Reply Guy Cap

Ask me about the chip on my shoulder

[DesertDweller_ROAR_4_A_CURE_4_HD @DesertDweller93]

While the data from a year ago on $QURE AMT260 set a very hi bar to clear, today's news is very good. Remember these patients are refractory to any current SoC. FIFTY percent of the patients achieved reductions of 79-100% in seizure frequency just months after treatment in the low dose.

No immunosuppressants needed in this treatment but could be on the table if needed in hi dose imo. Management has stated it could be done on an outpatient procedure basis, go into surgery center in the morning and go home same day with potentially life changing effects.

[Reply Guy Cap @ReplyGuy_Cap]

@defi_kay_ @ContrarianCurse 2nded on the Steelcase Amia. Incredible chair for the Money.

[Lizzy Lawrence @LizzyLaw_]

NEW: The FDA approved Sanofi's type 1 diabetes drug that was selected for the Commissioner's National Priority Review program. Staff and former CDER director Tracy Beth Høeg had previously disagreed on the approval.
https://t.co/p45oozU2OE

[mike @mike98572986]

$qure $clpt

Another decision that FDA staff didn’t agree with leadership, it’s really remarkable how Makary lied saying Prasad, Hoeg and himself didn’t overrule anyone and these decisions were unanimous between staff and leadership. If that were true why has at least 7 CRL’s or programs derailed been overturned and 7 more are currently in pending discussions. This has happened in a very small timeframe.

No wonder career staff disliked these people so much. People keep saying these people were fired for political reasons or because big pharma, no they were fired because the only decision in the room that mattered were people that clearly weren’t qualified to be making these decisions. Thank goodness for the American people that these corrupt officials were fired and sent packing. The reign of these 3 and that Schwarma person will go down as an asterisk in the history books as a very dark time for American innovation. Fortunately for us, Prasad is back where he belongs, talking about Fiber with very low views on YouTube. Talking about 💩 is where Prasad belongs in my book.

“STAT previously reported that Sanofi asked to pull its drug out of the program after former top drug regulator Tracy Beth Høeg disagreed with a staff decision to approve the drug. It's rare for a center director, and particularly a political appointee like Høeg, to get involved in individual scientific reviews.”

[uniQure @uniQure_NV]

Thank you @GinaKolata @nytimes for this profile of Nancy Wexler and the Huntington’s disease community. One line that stays with us: there are currently no available treatments for @huntingtonsdisease. We’re hard at work to change that. https://t.co/XK0Dmf1gXC

[The Nicotine Pouch Epoch @LaDivaNicotiana]

$QURE $CLPT THE TREPIDATION IS PALPABLE...

[The Inflection Economy @inflectionecon]

$TWST nothing like it in public markets. The game changing impact of AI in biotech/genomics is still in its infancy. Could get much more attention in 2H…

[mike @mike98572986]

$qure $clpt

Uhhh guys, FDA just gave the playbook, they just told us directly what they think about natural history for HD here.. This is the FDA speaking here. This was June 5, 2026, Lauren Holder also spoke. 2 hd people spoke. Why do you think the FDA specifically requested Lauren to be there and speak?? -) Not sure what else the new FDA can say, they basically just specifically called out HD and natural history..

Look who was one of the main hosts and also spoke

Teresa Buracchio- the same Teresa that said

“FDA official confirms plausible mechanism principles not exclusive to bespoke gene therapies”

Michelle Campbell FDA 35.48 to 36.38 woah….
Look what she says about HD..

FDA again 1:36 - 1:39- big time big time, FDA calls out HD and natural history.

“Natural history is going to be paramount and fundamental to how we really think about the understanding of the disease!”

“Whether it’s ALS, Huntington’s, or a neurodegenerative disease, The truth is the same, time matters.” @laurencurehd spoke from 1:28

https://t.co/hhBQTI38Ag

https://t.co/3a79ub7q2F

@peter_mantas @Tim_Corr @DesertDweller93 @Prof_Dollar @biggercapital @Christina4HD

[Hammer @that_grey_beard]

Mike obviously understands this space. I don't, but I read all the press releases and filings and checked all this language when I got involved over the past few months. If I was smart, this is how I would state my thesis. And add 'return to whence we came' (September 2025 PR)

[Rain Consulting Group @RainConsulting]

The FDA’s evidentiary framework was built for large trials and common diseases. ALS is neither.

Rare disease patients deserve advisory committees that understand urgency, totality of evidence, and the cost of delay. The past NurOwn AdComm understood none of this.

When a disease is 100% fatal, “wait for more data” is not a neutral decision. It’s a decision with consequences, costs lives, and is completely unacceptable. @FDA_KyleD @aVoice4ALS @iamalsorg @SecKennedy @houmanhemmati

[twotreesinthecenter @twotreesthere]

"What changed?" @US_FDA between June 2025 & March 2026 re $QURE's HD gene therapy on the accelerated approval path.
For me, the simplest answer tends to be the right one.

Please @FDA_KyleD @FDACBER Huntington's disease patients deserve so much better. https://t.co/zn7g7DLo6n

[Houman David Hemmati, MD, PhD @houmanhemmati]

🧬 Please read this article 👇 by @ZacharyBrennan at @endpts which outlines how @FDA not following its own policies is risking the demise of @gracescience - a biotech founded by a brilliant Nobel Prize winner that treats an ULTRA-rare pediatric genetic disease with gene therapy.

Despite having manufactured & tested the drug, and having enough to treat most of the kids with it in the USA, FDA is forcing them to make more drug at a cost of tens of millions and potentially years, just to check a box.

Why?

It’s time to be reasonable & common sense. No one is being helped by stonewalling a potential treatment for a devastating genetic disease.

[BioSpace @biospace]

As the FDA tries to clarify its intent for former FDA Commissioner Marty Makary’s plausible mechanism framework for bespoke therapies, experts emphasize expanding its scope to encompass rare diseases that affect more than just one or a few individuals.

https://t.co/w51bKcKobR

[Reply Guy Cap @ReplyGuy_Cap]

@peter_mantas @JasonGreenwal12

[tailrisked @tailrisked]

https://t.co/Q7Sk4a28Uw

[BioSpace @biospace]

The FDA must provide consistent and predictable regulatory frameworks if the U.S. is to maintain its leadership in gene therapy, one of the most consequential therapeutic fields of our generation.

https://t.co/ibj316GVmq

[Jesse Livermore @Jesse_Livermore]

Boom.

[Reply Guy Cap @ReplyGuy_Cap]

Both @Christina4HD and @KarlMiran crushed it.

[mike @mike98572986]

$qure $clpt

Put @Christina4HD in the hall of fame, this speech will go down in History as one of the greatest speeches of all time, I got chills listening to it. Amazing job representing the HD community, the whole community should be very proud of her.

[Peter Mantas @peter_mantas]

For the love of god it’s staring you right in the face

[Reply Guy Cap @ReplyGuy_Cap]

@Banana_Oncology Good analogy but pretty sure Sheep is Thor in those scenario.