Evan

@laurencurehd @DesertDweller93 @DrMakaryFDA @VPrasadMDMPH @johnarnold @uniQure_NV Here is to hoping order is restored and prior guidance is honored….

@DesertDweller93 @DrMakaryFDA @VPrasadMDMPH @johnarnold @uniQure_NV I can't tell you how much RELIEF I feel!

WOOHOO, looks like the last member of the @DrMakaryFDA @VPrasadMDMPH @johnarnold swamp is leaving also. Hopefully the stench from their tenure dissipates quickly and @uniQure_NV $QURE can move forward with AMT130 BLA filing. Let's hope their replacements bring integrity back to a once world respected institution. Congress and media, keep up the pressure and don't let them escape investigations. @SenRonJohnson @SenRickScott @RepAuchincloss @houmanhemmati @adamfeuerstein @armstrongdrew @l_e_whyte @temple_west @laurencurehd @JRenz0418 @rachelreising96 @BeckyQuick @davidfaber @AllysiaFinley @MariaBartiromo @bradloncar @docrodwong @LuckyPenguin10 @POTUS @SenateAging @SenBillCassidy @SenRandPaul @WhiteHouse @SusieWiles @StevenCheung47 @StevenCheung @Scavino47 @PressSec @SecKennedy @MariaBartiromo @RickSantorum @FoxNews @bariweiss @60Minutes @CNBC @WSJopinion @WSJ @statnews @BillAckman @Loftus @MartinShkreli

@mike98572986 It’s either the biggest troll in the world with an obvious contradiction, they are completely aloof, or they are going to fix this under the new Cher head and claim victory for the mess they created?

$qure $clpt Plausible mechanism framework fda meeting Tracy Beth Høeg- “This is for any population.” This applies to common diseases according to the FDA. Quote during the discussion from a different member that I believe I heard, it’s still going so will double check that quote. “When dealing with a slow moving disease, natural history is warranted.” Just listened to the FDA meeting on plausible mechanism. They talked quite a bit about natural history and external controls. When you listen to this, it’s hard to not think of Amt 130 on what they are asking for. What is funny is that Prasad created a program that completely goes against his beliefs and lines up perfectly with amt 130. One day we might have to thank Prasad because if you get Uniqure in a meeting with the FDA once this goes live the FDA is going to have a difficult time disputing line by line the arguments that this document focuses on. Uniqure can go line by line and put their program against this document and argue their case very easily. He basically makes the case for amt 130 with his document. They did specifically call out - is the targeted area known to be a cause of the disease? With Amt 130 that question is pretty easy with well documented data on what they are targeting. Still going on, will post if I was wrong on that quote if anything else interesting happens.

@mike98572986 Thoughts on any potential meaning?

$qure $clpt New document put out by Uniqure cdn.craft.cloud/fe68e1a2-b284-…

@mike98572986 Seems just like a large amount of recent positive data points continuing to build what seems to be a super large moat. Will they monitize it and execute??

@mike98572986 You should run clpt’s social media account 😂

$clpt Neurona Here we go We are happy to share that Jessica J. Roland, PhD, Senior Medical Science Liaison at Neurona, will be presenting a poster “Phase 3 EPIC (Epilepsy Cell Therapy) Clinical Trial Design” on Wednesday, April 22 from 11:45-12:45pm CD, and Peter C. Warnke, MD, Professor in the Department of Neurological Surgery, The University of Chicago Neuroscience Institute, will present “Clinical Update: NRTX-1001 GABAergic Interneuron Cell Therapy for Bilateral Drug-Resistant Focal Epilepsy” on Wednesday, April 22 from 1:00-3:00 PMD CDT at the American Academy of Neurology 2026 Annual Meeting.

@mike98572986 @HarrieScarlet Has the panel with Qure on it gone yet?

@HarrieScarlet Hard to say tbh

$qure $clpt There it is

@mike98572986 Too bad it’s CDER and not CBER?

@mike98572986 🤯we live in a simulation

$qure $clpt What in the world is going on here… Uniqure and a senior reviewer at the fda on the same panel? Alternatives to Placebo Controls and RCTs in Rare Disease Trials Placebo-controlled trials are often impractical or ethically challenging in rare disease settings. This session will explore approaches that use alternative comparators, such as within-patient comparisons or external data sources, and discuss how these strategies can be used to generate interpretable evidence while addressing feasibility, ethical considerations, and regulatory expectations. Moderator: Amy Comstock Rick, JD, Director of Strategic Coalitions, FDA Rare Disease Innovation Hub Panelists: •Oxana V. Crysler, MD, MHS, Clinical Associate Professor of Internal Medicine, University of Michigan, Rogel Cancer Center, a NORD Rare Disease Center of Excellence  •Wonyul Lee, PhD, Senior Statistical Reviewer, CDER, FDA •David Margolin, MD, PhD, Vice President of Clinical Development, uniQure, Inc. •Susana Zaph, PhD, Senior Director, Head of Quantitative Systems Pharmacology -US, Sanof

@mike98572986 Seems like she is dailing up her rhetoric vs past comments?

$qure $clpt Sarah Tabrizi spoke today at The 2026 Samuel Gee lecture “The main challenge we have is the delivery to the brain” Going to put the big stuff first, regarding amt 130 she said “Tfc is resistant to placebo, it is shown to be resistant to placebo. We haven’t seen this in any study. 2 people went back to work this is unheard of. They were both medically retired. I repeat 2, this is my commentary. I think she said this isn’t randomness but was writing down this so… By 3 years placebo definitely should have washed out. Motor function and cognitive all in the right direction in the trial. Psychiatric symptoms didn’t really change, they do have medication for that. A neurologist with 25 years experience that asked a question said he was shocked to see this data when it came out. Other trial experience- Aso trial intrathecal, was good for the cortex but didn’t get to the deep parts. 13 neurologists before the trial told her straight up it will never get to the stratum Generation hd1- high dose to try to get into the brain, caused issues. Generation hd1 - lower dose will read out this summer Amt-130 We target exon 1 importantly Brad Elder came up with the delivery strategy. The surgeries were fantastic. “FDA insisted on a sham at 1 year” 1 year is no use for a sham In Europe, they don’t think the sham is ethical. Enroll hd was agreed because it is current and updated. A long placebo is not possible, it would be at least 3 years. PSM is an approved method for gene therapies, referenced UK agreed to this a year ago also. Many gene therapies and drugs have been approved with PSM and external control studies. For the PSM, characteristics were exactly the same. NFL - this is molecular evidence which lines up with what we are seeing. We now know hd can be modified. It can be significantly modified. We know what we need to do. @peter_mantas @DesertDweller93 @Prof_Dollar @biggercapital