Biotech trader/investor with a focus on nucleic acid-based and -targeted biotech. Fiercely independent. https://xitter.cf/t.co/dfLqOuH7Uh

Germany
Joined November 2011
It was a great honour to present the results of the phase 3 INTerpath-001 clinical trial at the #ESMO26 Congress in Madrid. This international trial included 1137 patients, across 157 sites, whose melanoma had been surgically removed, but who were at very high risk of it returning. The trial showed adding #intismeran autogene, an mRNA based individualised neoantigen therapy, to pembrolizumab reduced the risk of recurrence or death by 42%. This is the first phase 3 study to show that a treatment built from the unique ‘fingerprint’ of a patient’s own tumour can reduce the risk of recurrence or death from cancer. That fingerprint is read by sequencing the tumour, then a ‘wanted poster’ of the cancer is made as mRNA, showing the immune system exactly what to look for and attack. Having succeeded in melanoma, this proof of concept could mark the beginning of a foundational shift in how we treat cancer. Read more >melanoma.org.au/news/persona… A huge thank you to all patients and teams involved. @myESMO @Sydney_Uni @syd_health @Science_Academy @AAHMS_health @FulbrightAUS @NthSydHealth @ausoftheyear
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Seven hundred and fifty eight subjects received a personalized mRNA, not one of which was described in an IND. Each made in 4 weeks from tumor biopsy. The process is the product. Hello personal CRISPR cures.
Today we announced detailed positive results from the Phase 3 INTerpath-001 study evaluating intismeran autogene (mRNA-4157 or V940), an investigational mRNA-based individualized neoantigen therapy, in combination with KEYTRUDA® (pembrolizumab), @Merck's anti-PD-1 therapy, in patients with high-risk melanoma (stage IIB-IV) following complete resection. These late-breaking results will be presented during a Presidential Symposium at the 2026 European Society for Medical Oncology (@myESMO) Congress. Read more: feeds.issuerdirect.com/news-…
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Today, with our partner @GSK, we announced the FDA approval for a first-of-its kind medicine for the treatment of chronic hepatitis B (CHB) in the U.S. Learn more: bit.ly/3UyByeq $IONS
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America should lead the world in turning medical breakthroughs into treatments for patients. HHS is cutting the delays, duplication, and unnecessary costs that hold clinical trials back. Through @ARPA_H, we are building a modern clinical trial system that generates rigorous evidence faster, reduces the burden on patients, and moves effective treatments from discovery to patients sooner.
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On the latest #BioCenturyShow @StephenPHansen speaks with @ArrowheadPharma CEO Chris Anzalone about solving the challenge of delivering #siRNA to extra-hepatic tissues, and how breakthroughs here can enable future growth opportunities for Arrowhead $ARWR buff.ly/DThToy4
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Congrats to the team at @ArrowheadPharma! This is a major milestone in the field, with opportunity for continued improvement! Great news for patients!
Today we announced interim Phase 1/2a clinical data for ARO-MAPT. The results represent an important step in clinical validation of our TRiM platform for delivering siRNA to the central nervous system. Join today’s webinar to learn more: bit.ly/3VMXyCD
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Replying to @RNAiAnalyst
$MRNA 2.0 is about to start.
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Replying to @RNAiAnalyst
The new mgmt team literally did not lower the price that Martin set. I haven't heard a single word of protest over what Daraprim is selling for ever since Martin was ousted
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Dirk Haussecker retweeted
Redacted Oct 2 filing: $LQDA filed NDA amendment to "revert to its previously adjudicated non-infringing approval for PAH" & "is working with the FDA to expedite approval of this supplement to its NDA to help ensure there will not be a disruption of Yutrepia availability for PAH"
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Dirk Haussecker retweeted
Out of $nktr completely broken down with no buyers in sight. Big clear out with some other names too, kamikaze large levered $mrna short @ $196.5
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#NEWS: Editas Medicine Receives Approval to Initiate First-In-Human Clinical Trial of EDIT-401 in Patients with Hyperlipidemia. Read the press release for details: bit.ly/4jKSqZt. #geneediting #HeFH #cardiovasculardisease #clinicaltrials
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BREAKING: President Trump says the White House considers anyone who uses the term “Artificial Intelligence” the enemy.
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Irkutsk, Russia: The U.S. Embassy is aware of press reports from the Irkutsk region of suspected pneumonic plague resulting in the death of an individual and of associated quarantine measures in, and closures of, hospitals in Irkutsk. We continue to track the situation closely. The U.S. government has limited ability to help U.S. citizens in Russia, especially outside of Moscow. The State Department’s Level 4 Travel Advisory (Do Not Travel) for Russia remains in place and states U.S. citizens currently in Russia should depart immediately. U.S. citizens planning travel to Russia are again strongly advised not to travel to Russia due to risk of wrongful detention and security concerns. More at: ru.usembassy.gov/health-aler…
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First announced patient dosing of an in vivo prime editing treatment (to correct ATP7B H1069Q, one of the most common genetic causes of Wilson disease). There are now at least three prime editing clinical trials underway—for CGD (ex vivo, with positive clinical outcomes), for Wilson disease (in vivo), and for alpha-1 antitrypsin deficiency (AATD, in vivo).
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Here's what we know. A 28-year-old technician at an "anti-plague institute" reportedly broke a tube containing plague pathogen on September 25. She died October 1 or 2. Nearly 200 people are now in medical isolation 2/n
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Minhua Chu
@chuminhua432Oct 2
$NVS signs a $7.8B+ deal with 🇨🇳 Abogen Biosciences to advance mRNA-encoded therapeutics, including an in vivo CD19xCD3 T-cell engager for autoimmune diseases. abogenbio.com/news/company/n… 💰 Deal terms: • $575M upfront • Up to ~$7.2B in potential milestone payments if all options are exercised • Potential royalties on future product sales • Exclusive worldwide license to lead asset ABO2203 • Options to exclusively license additional programs from Abogen’s RNA platform ABO2203 is an mRNA-encoded CD19xCD3 T-cell engager. Abogen says ABO2203 is the first mRNA-encoded T-cell engager to enter clinical evaluation for autoimmune diseases. Founded around RNA innovation, Abogen has an AI- and automation-driven mRNA/LNP platform. Its pipeline covers oncology, autoimmune diseases and vaccines, including ABO1108, a lyophilized mRNA shingles vaccine in Phase 3 trials in China.
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Dirk Haussecker retweeted
The US 10-year government bond yield is now trading at 5.30%. #economy #markets #yields #bonds
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Replying to @AppleHelix
yep -scibrunch founded by Wuxi Apptech guys who ran drug discovery work for revmed. given that erasca molecule may also be from revmed work at wuxi this smells bad. if money flowing to china is going to shops associated with western discovery work there it is super concerning.
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Replying to @RNAiAnalyst
Typically yes. However there’s a second approach (full disclosure: I invented it), namely to change the anti-VEGF molecule by adding a vitreous anchoring peptide to the non-business end of the molecule. You hijack the vitreous as your sustained release polymer. Been working on this at @OptigoBio for many years and finally preparing for the clinic. In preclinical models we show potential for 9-12 months without loading doses by preventing rapid diffusion out of the eye. Gene therapies cause a ton of inflammation. Variable expression. Need rescue doses. And may not be repeatable. optigobio.com/
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