
Oligonucleotide
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Search complete. 3 mentions across 2 episodes found for "Oligonucleotide".
Sep 24, 2026
Enabling Patient Foundations to Build the Next Generation of Genetic Therapies
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16:16Stephen RingleGUEST
We have a model that we call Togo.
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16:19Stephen RingleGUEST
That model does one really simple thing that's super important to any family that's gotten the diagnosis, and that is taking in that genetic information and looking across active approved therapies, clinical trials, off-label opportunities, as well as novel genetic medicines like we're talking about here, ASOs, gene editing, gene therapy, looking at all those different options comprehensively based on the patient's own data and background and recommending to them that these are the ones that might make sense.
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16:49Stephen RingleGUEST
And that process usually today either does not happen at all, and a, a patient is left to go Google or ChatGPT that type of information, um, which often has lots of, you know, errors or gaps, um, or, um, it, you know, it's happening very, very slowly.
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17:04Stephen RingleGUEST
You know, a team of MD PhDs is reviewing by hand a patient's eligibility for a novel genetic therapy, and that can take up to fifty hours o-of their time, which just simply does not scale.
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20:26Stephen RingleGUEST
Um, the same way that you would hire one general contractor if you wanted to build a house, instead of trying to go find the best plumber, the best electrician, so on and so forth.
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20:35Stephen RingleGUEST
We take that on, we manage all that, and we're the single point of truth for the patient.
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20:39Stephen RingleGUEST
Right now, we've got a double-digit number of genetic medicine programs across neurology and retinal disease, and we are working in both ASOs as well as gene therapies.
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20:50Stephen RingleGUEST
Um, all of our programs are preclinical right now, and that's 'cause we just started opening our gates to patients at the start of this year, twenty twenty-six.
Pelacarsen Failed To Meet Its Primary Endpoint, But The Science Keeps Going
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11:31Michael KorenHOST
Let me just ask you one other question.
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11:32Michael KorenHOST
ASOs work more in the nucleus of the cell versus the siRNAs that work in the cytoplasm.
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11:38Michael KorenHOST
Do you think that matters at all?
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11:40Seth BaumGUEST
It's a great point.