Jpath

@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.

@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.

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.

@A_May_MD @viewfromahill9 Good point about developing extended versions, but bi-specific antibodies have potential problems: steric hindrance, more auto0antibody effect, and far less flexibility with dosing each component. This market is big enough for ORKA, SYRE and APGE to be successful.

@viewfromahill9 Yes that’s what they’re doing. $ABBV has the bispec

$ABBV confirming this morning they are taking forward their extended half life IL23. Sorry $ORKA. There’s yet another competitor for you. Oh, and $SYRE…. $ABBV also has an extended half life BISPECIFIC antibody for both IL23 and TL1A. $SYRE has both of those programs individually…their plan is to give 2 separate drugs together. $ABBV already has it as one drug. This makes a *huge* difference in the clinical trial cost and speed, because $SYRE is going to have to run insanely expensive trials with multiple different combo arms (FDA requiring this). $ABBV will breeze right through and with much less expense. See $JNJ’s P2 UC study enrolling 600 patients (!!!!!!) with 1:2:2:2:2:2 randomization (no, I’m not making a joke - 1:2:2:2:2:2). Good luck $SYRE. Better raise a few billion more. Going to be a long haul just to compete against a bunch of generics and branded extended half life bispecs antibodies to boot! “But $SYRE has a4b7 too!!!” Clearly these pharmas aren’t bothered by making these extended half life antibodies themselves. Why pay a premium (maybe $12B??) on top of the current $8B price tag today for an early stage asset? Again, this thing is open label P2a stage……. We got extended half life antibodies at home. Pop the bubble.

@A_May_MD The same could be said for Keytruda and Opdivo and look how that turned out.

$ORKA shows P2a IL23 data that (almost) matched the IL23 knockout study. Stock rallied. $800M raise at almost $6B valuation. Similar story for $SYRE with its *OPEN LABEL* a4b7 data in UC. Both *after* already rallying >100% recently. What exactly did we learn from these readouts that wasn’t fully expected? Seriously, nothing at all. You used to have to take risks to get upside on binaries in biotech. Not in the YTE cabal! Apparently these stocks are somehow perpetually mispriced until they can show us some tiny open label trial that proves their drugs meet bars of the drugs that are already on the market. Oh you’re buying at +300% today becuase a month ago you didn’t know if a4b7 or IL23 worked? Ok, sure 👍🏻

Remember when Bryce Underwood did this? Hope to see much more of it this upcoming season! Go Blue

@RNAiAnalyst I am currently at 10% in cash and working my way up to 20%.

Fellow biotech investors, how much of your #portfolio is in 'cash' right now.

@adamfeuerstein sounds like the current administration.

$SGMO is the clown car inside the burning dumpster inside the runaway train careening off the tracks of biotech.

@robbys_toupee Leave it to a pathologist to be the truth sayer. She'll be back for the next shift.

Shoutout to Joy Kwon for doing what every medical student is afraid to do: leave on time

@MBbiotech @ShamsCharania @DanLamothe Sorry, all the video evidence contradicts what you're saying.

The Timberwolves-Warriors game in Minneapolis has been postponed by the NBA, sources tell ESPN, in wake of a man shot and killed Saturday in a confrontation with federal agents in the city. It's the second shooting there in a week, third this month, involving a federal officer.

@RNAiAnalyst @IHateWabbitsToo Bye bye

@IHateWabbitsToo If $prqr has been giving guidance 'by year-end' knowing they cannot meet guidance, then I'm gone.

$prqr has less than a week to keep their promise of clinical data in 2025. Today is another holiday in the Netherlands, so probably next Monday announcement.

@DavidSacks I hope you go back to South africa

As a response to socialism, Miami will replace NYC as the finance capital and Austin will replace SF as the tech capital.

@semodough Merry Christmas.

MERRY CHRISTMAS to all my Twitter buddies - wishing everyone Peace & Good Health

@nicksortor idiot

Brown University is a shining example as to why nobody gives a sht about your “degree” anymore. The “intellectuals” with a $100K+ piece of paper hanging on their wall are some of the most incompetent people among us.

@mikejon39810746 @rcarian Stupid

@rcarian UM can't fire him because it'd go against their virtue signalling and DEI policies.

Michigan Football is a Billion dollar business. Warde is AD (CEO). Warde’s poor judgment, failures and now COVERUP are now legendary. In any other business he’d be fired already. Fire him and let’s move forward. NOW!

@PaulKersey0kr @rcarian Stupid

@rcarian The DEI runs deep in Ann Arbor…

@KirkGossett @RNAiAnalyst Explain.

@RNAiAnalyst ARWR will dominate WVE

$wve by addressing fat, and visceral fat in particular, they are opening up an entirely new category of metabolic drugs. Co maybe should have more proactively conditioned the Street on focusing on metabolic health, not weight loss per se. Your avg 75 year-old does not really need to lose muscle with weight, but keep his internal organs free from inflammatory fat.

@rcarian The DEI stuff was not necessary.

Here’s my 1st X post about Sherrone Moore on 12/2/25. This is not a spiteful post. I was a scholarship QB from CA and wore the maize & blue. But the institution of Michigan Football is too storied for Moore’s bullshit and he needed to be fired. Warde the AD needs the same treatment for covering this up.

@PimpTruman @rcarian wow

@Jpath5211591 @rcarian About that

Uof M scandal being covered up by DEI AD Warde Manuel and Board of Trustees. Sherrone Moore, 1st time HC and DEI hire, impregnated office employee. All actively covered up by the above. FIRE all of them. Clean it all up, including hiring by skin color & not merit. Source 100%!

@AppieAnanas Thanks for replying, but I went through the transcript on the Quartr app, and there is no mention of manufacturing that I can see.

@Jpath5211591 There is no official confirmation, but ProQR’s comments at Evercore about moving to enzymatic, scalable, stereopure manufacturing align closely with the technology Codexis (and Bachem) provide. So the Codexis angle is plausible but still unconfirmed.

Thread 🧵 | Why AX-0810 is in the clinic and AX-1412 isn’t — and why this matters for $PRQR investors (Credits to @ValueForAll1):

@AppieAnanas Great insight. Where did you find the collaboration with codexis?

9/
To be clear:
ProQR has not yet confirmed that AX-1412’s delay is manufacturing-related.
But the logic is strong, and it aligns with:
• the CEO’s recent comments
• the Codexis collaboration
• the shift toward stereopure enzymatic production