ClearPath @ClearPath100

@martin_casado I don't think much has changed in how SF looks at 2 pm on a Saturday!

Very true, with the caveat that not every sub domain is equally constrained.

Dina Yerlan @dina_yrl

2 days ago

fyi biology is a real world verifiable domain bottlenecked by ground truth data

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When @mickymalka was 24 years old, he met Charlie Munger. He owned just 1 share of Berkshire, but Charlie told him he was the wealthiest person in the room: "Charlie looks at me and says, 'You must be very wealthy to be here! I said, 'Actually, no, I only own one share?' He says, 'You don't get it. You are the wealthiest guy in the room. You have the power of time. All of us here are in our last chapter. You're just starting your time. You will compound for the next 70 years of your life. So never forget the rule I'm about to tell you: you only need to get rich once.' And then he says, 'And now I'm going to eat, and just sat down and ate."

David Senra @davidsenra

2 days ago

.@mickymalka is the founder of @RibbitCapital — the firm behind Revolut, Robinhood, Nubank, Coinbase, and more. He bought his first share of Berkshire Hathaway when he was 13, in Venezuela, with money he borrowed from his grandfather. His grandfather charged him interest. He

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Wow. This is literally e/acc. Von Neumann was the closest thing we had to biological ASI.

Alexander Fitzgerald @AFitzgerald1992

4 days ago

No one is talking about the hardware singularity, though Von Neuman did

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Charts of the Day: AI is changing the way we use Search.

Elon Musk on why we'll find a galaxy full of long-dead civilizations that never left home "I suspect that if we are able to go out there and explore other star systems, there's a good chance we find a whole bunch of long-dead one-planet civilizations that never made it past their home planet" "There are various explanations for the Fermi Paradox. One is these great filters which civilizations don't pass through. One of those great filters is: do you become a multiplanet civilization or not?" "If you don't, it's simply a matter of time before something happens on your planet, either natural or man-made, that causes us to die out. Like the dinosaurs. Where are they now? They didn't have spaceships"

Very cool

Kevin Weil 🇺🇸 @kevinweil

5 days ago

I love this so much. The future is going to be awesome.

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It will happen

Peter H. Diamandis, MD @PeterDiamandis

7 days ago

Never, ever bet against @elonmusk! Here's @NASAAdmin on why the Moon base and Mars will come into existence. "If Elon and SpaceX are betting on this right now, there is no reason to to believe this will not come into existence. I never bet against an extremely well-

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Sufficiently advanced engineering is indistinguishable from art

Arthur MacWaters @ArthurMacwaters

2 weeks ago

Sufficiently advanced engineering is indistinguishable from art

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@DesertDweller93 He’s saying FDA agreed to limit its review to 3 yr data. The real hammer is in the next sentence — $QURE would be “delighted” to share 4 yr data IN THE CONTEXT OF that review. If 4Y data were subpar, surely they’d adhere to “self-contained” 3Y standard, not invite scrutiny. 🚀

$QURE PT hiked to $75 by Wells Fargo.

A reminder of where we stand with a few snippets from yesterday's $QURE earnings call. We are on the verge of a huge breakout this quarter. Hold on tight, it will be a fun ride.

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I have to retweet what Kylie said today during the $QURE earnings call. This is so underappreciated and not priced in. Best management in the business and you can take what they say to the bank.

DesertDweller_ROAR_4_A_CURE_4_HD @DesertDweller93

7 days ago

Kylie O'Keefe closing remarks on $QURE AMT130: "We remain energized by the early clinical signals from both programs and are laying the strategic groundwork in parallel with clinical development. I'll close by saying that we believe the opportunity to potentially deliver the

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This

Dima Zeniuk @DimaZeniuk

a week ago

Insane drone footage of Starship’s first ever intact soft splashdown after a full landing burn 🤯

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@johnkmason Did not expect to be able to add at $38 again. Same type of Christmas come early opportunity as when $QURE dropped to around $8.5 early April last year on the tariff meltdown.

Two CTGTAC briefing documents in 48 hours. Here is the clear mechanistic case that the failure modes don’t transfer to AMT-130. 1. Prespecification. $CAPR’s briefing doc is in fact an argument for $QURE. FDA’s objection there is SAP v3.0 modified post-unblinding, never submitted, never agreed. $QURE’s estimand was locked at a Q2 2025 Type B before the 36-month cut: 3-year change in cUHDRS, high-dose vs propensity-score-weighted Enroll-HD external control, with PS-matched as a prespecified sensitivity. FDA agreed cUHDRS as a registrational intermediate clinical endpoint. There is no post hoc surface to attack. 2. No contribution-of-effect problem. $REPL’s fatal defect is deconvolution: single-arm combo, can’t isolate RP1 from nivo. AMT-130 is one-time monotherapy. Background HD care is symptomatic only (VMAT2 inhibitors, SSRIs); nothing disease-modifying to subtract. The question FDA couldn’t answer for RP1 doesn’t exist here. 3. Internal dose-response plus a concurrent imitation-surgery arm. The AMT-130 Phase I/II randomized 10 patients to imitation surgery. 29 dosed, 12 low / 17 high. High dose consistent across endpoints; low dose variable. Practice effects, rater drift and expectancy do not sort by vector dose. That’s within-study quasi-experimental evidence neither REPL nor CAPR had. 4. Component-level coherence. All four cUHDRS constituents moved the same direction at 36 months: • TFC −0.36 vs −0.88, p=0.033 (met key secondary) • SWRT +0.88 vs −6.98, p=0.0021 • SDMT −0.44 vs −3.73, p=0.057 • TMS 2.01 vs 4.88, p=0.17 Composite: −0.38 vs −1.52, 75% slowing, p=0.003. A measurement-artifact story now has to explain concordance across a clinician-rated functional scale, a motor exam, and two speeded cognitive tasks simultaneously. TFC especially. It isn’t effort-sensitive the way SDMT is. 5. An orthogonal, rater-free biomarker. CSF NfL: post-surgical spike, then below baseline in high dose, −8.2% from baseline at 36m and sustained. Natural history over that window is a ~35-40% rise (Tabrizi). NfL is an immunoassay. No adjudication, no verification dispute, no “assessment methodology limits verification.” This is precisely what REPL lacked when FDA re-derived its scan reads. And it sits on a coherent chain: AAV5-miHTT -> HTT lowering -> reduced neuroaxonal injury. 6. The external control is the best in neurology. $REPL’s CRL cited heterogeneity limiting interpretability. HD is the inverse: monogenic, autosomal dominant, CAG length the dominant determinant of onset and rate. Enroll-HD is prospective, protocol-standardized, global, identical UHDRS instruments. Early manifest HD loses ~1 cUHDRS point/year, tight enough that FDA blessed the endpoint. Prognostic determinism is what validates an external control, and HD has more of it than almost any indication in medicine. 7. It survives a haircut. Halve the point estimate and you still have ~37% slowing against zero approved disease-modifying therapies. Three years of high-dose decline is less than one year of natural history. 8. The regulatory sequencing is inverted. $REPL: staff yes, leadership no. Twice. $QURE already absorbed the “no” (Jan 2026 Type A: FDA recommended randomized sham control), then got reversed in June at the level that overrules. FDA also made confirmatory-design alignment a precondition of filing. The exact fight that killed RP1 gets settled before submission, not after.

@RaminNasibov So the rumor is true then. Sounded far-fetched at first;)

if not you then who

@HighyieldHarry Just the beginning of this trend + there will be more zombie funds, in particular in the smaller end of the market

Another job posting related to commercialization listed today at $QURE Senior Manager, Patient Advocacy. Here are the recent job postings. uniqure.careers.hibob.com/jobs

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