Reply Guy Cap

CSL's #hemophilia B gene therapy, licensed from $qure, is slowly becoming a success story, no matter the temporary manufacturing setback: $57M in H2 2025 sales following a slow start in 2023/4, and 51 out of 54 patients prophy free after 5 years! $sgmo

Oh, *now* the FDA has decided to have a public hearing on the national priority review voucher program. After four drugs have completed the process. A year after the program was announced. This must be that "radical transparency" that @DrMakaryFDA keeps yapping about.

30,000 Americans are symptomatic with Huntington's disease right now. Another 200,000 carry the gene. There is no approved treatment that slows progression. None. AMT-130 showed 75% slowing at 36 months. The 48-month data drops this summer. If the numbers hold, the FDA will have to explain why it spent months calling this therapy "failed" instead of reviewing it. $QURE #HuntingtonsDisease #AMT130

For those who don’t have access to the FT. Below is the article on Arnold Ventures from December directly linking Vinay and Katherine and specifically calling out uniqure $qure @DesertDweller93 @biggercapital @peter_mantas @MartinShkreli @canoebrookbl @adamfeuerstein

Funny little fact pattern. 1.Szarama wrote the Arnold Ventures letter arguing against external controls in December 2023; 2.She was appointed CBER Deputy Director October 8, 2025; 3.FDA rug pulled on AMT-130 22 days later; 4.No public recusal record exists. @adamfeuerstein @LizzyLaw_ @SenRonJohnson @SenBillCassidy @RepJasonSmith @RepAuchincloss @SenateAging

This is something that isn't discussed enough about the @uniQure_NV $QURE AMT130 trial when discussing a placebo arm. After only 12 months, 6 out of 10 WERE NO LONGER ELIGIBLE to cross over. Think about what @VPrasadMDMPH & @DrMakaryFDA are asking of patients. In just 12 months, patients in the sham portion of the trial WERE NO LONGER ELIGIBLE to get treatment. They risked their lives to take part in the trial then progressed to the point of no longer being able to be treated with the real stuff. This is insane. This also highlights the lies that Prasad told the group of reporters that he called together last month when he stated the sham control could be designed where patients were only under anesthesia for 1/2 hour and received a nick in the scalp. It shows he either flat out lied OR EVEN WORSE, didn't even take the time to READ the trial design before making his decision to reverse prior guidance. No way he could have made those comments after reading how the procedure is performed. Either that or he is CORRUPT and in the pockets of someone outside the agency, very possible. ehdn.org/wp-content/upl… The other thing to point out is how the EU regulators wouldn't allow the sham arm because it is unethical. How can these morons be in charge of our once esteemed FDA? Dr Makary needs to go NOW as he is destroying our biotech industry at an unbelievably rapid pace. @SenRonJohnson @SenRickScott @RepAuchincloss @adamfeuerstein @BeckyQuick @l_e_whyte @SenGillibrand please don't let up the pressure on the leadership of the @US_FDA, make them go back to prior alignment and allow AMT130 to be filed. Give the HD community a shot at receiving the only treatment that has demonstrated a real slowing of disease progression.

Narcos S01-S03 - 1080p BluRay - With Featurettes - 63.34 GB gdflix.dev/pack/KGDmHY0w85 Featurettes: - Deleted Scenes - Behind The Scenes & More.

$QURE $RGNX More than 100 marchers took to Capitol Hill on Wednesday, carrying a coffin to the FDA in a symbolic protest highlighting lives they say could be lost to regulatory delays affecting a group of rare metabolic disorders, according to a release shared with Fierce. fiercebiotech.com/biotech/rare-d… The demonstration comes as controversy around the FDA’s rare disease decisions continues to escalate following uniQure’s heated back-and-forth and several unexpected rejections. @SenRonJohnson @RepAuchincloss @realDonaldTrump @LauraLoomer

From the Science Lab to the Medicine Cabinet: How China is Cornering the Market on Our Medicines 03-18-26 x.com/i/broadcasts/1…