Appie Ananas

445 posts

Appie Ananas

Appie Ananas

@AppieAnanas

just my own opinions

Katılım Aralık 2024
10 Takip Edilen110 Takipçiler
Appie Ananas
Appie Ananas@AppieAnanas·
@Jpath5211591 @RNAiAnalyst That’s possible. But building a gen2 candidate before first human data can also reflect platform maturation. If AX-0810 shows: • target engagement • biomarker effects • acceptable safety Axiomer may still validate even if potency improves later.
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Jpath
Jpath@Jpath5211591·
@AppieAnanas @RNAiAnalyst You are underestimating the importance of editing efficiency; they are pursuing a second generation version to improve efficiency which may be a red flag for the upcoming read out.
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Dirk Haussecker
Dirk Haussecker@RNAiAnalyst·
Savage! Just as $WVE and $BEAM have made their celebratory laps, in comes $krro with a real genome editing alternative for AATD: 90% RNA editing (in mice) possibly working out as something similar in the clinic with monthly dosing. Capitalism working for patients. Good.
Korro Bio@KorroBio

We’ve selected KRRO-111 as a development candidate for the potential treatment of #AATD. In preclinical studies, KRRO-111 achieved >90% RNA editing of SERPINA1 transcripts, translating to ~90% repaired functional AAT protein. Learn more: ir.korrobio.com/news-releases/… $KRRO

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Steve Martin
Steve Martin@SteveMartihpon·
@AppieAnanas 2/Many are trying to compare $PRQR to $WVE $BEAM $KRRO and I believe they’re doing a disservice by misleading investors. I’d love to see an”X debate” with you and some (mainly one)the ProQR bashers. Today’s update does that and clearly separates your understanding from the others
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Appie Ananas
Appie Ananas@AppieAnanas·
1/ Another important scientific presentation from $PRQR just dropped, this time focused on PNPLA3 and MASH/MASLD. And strategically, this may be one of the clearest examples yet of what ProQR is trying to build with Axiomer.
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Appie Ananas
Appie Ananas@AppieAnanas·
ProQR seems increasingly focused on proving that RNA editing can become more than a rare disease tool. The long-term vision appears to be: programmable metabolic and functional biology through RNA engineering. $PRQR
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Appie Ananas
Appie Ananas@AppieAnanas·
14/ It increasingly feels like a coordinated effort to prepare the market for broader platform validation ahead of AX-0810 human data expected this quarter.
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Appie Ananas
Appie Ananas@AppieAnanas·
@RNAiAnalyst $KRRO potency looks impressive. But cross-comparisons between AATD and bile acid modulation may not be entirely straightforward. $PRQR core bet appears to be that limited editing can still drive meaningful pathway modulation. The upcoming human AX-0810 data will be the real test.
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Dirk Haussecker
Dirk Haussecker@RNAiAnalyst·
Back of envelope: $KRRO 111 achieves 90% instead of 50% editing in mice at 1/3 of the dose that $WVE 006 did. At least 3 years behind, but a very meaningful step-up for #RNAediting in AATD. $PRQR
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Appie Ananas
Appie Ananas@AppieAnanas·
@Kerny13402791 Ended Q1 2026 with € 81.1 million cash and cash equivalents, supporting runway into mid-2027 ProQR expects to deliver multiple clinical data readouts across its pipeline within its current cash runway, which extends into mid-2027. proqr.com/press-releases…
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Kerny
Kerny@Kerny13402791·
@AppieAnanas $prqr future may be bright but soon they have to do an ugly raise to fund all the science...
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Appie Ananas
Appie Ananas@AppieAnanas·
1/ $PRQR just released two new scientific presentations (TIDES USA + ASGCT) and together they paint a bigger picture for the Axiomer RNA editing platform
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Steve Martin
Steve Martin@SteveMartihpon·
@AppieAnanas Great summary. I appreciate your hard work & insight, Thanks Extremely BULLISH on $PRQR
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