I should also mention the PTC/Novartis approach is the same as Skyhawk.
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Alex Harding
3.2K posts
Alex Harding
@AlexHarding7
An internist in biotech
Boston, MA Inscrit le Mart 2012
148 Abonnements3.4K Abonnés
These results in mice suggest that HTT1a reduction may be necessary for benefit in HD. Calls into question Skyhawk's approach, which does not address HTT1a (and supports UniQure's).
science.org/doi/10.1126/sc…
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@EricTopol @NatureMedicine Only 22 of 888 (2.5%) had an actionable result with WGS that wasn’t picked up on the 523 gene NGS panel. Very different vs the 40% figure you cited. There’s also th challenge of interpreting variants of unknown significance with WGS.
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Why should tumor whole genome sequencing (WGS) be done for cancer?
In real practice of medicine study of 888 patients with solid cancers, WGS directly led to clinical consequences in over 40%
@NatureMedicine
nature.com/articles/s4159…
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Alex Harding retweeté
this is the first major mention i've seen of arnold venture's influence on this fda. arnold is said to be have been a major backer of prasad's research (i don't have proof of this, have just been told many times), and as wsj notes, szarama worked at arnold before prasad brought her in. most aren't aware arnold ventures was the major backer of ICER, the group that defines the value of medicines for the UK (which is set at a cap of roughly 30k pounds per human life year). it is also the reason the UK has dropped below basically all other developed countries when it comes to access to rare disease medicines. in short, arnold doesn't believe rare disease medicines are worth it, and it's natural to conclude prasad and others were bringing that philosophy to fda. now it's fine to have that view as a society (as the UK chooses to), but the trump administration represented the opposite, which is to accelerate access to rare disease medicines.
wsj.com/opinion/one-ex…
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Alex Harding retweeté
If you've had a kidney stone, you've been advised that the most important thing to prevent another bout is to increase hydration. Now a randomized trial of hydration in over 1600 participants showed no benefit, despite evidence of increase during volume. thelancet.com/journals/lance…
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Alex Harding retweeté
How specific are therapeutic monoclonal antibodies, really?
In our new paper, @Yile_Dai led a collaboration with Adimab to profile 174 FDA-approved and clinical-stage mAbs against 6,172 human extracellular proteins.
What we found surprised us.🧵
sciencedirect.com/science/articl…
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"The AI platform came first and then the pipeline that it generates will come second" ⏩ "We'll spend $1B on 'AI' and then we'll license some me-too assets."
It's crucial to build a platform with product(s) in mind first, not as an afterthought!
fiercebiotech.com/biotech/xaira-…
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Alex Harding retweeté
Lowering LDL will extend lifespans.
The trials, the genetic studies, and even the prospective cohort studies all agree that the more you lower LDL, the greater the reduction in your risk of heart disease.
This is one of the clearest chronic disease prevention findings there is.
Crémieux@cremieuxrecueil
This is good! More people should be on statins. They are basically a wonder-drug. They have very few side effects and almost no serious ones, and they deliver huge reductions in cardiovascular risk and extension in lifespans, for $2 to $10 per month.
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@johnarnold It’s a valid point but if we decrease med Ed it becomes increasingly blurred with mid-level training. Maybe we need more NPs and PAs (AI supported) and MDs should be highly trained consultants for difficult cases.
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AI should allow med schools to rethink whether 4 years is still necessary for med school. If students can focus more on clinical practice and less on memorizing the Krebs cycle and molecular bio, many programs could eliminate a year, reducing both costs and physician shortages.
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@cremieuxrecueil @CaryKelly11 Also we shouldn’t consider an LDL of 130 mg/dL “normal” anymore especially in high-risk people.
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@CaryKelly11 Because it's observational and the cross-sectional relationship is confounded. Lowering LDL still greatly reduces the incidence of heart attacks.
Hope that helps!
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Also in the real world...
People with lower or normal LDL tend to have more heart attacks than people with higher LDL.
At least in this study of 136,000 they did.
How inconvenient
Neil Stone@DrNeilStone
Meanwhile in the real world Heart attack rates and deaths trends since statins widely used
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Maybe he does care deeply about patients and wanted to protect them from interventions that he felt were unlikely to benefit them. But if that was his motivator, it didn’t come through.
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Probably not the reason he was fired but the bigger problem was his failure to demonstrate empathy for the patients suffering with diseases who could (potentially) benefit from the drugs he regulated. He seemed to care more about sounding smart than he did about helping patients.
Matthew Herper@matthewherper
5 lessons from Vinay Prasad’s turbulent tenure at the FDA Prasad brought his convictions to the agency — and reaped chaos. My thoughts on an eventful week for one of the most outspoken people in medicine. statnews.com/2026/03/08/vin…
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@Bob_Wachter @DShaywitz Nice article and IMO probably a generalizable observation: AI will have a huge impact on how we work, but it will take longer than the AI evangelists think. Human behavior and policy/regulation are slower to change than software.
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My latest Substack:
Why predictions that radiologists will be replaced by AI (including, famously, by genAI founding father and Nobel laureate Geoffrey Hinton) have been wildly off base, and what that teaches us about job replacement for doctors.
robertwachter.substack.com/p/why-do-you-s…
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Alex Harding retweeté
the oral wegovy launch continues to defy my expectations.
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FDA relaxing comparitvae efficacy study requirements for biosimilars
jamanetwork.com/journals/jama/…
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Alex Harding retweeté
Sharing our family’s story this week. Thanks to @Kramer_Katie for her beautiful work on this piece. ❤️
youtube.com/watch?v=-SD8-Y…
YouTube
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Alex Harding retweeté
This is really a good take. The key issue here is that there is subjectivity around "substantial evidence", "reasonably likely", "clinically meaningful". We need to better define the regulatory criteria that we want as a society through legislation. In the mean time, we should be doing more AdComs for these things where all stakeholders can be heard and a decision can be made by a panel of experts. Empowering one person at FDA is problematic for a number of reasons.
Adam Feuerstein ✡️@adamfeuerstein
In this week's Biotech Scorecard newsletter: The extremism of the FDA’s Peter Marks and Vinay Prasad has come with costs Two regulators, two extreme regulatory philosophies, one replacing the other. The rare disease community is suffering whiplash. Drugmakers ( $QURE most recently) are frustrated. Investors are sitting on their wallets. Adding to the confusion: The current FDA leadership often talks like Marks, but acts like Prasad. statnews.com/2026/03/05/vin…
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Alex Harding retweeté
Amazing quote in this NYT story on the FDA from @By_CJewett.
Janet Woodcock, a top drug regulator for decades, said reversals for a promising therapy send shock waves through the industry.
“The Huntington’s refusal I thought was truly evil,” she told NYT. “I just feel so bad for those people.”
nytimes.com/2026/03/05/hea…
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Alex Harding retweeté
Our study led by @SpinksCat @SunithaMargaret utilizes @AllofUsResearch estimating the prevalence of genotypic heterozygous FH in the US to be 1 in 287. Despite higher rates of lipid-lowering therapy, LDL-C values were suboptimal for the vast majority jamanetwork.com/journals/jamac… @JAMACardio
@gina_peloso @tigerstatdoc @buutrg @kparuchuri @HornsbyWhitney
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Alex Harding retweeté
In the generation before statins, more than 40% of people with familial hypercholesterolemia had some sort of major cardiovascular issue before age 40.
Their kids get on statins from an early age and end up being totally fine.
Statins save their lives.
Alan Couzens@Alan_Couzens
Something really important to know right now with all the B.S. around the cholesterol-CAC relationship going on at the moment... Somebody with familial (genetic) hypercholesterolemia is *born with* LDL-C levels of 160-400 mg/dL. Guess when these levels show up as elevated CAC? 1 year later? 3 years later? 5 years later? Try 30 years later! And in these cases of genetically high cholesterol, atherosclerosis almost always develops if left untreated. My advice: 1. Pay absolutely zero mind to any study trying to link LDL-C and CAC progression that is not multi-decade. 2. Don't (unnecessarily!) play Russian Roulette with your health!
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