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The Big4Bio PodcastThe Big4Bio Podcast shares podcasts that give ins Author: Big4Bio
The Big4Bio Podcast shares podcasts that give insight and information about/from biotech and life science leaders and organizations within the Big4Bio regions. Check out our Playlists to hear podcasts by region. Language: en Genres: Science Contact email: Get it Feed URL: Get it iTunes ID: Get it |
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Every Moment Matters: Developing an N-of-1 ASO for a Child with an Ultra-Rare Disease
Wednesday, 25 February, 2026
Advances in sequencing, the emergence of antisense oligonucleotide therapies, and the advent of induced pluripotent stem cell technology are among the combination of factors that have made bespoke medicines for individual patients scientifically feasible. In the case of ultra-rare, genetic diseases, a child may face a progressive, life-threatening condition with no available treatments. As we continue our series on discovery with Charles River Laboratories, we take a look at how the organization partnered with global collaborators and a patient’s family to design, screen, and advance a highly selective antisense oligonucleotide therapy targeting a pathogenic allele underlying a child’s ultra-rare epileptic encephalopathy. We spoke to Charles River Lab’s Associate Research Leader Blanca Torroba and Group Leader of Neuropharmacology Johanna Uhari Väänänen about how they moved from concept to a candidate for a custom ASO therapy, steps they took to accelerate the process, and the challenges of advancing N of 1 medicines to an IND.nbRb
