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CRISPR ($CRSP) - Sector Tailwinds, Insider Buys & Massive SI
Top bio hedge funds Avoro, Vestal Point (formerly of Point72), and Affinity are all in $QURE
$QURE – Legit Buyout Bait With 3–5x Potential
Diversified biotech short portfolio - completely tweaked on naked short calls (-20 500$)
QURE, a win for value, a win for patients! Undervalued by +60%
Biotech Pennystocks Growth for 2021 + ($ALRT) rundown
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I was going to buy QURE calls, I was going to, I swear it was in my plan. Fuck I always miss myself.
Honestly full port into leaps of QURE if you want more gains
Did ya see QURE yet? 80% up in one day, fucking crazy
uniQure (QURE) said Wednesday that it intends to submit a Biologics License Application for the accelerated approval of AMT-130 to treat Huntington's disease in Q3. uniQure said the decision was made after a meeting with the US Food and Drug Administration, during which the regulator said that its three-year analysis from a phase 1/2 study would be acceptable as the primary basis of the application. The FDA also seeks to align with the company on the confirmatory study design before the application is submitted, including consideration of a standard care control group rather than a sham procedure. Both CLPT and QURE up big on the news.
Congrats to the 2-3 other people owning QURE on here.
New leadership for one. The new head has already started making changes to streamline experimental drugs. It's why biotech has done so well this week. March wasn't even a disapproval, they just wanted a full double blind study. QURE and HD groups want to use known HD progression as a base and give patients the drug and gather data that way.
When QURE released their three year data last year, the stock went to $71. Four year data starting to come out now. Approval probably gets it well over $100. Also, FDA meetings today for HD groups. Love this.
I also generally avoid it, other than established players. Did well on VRTX a.few years ago. QURE is sort of a special case I read about. It seems pretty solid and the fact that HD patients are actually protesting and lobbying Congress on its approval....seemed worth a small position. CLPT is a platform. I do love a good platform business.
QURE and a few other names running today. The FDA asked another company with a flawed trial to resubmit its data, hinting at more approvals coming. Still think this is a $100+ stock once approvals start coming in. CLPT still hasn't moved much, but is starting to get some traction. Also could 3-4x just an an AMT-130 approval, much less all the other shots they have.
The FDA is wanting a full double blind placebo test. A few problems with that process in this case. 1) AMT-130 is delivered via injection into the brain. So for the placebo group you need to open the skulls of HD patients and do.... nothing. Very few doctors want that risk. 2) HD had no course of treatment. So of you spend 4-5 years doing a double blind test, tons of people go without treatment. On too of that.... 3) AMT-130 has a specific window in the progression of HD in which it can be administered. After that, nothing. So if people that are eligible don't get it now, it's a death sentence. You're literally asking people to die so that we can get 4-5 years of data. So what QURE is asking for (and this isn't a new class of test) is to start administering their drug. Start collecting data using baseline data on existing HD patients in lieu of a placebo group. This allows HD patients to start getting treatment now. If it works, great. If not, those patients have literally lost nothing since there already was no treatment to do anything meaningful for them.
Interesting, wonder why they have been blocking trial expansion. I remember you brought this up when I posted a question about health stocks. I actually started a position in NBIX kinda based on what you said about QURE. Their INGREZZA drug treats tardive dyskinesia by reducing the involuntary movements caused by dopamine dysregulation. Patients with Huntington's Disease are given the drug for the same reasons.
Good news for QURE...a 4th year high dose trial patient posted their experience online. Basically they went from being on disability with minimal function to holding a job. Keep in mind, Huntington's disease has previously had no actual course of care. It was progressive and terminal. For a patient to actually recover and join the workforce.... absolutely unbelievable. And the FDA has been blocking expansion of trials.
QURE/CLPT up big on news the FDA commissioner is getting canned. Good riddance. Link to an update of the QURE saga [here](https://open.spotify.com/episode/2QnVSmT61zN8CKEzKwQNLS?si=rmp8MH0JTbOITGqpU1vMew).
#TLDR --- **Ticker:** QURE **Direction:** Up **Prognosis:** Buy / Hold shares for the $80 bounce back **Catalyst:** FDA drama and the impending firing of Marty Makary **Required Medical Equipment:** A drill for the placebo group (0/10, do not recommend)
QURE earnings/operations update: Advancing FDA interactions on AMT-130 for Huntington’s disease; Type B meeting scheduled for the second quarter of 2026 ~ ~ Progressing AMT-130 toward expected UK regulatory submission; MAA on track for third quarter of 2026 following successful pre-submission meeting with UK MHRA ~ ~ Enrollment in AMT-260 temporal lobe epilepsy program on track; clinical update from first cohort in Phase I/IIa study to be presented at the Epilepsy Foundation Pipeline Conference ~ ~ Presented updated data from AMT-191 Phase I/IIa in Fabry disease study showed sustained increases in α-Gal A Enzyme Activity and stable Lyso-Gb3 levels; subsequently all 11 dosed patients have discontinued enzyme replacement therapy ~ ~ Strong balance sheet with $586.6 million in cash, cash equivalents and current investment securities as of March 31, 2026 and runway into the second half of 2029 ~ Love that the cash on hand takes them to 2029. Probably no need for dilution. Second half of this year is going to be filled with potential catalysts.
QURE news: Submission of a UK Marketing Authorization Application for AMT-130 is expected in the third quarter of 2026 ~ ~ Type B Meeting with U.S. FDA granted in second quarter; expect to discuss potential Phase III design and analysis plan for AMT-130 four-year data ~ ~ uniQure actively pursuing additional ex-US regulatory pathways to support potential registration of AMT-130 in international markets ~
Bio generally scares me as well. There's a good deal written online about both names and for a full rundown I'd highly recommend reading it. If you find Peter Mantas he's done great work, but there are others. QURE has a drug called AMT-130 that it is for Huntington's disease. In clinical trials it actually appeared to have reversed the effects of the disease, which is otherwise fatal and had no cure that even stops it's progression. However, the FDA declined to approve AMT-130 because they want a double blind full placebo test. Two obvious problems there for QURE. Huntington's is kinda rare so running a multi year broad trial is really expensive and time consuming. Also, AMT-130 is injected into the brain via surgery. So you're asking doctors to do a brain surgery on placebo patients as well, which is borderline unethical at best. In the meantime, people with HD have no cure and will likely die waiting for the test. It's a big to do that includes lobbying by members of Congress and FDA resignations. I won't recount the full saga here. So, basically if the FDA clears it, this drug is massive. If not, the company is a zero. At the very least, it's an interesting read. CLPT is somewhat tangential. Remember that brain surgery required to administer AMT-130? It's done using the CLPT surgery platform. There are a few other existing therapies that use it as well, but AMT-130 would have by far the highest TAM. As a result, they have been trading somewhat in lock step. However, while QURE is a single drug play, CLPT is a platform. If the FDA clears AMT-130 there is likely to also be other therapies developed and approved using CLPT technology. There's a big push to change the level of testing needed for rare and extremely fatal diseases. Most Huntington's patients would gladly take an "experimental" therapy because otherwise they have a death sentence. A lot of people want the FDA to let them. While in the short term the two names are linked, I think CLPT has a multi year potential as new therapies get built on its platform. Ray Kurzweil (back in 1999!) did some writing on computer and AI implants in people which really stuck with me. CLPT is a potential winner if that type of thing happens. So I'd probably sell QURE of it surged on FDA approval, CLPT is one I want to hang on to.
In financials I'm big on ABX. Still extremely cheap here. I own a ton. JXN also decently cheap. I own, but it's not as exciting. KFS is technically a financial, but has a lot of other businesses going. In healthcare.... ALHC really has me interested, but I don't own it yet. They have a model of health insurance that actually tries to help it's most sick customers and thus lowers costs long term. CLPT and QURE are extremely cheap if their thesis plays out, but also early stage biotech is risky. So beware. TWST has gotten expensive, but is a great watchlist name. They make synthetic DNA for medical testing.
What bugs me about QURE is some big insider selling right after the initial spike after the results were announced.
I had all kinds of counter plays that I’ve been gradually cashing out but those are running out. Volatility, energy, speculative things that worked out like when QURE dropped to $9
Target for USLM.... it's probably around fair value. Limestone companies tend to sell for 18-20x EBITDA on the private market, so I've read. So it's a little cheap now. I'd really like to see $100 or so for cushion. I don't have insight into MLI. I know they did great during COVID and I think they have some leverage to the copper price.....but not sure beyond that. I have thrown a few bucks at BNED recently, building a position slowly. I think it should be a $20-25 stock, so there's a good deal of upside if they keep playing out their thesis. Airport stocks are getting decently cheap again. I own CAAP and I might add, though I'm not eager at this point. ABX has been selling off for no reason and I might add there too. MIAX is another name I own that I really like the valuation on. Ditto WBI. On the whole I'm practicing patience. I've revised a number of my thesis due to the war. As a result I've become less bullish (and I was actually pretty bullish in February) so there's not much I'm anxious to throw cash at. A few special situations, like QURE that I sized pretty small as basically a moonshot type play. What kinds of names are you interested in? Looks like you're into buy and hold for a long time compounders?
I have a lot of cash because I closed out a few trades recently. NATL got taken out. Closed QURE calls and all my fertilizer calls last week.... So I've been looking for opportunities, but charts are getting ugly on a lot of names. Just practicing patience.
About time. That bitch that got fired from the FDA deserves to be barred from making executive decisions ever again. QURE is legit
man its insane how much QURE went up due to confusing statements from the FDA
Insane reversal on QURE. FDA is a joke right now
QURE after hours bitches stop buying US stocks this country is cooked
Second this. IBRX definitely will become like an OKLO in the near future but it’s still heavily shorted, and the FDA still has to approve data from its trials and approve another sBLA submission after failing from last year, which, in related FDA news: the agency is already getting an insane ton of pushback after an assault on QURE – will be a fascinating few months ahead: https://www.nytimes.com/2026/03/05/health/fda-drugs-rare-diseases-rfk-jr.html
QURE can easily enter the European market. The fact that it's trading at $9 didn't make sense to begin with. US is not the sole TAM. They're also from the Netherlands so I suspect this would be easier than getting through the FDA. Once they've demonstrated that their market entry to Europe was successful, it might be easier to get FDA's buy-in. Or to your point, we can wait out Makary and Prasad, the two ignorant idiots. Nonetheless, it's an easy buy right now.
First comment - QURE short ratio is 5+. It's a tiny halt billion mkt cap. You know what it means 🚀
What are you on. QURE entirely depends on Makary being there or not. > where they are in the testing and approval cycle and timeline? This is irrelevant, the effects are clear. It all depends on whether the methodology of the trial is accepted.
The QURE news is really terrible for people with rare neurological disease. The FDA apparently wants double blind placebo tests for all proceedures because they are afraid of "drilling holes in someone's skull" for no reason. However, to do a double blind test of a surgery....you have to drill holes in people's skulls and literally not put anything in them. Just open a skull to be a placebo patient. Literally makes no sense.
RIP QURE. FDA did you dirty.
CLPT (mods, I think it's still high enough market cap to discuss, please let me know if I'm wrong) was forecast for $50 million in sales for 2026 before QURE hit big. Assuming they now get nothing from QURE, they're trading at 5x 2026 sales. Also, at $50 million they are projected to be cash flow positive as they have self sustaining scale. So 5x sales for a medical platform growing 25%+ with a lot of upside optionality..... Pretty interesting. Companies like this tend to go for 10x sales or more.
QURE has $10 per share in cash
Obviously the PSKY WBD NFLX drama will dominate. But the QURE and RGNX thing is so compelling, with actual life and death consequences. Market is saying RGNX is in the clear and QURE is finished but I’m taking the contrarian play.
He’s a MAGA clown. But anyway, I’m in with premarket buy on QURE. I’ve been reading the monographs and trusting my own education. I think the market may have this wrong, at least on the medicine. I’ve got a nice car or a not nice residence on the line here that the entire market is wrong, and that the MAGA gets it right eventually. Currently RegenXBio is up this morning after being up yesterday too. QURE is tanking, obviously. Reverse translating his quip it seems more applicable to RegenXBio’s submission than Qure’s.
It’s a fascinating story. Last year one of my biggest gains came when QURE jumped 400% on their claimed Huntington’s breakthrough. I recently had a more modest 20% gain after it got sold off to $20. Tempted to enter it again here. It’s not yet clear if Makary was talking about something else. Makary is, of course, another of this crime family administration’s clown troll appointees. But a clue in his comments says the treatment he was disparaging had “no effect”. We can debate the viability and immensity of the uniqure treatment. I certainly had worried that such an extensive and expensive surgery would limit uptake, which was one of the reasons I sold it even before it hit the peak. But nobody can honestly say the treatment had “no effect”. That’s the clue he might be speaking about something else. Some are suggesting he was referencing RegXBio.
From the WSJ: "Today the Food and Drug Administration is announcing new guidance to implement the Plausible Mechanism framework. It will facilitate the development of bespoke therapies that target a specific problem, such as a genetic abnormality, that causes severely debilitating or life-threatening diseases. In these cases, no randomized trial will be required because it’s simply infeasible." QURE ripping.
I think there was someone else better on QURE to rebound? Starting to rebuild the position now that it’s back to the teens.
here is an idea: short-dated QURE calls. super speculative and I have no opinion on the science, but the minutes from the FDA meeting in January should be released any day now. There was definitely some kind of pressure from the Huntingdon's community
HC Wainwright & Co. analyst Patrick R. Trucchio reiterates uniQure (NASDAQ:QURE) with a Buy and maintains $70 price target.
What is your thesis and PT? As you might recall, QURE was one of my big pushes in 2025 and since I sold the rally, one of my biggest gainers. I’ve contemplated trying to repeat that on the idea that their FDA problem gets resolved. But one of the reasons I sold the top was that their treatment, even if promising, requires 12 hours of ultra advanced and expensive surgery and isn’t really a cure so much as the only possible treatment. I’m left wondering what the “right” price is for it should they get the FDA back on board?
QURE with some positive data released this morning. Not a big FDA announcement, but more supporting evidence that they might actually have an effective Huntington's treatment.
Bought some April calls on QURE. It's showing signs of life with a massive push to have the FDA approve it's drug. It would be the only effective treatment for Huntington's disease out there, apparently with huge success. I'm hoping the few days of buying are a signal....
Loading up QURE and FNMA tomorrow
There's a lot of things that look/sound very promising in biotech that for whatever reason (especially with small/micro caps) don't make it to the finish line or - see a situation like QURE recently - suddenly face unexpected roadblocks/detours. Most biotechs are not profitable and good news will often be used as an opportunity to raise $ by selling more shares. It's the riskiest, most volatile sector.
I see you have QURE. Any reason why you don't have CLPT?
How do you feel about QURE in light of the FDA’s change of course with its reasoning for ATRA’s 2nd CRL, that the single arm design is now inadequate.
Personally I have significant long positions in TARS, NBIX and ABBV (although note I’ve been in these over a year and am not adding at these levels). I also hold SRPT and QURE. SRPT I also bought near book value when it was in the 13s QURE is a good example of biotech. Everything looked good and they were aligned with FDA on the trial design. Then last minute FDA reversed course and it’s now down 50% from my cost basis. But I sized accordingly given the historical control uncertainty so the damage isn’t too bad…
Anyone going into $QURE?
I’m up about 30% on my ABVX which is ironically about how much I was up on my QURE before I got rugpulled by the FDA lol. I don’t learn anything because I’ve put a lot more into ABVX, hopefully it pays off
That's very impressive you're still standing after $QURE.
Is QURE good investment now?
Quick in and out of AMCI today. If momentum continues, prob hop in for more green. Also watching KXIN and QURE. Volume isn't high on these two, but watching to see if it picks up
The FDA has nuked numerous studies citing historical or external controls, which are only justified in cases of rare disease - muscular dystrophy, Huntington's (QURE recent debacle), etc. There are plenty of PD patients. For my part, if biomarkers show statistical significance (or even close in this small N), I will look for a strategic point to increase my exposure - just saying I hope the company plays by the book with the FDA.
I had massive gains .. lost most of it with newer buys in Nov Oct. had I just kept my old buys from years ago like ionq/ palantir / iren / ect.. or sold at the height in Oct … had motherfucjin QURE and should have sold it as I know bio pharma is finicky…
Despite what mainstream media says, the clinical data on QURE is weak. I don't think it does anything. But of course the issue is QURE claims they previously had guidance from the FDA to use 3 year open label data vs external control. Vinay Prasad is widely hated among biotech investors for this reason. We don't care that the bar is high, but what we don't like is FDA backtracking on previous guidance. Back to QURE, I don't know what will happen. Obviously there will be a lot of political pressure to approve. But this is not the gamechanger people say it is.
What are your thoughts on FDA withdrawing support for QURE phase 1/2 data for huntingtons disease?
I sold after the QURE FDA debacle. I'm going to wait until after QURE earnings and then reevaluate. I agree this is one of the best biotech stocks out there for a long play but if none of the companies using their tech can get their procedures approved, it's going to be a long haul.
DDOG my god. I went light bc I got blown out this week by QURE SLNO. Blue sky ahead wonder if it’ll move to $200
I really only have two questions: What is QURE? Why did I buy it?
We definitely need clarity on rate cuts. Overall I took a huge gut punch the past week (QURE and SRPT kicked all of biotech in the nuts). Feel good about the rest. Will try to buy some dips
Biotech is so fun. It's an insiders dream. Look at SRPT and QURE. I wish I was one of the patients in phase 3 clinical trials. I'd fake a cough and make these bulls lose billions.
I MISSES FUCKING +50% from QURE rebound within 5h holy fuck I would make 3k FUCKKKKK
How the fuck are the April 2026 QURE calls I got at 10 am up 84 percent right now, should I hold this shit
I need a cure for the QURE beating I’m taking
What is with QURE? I have one $25 put that I have purchased for 50 bucks when it shot up to 55 in September, hopefully it will print today.
FDA is so insanely corrupt. In fact, every single cabinet is led by just self-centered power hungry morons. FDA agreed to a trial design for QURE. They ran the trial for 3 years. It is proven to work in Huntington’s disease which has no treatment available. Dumbass Vinay Prasad comes in and makes it about himself and his ideology again. Just like he did with SRPT. Absolute dysfunction
If not for QURE this would be a pretty good day for me
Losing a bit on QURE. Current FDA is in shambles rn
Hope nobody was in the QURE drop.
Glad I sold QURE a couple weeks ago
What... Just happened to QURE.
check out $QURE and $ABVX, big binaries coming with voucher for approval on QURE (regardless of if it works or not, long story lmao look into FDA dealing with DMD and serepta..) and ABVX will likely get FDA approval and/or acquired this year or next year. I'm in shares and have been for a bit but considering calls.
Oh wow, just took a look at QURE. It's still going up huh. I took profits already (\~35%) but daaaang.
Here's a few tickers: (1) Calls for Jan/April-2026 for SLS - December binary events. $10-$50+ move possible. Why: read their Jan-2025 news on phase 3 GSP:) (2) QURE price target $120, buy leap calls or equity now. (3) CLOV leaps 2027. Price target $20+ (4) PFE - equity or leap calls. (5) INTC leap calls 2026 for $48-55 strike or load the equity. The above suggestion is valid until tomorrow friday evening. Good luck
$QURE still not at all time highs even after curing Huntington's disease...waiting on the second pop.
I've hit top 3 of leaderboard (100+ comments) for like the last 4 weeks straight. It's because during trading hours I shitpost to figure out the news / how everyone feels. It works. I found out about the mango tweet right after he did it, I found about about the crypto short, I found out about random tickers / unrelated things (ie: QURE when it was \~$45), etc, etc. It's basically a source of news for me. It works. I'm regarded, but not thaaaaat regarded.
I’m trying to buy a ton of QURE stock if it dips further
Sold around 3x after tariffs announcement, didn’t want to risk it next week… Are you watching QURE
$QURE $QURE $QURE let is squeeze baby
QURE is gonna cure my poverty lads
Didn’t $QURE have some positive results using gene therapy to cure Huntington’s disease? I think that might be a potential catalyst
we know. But just like any other ticker we buy here so long as people are shilling there will be buyers. See: QURE & VKTX. Both were actually pretty good trades recently. I missed VKTX but saw it being shilled. I got into QURE and made \~30% in a week