[GUEST ACCESS MODE: Data is scrambled or limited to provide examples. Make requests using your API key to unlock full data. Check https://lunarcrush.ai/auth for authentication information.]  mike [@mike98572986](/creator/twitter/mike98572986) on x XXX followers Created: 2025-07-14 05:20:53 UTC Some thoughts on $qure and amt-130, $Clpt and $capr I had been thinking about amt-130 and was looking over some past calls and notes that I thought would be good to discuss. Sometimes, as information comes up it is good to look backwards. I had been thinking about why they are so confident on the upcoming X year data. Q1 2025 conference call “On X questions. So number one, how confident are you on the 3-year follow-up data on the cUHDRS? And what would you consider as a good outcome? And I have a follow-up.” “Sure. As you know, we've been monitoring these patients for a long time. We have not been doing any additional analysis on the data. But it's very clear when we submitted to the FDA included data X years from these patients, but we also have some other patients on the low dose that have been treated even much longer than that. We have no indication at all that we lose any of the efficacy over time. As a result, we believe that the dose-dependent reduction in cUHDRS that we observed at the 2-year data will be maintained when we evaluate the 3-year data. So we feel very confident about our results going forward.” June 2nd status update call Definitely ballsy comments by Uniqure in that cc, Like, why even say that, you could have just said we expect to stay the same, but are going even further out but kind of telling people they expect better numbers “We do expect that the reduction or slower decline will continue past year X and into year X and more.” “And I expect that the X years, this will continue to remain steady and actually become even more salient when we compare to natural history.” Once we got the X year data we have At that time, we will have, as Matt said, XX patients with X years of follow-up, XX patients with X years of follow-up, X patients with X years and actually X patient with X years. But the totality of the data will be received. The primary analysis will be done on the 3-year data because that's where the majority of the patients that is going to generate meaningful understanding of the rate of progression of the disease. Press releases from Uniqure June 16, 2021 1st high dose August 30, 2021- 2nd high dose So basically, management has real time information on the X patients for the X years and X patient for the X years. The bla if everything goes well would mainly be based on the X year cohort data and will be on the high dose, out of those X patients on the X yr, X would be in the high dose. Obviously a small sample size but the X low dose with X years and X for X years are showing them that even on the low dose the efficacy is sticking so it would be highly probable that the high dose would obviously be sticking. By having those real time indicators it is giving them high confidence to make those statements. Recent Conference call “Yes. So the CMC piece, when you really think about cell and gene therapy space, the CMC part of this has always been the tricky area. That's where complete response letters and clinical holds and comparability issues have come out. We believe at UniCure that we are in a very good position in that regard. And there's a number of reasons why. One is, we ourselves have been deeply involved in doing this would be our third approved AAV gene therapy. So just as a company, we have a very deep understanding of CMC and the critical success factors associated with that. But more importantly, the Hemgenex process of manufacturing is highly consistent with what we do for AMT one hundred and thirty. Not only that, but it utilizes largely the same AV serotype. It's produced at the same scale and it is produced at the same facility by the same people that know how to manufacture Hemgenics. So I think we feel that we are in a good position to do what's needed on CMC.” XXXXX engagements  **Related Topics** [$capr](/topic/$capr) [$clpt](/topic/$clpt) [$qure](/topic/$qure) [Post Link](https://x.com/mike98572986/status/1944628144436293841)
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mike @mike98572986 on x XXX followers
Created: 2025-07-14 05:20:53 UTC
Some thoughts on $qure and amt-130, $Clpt and $capr
I had been thinking about amt-130 and was looking over some past calls and notes that I thought would be good to discuss. Sometimes, as information comes up it is good to look backwards.
I had been thinking about why they are so confident on the upcoming X year data.
Q1 2025 conference call
“On X questions. So number one, how confident are you on the 3-year follow-up data on the cUHDRS? And what would you consider as a good outcome? And I have a follow-up.”
“Sure. As you know, we've been monitoring these patients for a long time. We have not been doing any additional analysis on the data. But it's very clear when we submitted to the FDA included data X years from these patients, but we also have some other patients on the low dose that have been treated even much longer than that. We have no indication at all that we lose any of the efficacy over time. As a result, we believe that the dose-dependent reduction in cUHDRS that we observed at the 2-year data will be maintained when we evaluate the 3-year data. So we feel very confident about our results going forward.”
June 2nd status update call
Definitely ballsy comments by Uniqure in that cc, Like, why even say that, you could have just said we expect to stay the same, but are going even further out but kind of telling people they expect better numbers
“We do expect that the reduction or slower decline will continue past year X and into year X and more.”
“And I expect that the X years, this will continue to remain steady and actually become even more salient when we compare to natural history.”
Once we got the X year data we have
At that time, we will have, as Matt said, XX patients with X years of follow-up, XX patients with X years of follow-up, X patients with X years and actually X patient with X years. But the totality of the data will be received. The primary analysis will be done on the 3-year data because that's where the majority of the patients that is going to generate meaningful understanding of the rate of progression of the disease.
Press releases from Uniqure June 16, 2021 1st high dose August 30, 2021- 2nd high dose
So basically, management has real time information on the X patients for the X years and X patient for the X years. The bla if everything goes well would mainly be based on the X year cohort data and will be on the high dose, out of those X patients on the X yr, X would be in the high dose. Obviously a small sample size but the X low dose with X years and X for X years are showing them that even on the low dose the efficacy is sticking so it would be highly probable that the high dose would obviously be sticking. By having those real time indicators it is giving them high confidence to make those statements.
Recent Conference call
“Yes. So the CMC piece, when you really think about cell and gene therapy space, the CMC part of this has always been the tricky area. That's where complete response letters and clinical holds and comparability issues have come out. We believe at UniCure that we are in a very good position in that regard. And there's a number of reasons why. One is, we ourselves have been deeply involved in doing this would be our third approved AAV gene therapy. So just as a company, we have a very deep understanding of CMC and the critical success factors associated with that. But more importantly, the Hemgenex process of manufacturing is highly consistent with what we do for AMT one hundred and thirty. Not only that, but it utilizes largely the same AV serotype. It's produced at the same scale and it is produced at the same facility by the same people that know how to manufacture Hemgenics. So I think we feel that we are in a good position to do what's needed on CMC.”
XXXXX engagements
