the Year Award for their work on advancing core antisense research Published Oct 10, 2024 7:05AM EDT lithograph frame