Soufflé getting provocative in its first public data disclosure at MDA
Novel target for muscle-directed AOCs, seemingly capable of outperforming TfR1
$DYN $RNA $NVS
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Professor Oak
2.1K posts
Professor Oak
@Prof_Oak_
Biopharma Musings | Genetic Medicine & Rare Disease | Buyside Turned Operator | Not Investment Advice | DYODD
Beigetreten Aralık 2020
739 Folgt13K Follower
Professor Oak retweetet
One fun takeaway from my Lilly story: looking at $LLY from a market cap/employee ratio.
Far more in line with tech giants like $META $GOOG $MSFT than pharma peers like $MRK $AZN $PFE:
endpoints.news/eli-lilly-ceo-…
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and Science is better for it
BioT/X, on the other hand, not so fortunate
Nature Portfolio@NaturePortfolio
For Pokémon’s 30th anniversary, @Nature spoke to scientists from around the world about how their work has been shaped by playing Pokémon games, watching animated TV series and films and trading cards in school playgrounds. go.nature.com/4rIM2Db
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Professor Oak retweetet
Assortment of targets / programs that caught my eye recently, mostly earlier-stage stuff:
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Interesting novel capsid experiment shaping up in $ALPMY's (via Kate / $NVS) 2nd-gen XLMTM program
Ph1/2 design shared at MDA. Notably, high dose of 2e12 vg/kg is ~175-fold lower than ASPIRO's 3.5e14
Trial should ultimately provide the first real "head-to-head" of WT vs. Engineered AAV in a clinical setting
Professor Oak@Prof_Oak_
$ALPMY taking an asset-centric, bolt-on approach to bolster its AAV pipeline First, $TSHA deal for TSHA-120 (GAN) & TSHA-102 (Rett) Now, Kate deal for KT430 (XLMTM) Despite $BOLD's failures, encouraging to see continued investment in the wake of others' exits (PFE, TAK)
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With horizons as compressed as they are today, doubt Atrium gets much buzz before this time next year
Consequently, next 6-9 mo. likely to offer compelling entry points for those with longer duration
At the right price, should be a time arb layup $RNA
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Inexpensive at ~$20M EV, but $270M of birthday bucks will dwindle quickly
From $50M burn in ’25 - + standalone overhead, clinical costs, etc. - to $80-100M+ in ’26-28
Unsurprisingly, necessitates financing on ‘1072 FIH data by YE27 (if not before), prior to ‘1086 FIH data
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Turns out, $RNA is very far behind
Per SpinCo 8K, Atrium guiding to 2H27 IND & 1H28 Ph1 for PLN-DCM, more than 2 years after $IONS / $AZN
Professor Oak@Prof_Oak_
$IONS / $AZN disclose Phospholamban (PLN) DCM TfR1-siRNA program, which began a Ph1 in December $RNA SpinCo pursuing the same, and likely not far behind
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Aurora Anthropic’d by $BEAM. Must be how SaaS feels right now
Robert Kruse, MD, PhD@RobertLKruse
Aurora Therapeutics launches with $16M in VC funding to do gene editing for PKU with FDA platform approach in early January, and then today $BEAM announces they will do the same thing, and are only months away from the clinic. Tough out there in biotech. technologyreview.com/2026/01/09/113…
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Standard review for Anito Cel $ACLX $LEGN
Guess CARVYKTI’s inevitable & irrefutable -100% QoQ print will have to wait until 2027. Thanks, Vinay
Professor Oak@Prof_Oak_
But I thought $JNJ only cared about TCEs and CARVYKTI market share was going to zero $LEGN
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Professor Oak retweetet
Interesting optionality on top of a base biz that has been way oversold $LEGN
Jacob Plieth@JacobPlieth
$LEGN quietly making a big push into in vivo Car-T: LVIVO-TaVec100 (CD19 x CD20) LVIVO-TaVec200 (GPRC5D) LVIVO-TaVec400 (target undisclosed) ...all recently entered human trials.
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VAV1 is a perfect case study of current US-China fast-follower dynamics. "Undruggable" target, novel biology, minimal validating POC - and yet...
$GLUE MRT-6160 disclosed Jun-24
Patent published Jul-24 (Priority Date Jan-23)
$NVS deal Oct-24
Ph1 data Mar-25 (9M post-FPI)
Ph2 start 2026
InnoCare ICP-538 disclosed w/ IND Feb-26
Ph1 data 2026 (likely <9M)
Ph2 start 1H27?
~9-12 months behind, despite starting 2+ years later
Minhua Chu@chuminhua432
🇨🇳 InnoCare Pharma announced that China NMPA has approved the IND application to conduct clinical trials of ICP-538, a VAV1-directed molecular glue degrader (MGD). InnoCare said ICP-538 is the first VAV1 degrader approved to enter clinical trials in China and the second globally. innocarepharma.com/en/news/activi…
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Professor Oak retweetet
These comments in Nature Genetics on a GWAS signal for cerebral amyloid angiopathy at APOE are a great case study in the challenges of interpreting genetic associations even with abundant data.
Worth reading for anyone interested in translating GWAS hits to drug targets👇
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But I thought $JNJ only cared about TCEs and CARVYKTI market share was going to zero
$LEGN
FiercePharma@FiercePharma
J&J fleshes out US investment plan, telegraphing $1B cell therapy plant and 500 new jobs in PA fiercepharma.com/manufacturing/…
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99% of the discourse re: “Eroom’s Law” has focused on inputs - predictive validity of preclinical model, shortage of novel targets, physical constraints on design/screening, etc.
Not nearly enough attention paid to the structural economic forces that incentive herding and incrementalism throughout the extended, interconnected biopharma ecosystem (e.g., perceived Big P preferences -> VC’s M&A-supported thesis -> incrementalist NewCo)
The latter is reinforced by the former, of course, but ultimately, the “solution” will as much a product of economics as of biology
Jack Scannell@JackScannell13
It may be that failure to count competitors stampeding towards your de-risked target is now the single biggest reason drug R&D programs fail in Phase 2 & 3. "Commercial" termination overtakes "efficacy" circa 2019: nature.com/articles/d4157… Recent oncology example (TIGIT herding): bmjoncology.bmj.com/content/5/1/e0…
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Sad day for the Vampire Community. They deserve better from us
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