Longroader
@Longroaderstar
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Biotech seeker, Fund manager. Not financial advice
Global
Joined December 2021
$sgmo Expected low volumes around pivotal FED meeting and pre Fabry news but the large buy queues at 51 got my attention. Few days.
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WSJ Op-Ed on Elevidys. $SRPT In a WSJ article dated today (Sep. 14), Elizabeth Currid-Halkett, mother of a child with DMD and a USC professor, argues that DMD should be included in the Recommended Uniform Screening Panel for newborns. (Public comment period for adding DMD closes
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$arwr $4.2b mkt cap, less than 50% of $ions and 10% of $alny Keep that in mind.
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$sgmo Last sentence says "Follow our progress...". Draw your own conclusions.
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$xbi #xbi Gold lining for bioinvestors, money should flow back to USA innovation. Good for small caps. https://t.co/jg83CgbR4b
nytimes.com
Behind the scenes, major pharmaceutical companies and Trump-tied billionaires are furiously lobbying in opposite directions over proposed anti-China measures.
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$arwr Why are we are not at $5b already with such an excellent pipeline.
ir.arrowheadpharma.com
Target the gene, silence the disease
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#BLSdata Not only that, the traders and investors who lost money with these fake numbers could sue the hell out of them.
This is another BLS scandal, and Trump was absolutely right to fire the BLS commissioner. What this means: 1). There was virtually no job creation in the last year of the Biden admin. 2). The Fed should have started cutting in February.
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$trml $xbi #xbi Novartis busy reinvesting pipeline - Anthos, Argo, $rgls $arwr and now Tourmaline. https://t.co/zLMgIzxwvC
bloomberg.com
Novartis AG agreed to buy Tourmaline Bio Inc. in a deal valued at about $1.4 billion as the Swiss drugmaker continues to pursue bolt-on deals to boost its drug pipeline.
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$xbi #xbi Might see BP pull the trigger this weekend on some closely followed names before things get too expensive. XBI 100
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$prqr "Hosting fall Analyst and Investor Event (virtual), featuring detailed AX-0810 Phase 1 trial design and 2025 data expectations, plus updates across differentiated liver and CNS pipeline" https://t.co/1npdX0P4DT
proqr.com
ProQR is a clinical stage biotech company based in the Netherlands that discovers and develops unique RNA-based therapies for severe genetic disorders.
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$prqr Van Herck added 1.07m shares, now 12.6%. https://t.co/TRU56TXUCx
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The labor market is far worse than you think: Not only was June's jobs number negative, but the US economy lost -357,000 full time jobs in August. And, the US gained +597,000 part-time jobs. Millions of Americans are piling into part-time jobs because it's all they can get.
This is absolutely insane: The US just revised the June jobs report lower for a SECOND time for a total of -160,000 jobs. Now, the US has officially LOST -13,000 jobs in June, the first negative month since July 2021. What just happened? Let us explain. (a thread)
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$QURE "AMT-191 showed a manageable safety profile....." Two SAEs out of 4 patients at higher dose. Second cohort with 1/3 dose seems much better. Still much farther behind than $sgmo with excellent safety and BLA early 2026.
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$sgmo @grok This is interesting, I'm beginning to like Grok in its discourse. So much improved.
@BiotechAnalysst @RNAiAnalyst @meangenebio Agreed, the positive eGFR slope in Sangamo's STAAR study is notable—latest data shows +1.97 mL/min/1.73m²/year at 52 weeks (n=32). In contrast, other Fabry treatments like Fabrazyme and Galafold typically show negative slopes (-2.2 to -0.4). If long-term data holds, this could be
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$sgmo The dip mostly comes before the pump, to scoop up. Nothing has changed, its a positive slope and a cure.
investor.sangamo.com
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
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$wve $prqr $krro Although results not as good as $beam, it is still positive and validates the tech. This is totally new, with excellent safety, lower doses and other benefits. Surely will find its niche which RNAi and Crispr can't.
globenewswire.com
Achieved durable production of serum AAT protein at levels associated with a lower risk of AATD liver and lung diseases following repeat 200 mg doses of...
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$SLN Is this the cheapest RNAi? Very quiet here. $arwr $ions $alny
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Federal Reserve Gov. Christopher Waller: "We need to start cutting rates at the next meeting... Over the next 3-6 months, we could see multiple cuts coming in."
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$arwr $200m upfront for a preclinical. Market is not paying enough attention yet.
ir.arrowheadpharma.com
Target the gene, silence the disease
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