Webinars

Date: May 23, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: Antisense approaches for the treatment of Duchenne muscular dystrophy and recent advances to address DMD brain comorbidities Description: This presentation will cover the development of ASO-based approaches for the treatment of Duchenne ...

Date: May 16, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: Validation lab: allowing standardized in vitro and in vivo experiments for candidate treatments for Duchenne muscular dystrophy Description: The Validation Lab was established in 2015 with support from the Duchenne Parent Project ...

Date: May 2, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: In-silico siRNA Off-Target Predictions: What Should We Be Looking For? Description: Assessing sequence-dependent off-target risks is a critical step during siRNA development to ensure the safety and specificity of therapeutic candidate molecules. ...

Time: 8am PDT / 11am EDT / 5pm CEST Date: April 25, 2024 Title: In vivo Optical Control of Gene-Silencing in Japanese Medaka Fish Embryos Description: The siRNA oligonucleotide duplex inherent to biology, functions by binding and cleaving mRNA, a process known as the RNA interference ...

Time: 8am PDT / 11am EDT / 4pm CET Date: March 21, 2024 Title: RNA Targeting Technology – Chemistry and Delivery Description: Chemistry coupled with pharmacology, both preclinical and clinical, forms the basis for the pipeline we manage at Ionis today. This presentation will focus on ...

Time: 8AM PST | 11AM EST | 5PM CET Date: February 22, 2024 Title: Advancing Therapeutic Approaches for Erythropoietic Protoporphyria Description: Currently, no curative treatment is available for the rare disease erythropoietic protoporphyria (EPP). The genetic background of EPP is well understood; a loss-of-function mutation on ...