BioBoyScout

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BioBoyScout

BioBoyScout

@BioBoyScout

Independent Biotech Analyst & Patent Atty. Robert Toczycki, JD, MBA. Developer of online biotech valuation calculators. Chicago native. Opinions my own. RNAi

参加日 Şubat 2015
852 フォロー中4.2K フォロワー
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BioBoyScout
BioBoyScout@BioBoyScout·
THE YEAR OF TAU: ARO-MAPT, the CNS Delivery Breakthrough, and Why 2H2026 Could Redefine $ARWR. My most important research on Arrowhead. The Street doesn't know what it's looking at. This paper makes the case. The re-rating event is coming. drive.google.com/file/d/1Nskx5B…
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BioBoyScout
BioBoyScout@BioBoyScout·
My next $ARWR white paper is in the works. It makes everything I've written before look tame. Give me at least a couple more weeks. Fair warning: you're not ready for what's in it. Buckle up, what I found will stop you cold. 🧬 #RNAi
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BioBoyScout
BioBoyScout@BioBoyScout·
"ARO-MAPT is not an Alzheimer's drug bet. It is a bet that $ARWR has solved the hardest problem in RNA therapeutics, getting #RNAi molecules into the brain after a subQ injection, and that the market has not yet priced what that capability is worth." drive.google.com/file/d/1Nskx5B…
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BioBoyScout
BioBoyScout@BioBoyScout·
The most detailed research I've ever published on $ARWR will be dropping soon. Very long and thorough. Covers angles I haven't seen discussed anywhere on the Street. I don't think analysts know what they're looking at right now. Re-rating event incoming. Stay tuned. #RNAi
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BioBoyScout
BioBoyScout@BioBoyScout·
@GeneInvesting 12/ Greatly appreciate the thoughtful pushback. This is exactly the kind of discussion that sharpens the debate on both sides. Too bad the major influencers avoid this topic entirely. #GeneEditing #RNAi $ARWR $ALNY
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BioBoyScout
BioBoyScout@BioBoyScout·
@GeneInvesting 11/ Gene editing enables powerful biology. RNAi aligns more naturally with how medicine is practiced, reimbursed, and scaled today. Those can both be true.
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Gene Investing w/Anthony 🧬
Gene Investing w/Anthony 🧬@GeneInvesting·
1/n RNAi versus Gene Editing 🧬 A Rebuttal Thread 🧵 (based on recent white paper from @BioBoyScout) List of the 5 key CRISPR in vivo programs: 1. HAE • RNAi tried and failed • ASOs like $IONS can’t compete on efficacy and are extremely expensive annually • Gene editing appears the clear winner here 🥇 2. ATTR • $ALNY created a big Pharma essentially off of ATTR and looks poised to capture large % of this market moving forward • That said, a one & done option appears highly desirable to patients (Nex-Z $NTLA was enrolling as fast as Vutrisiran) and 89% of cardiologists were extremely/very likely to prescribe (n=232) • Huge $20 billion plus market and will Alnylam likely make way more money than Intellia? Sure, but factoring in their market caps $1.38b vs. $41.92b, it’s clear to see who has more upside from a huge market like ATTR 3. AATD • $BEAM and $PRME appear poised to dominate this market 🥇 • RNA editing data has looked weak 4. WD • $PRME looks poised to own this market with little competition 🥇 5. CF • I see no company in the RNAi space that looks positioned to compete with Prime • too early to definitively call Summary: Arguably 4.5/5 in favor of gene editing, with RNAi likely making much more money in ATTR, yet arguably already priced into Alnylam’s large valuation. CF is too early, but no one can compete with prime editing if delivery is advanced enough. Looking above, I see gene editing “easily outpacing” RNAi with 3 successful in vivo companies (NTLA, BEAM, PRME). Most gene editing won’t be gene silencing, and even on HAE there was no RNAi company who could compete.
Gene Investing w/Anthony 🧬 tweet media
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BioBoyScout
BioBoyScout@BioBoyScout·
Illinois is headed to the Final Four! Iowa was cooking in the first half, 57% shooting, up 4 at the break. Then someone gave the Illini a shot of RNAi. Second half? Iowa silenced. #Illini #MarchMadness
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BioBoyScout
BioBoyScout@BioBoyScout·
@Hall8Jack ALK7 vs INHBE isn’t about better biology, it’s about clinical impact. ALK7 hits adipose directly → real weight & visceral fat loss, muscle preserved. INHBE complements it upstream. Trial design + patient targeting = why $ARWR leads.
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BioBoyScout
BioBoyScout@BioBoyScout·
$WVE hit the biology, but trial design limits impact. $ARWR got it right with its superior trial designs: combo therapy, higher BMI patients, longer follow-up = real weight loss, visceral fat reduction, and muscle preservation. Outcomes matter. $ARWR is now clearly in the lead.
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