$ntla fell like a rock.

$srpt sold $160M in stock today; also gave $arwr $50M worth of stock as part of $100M milestone.

In the end $arwr did $srpt a favor here. A partner that can be counted on in difficult times. You dont see that often in the stock market.

'Desiccated thyroid extract' instead of synthetic alternative. Maybe we should go back to the good old times when #diabetes, #hemophilia and #HAE were treated with purified extracts. HCV...HIV... Beef tallow for frying French fries next?

Would cowpox isolated from cows be an acceptable vaccine to you guys? Bring back the good ole days. #FDA #HHS

💯at some point, the buck stops with Makary. $repl $srpt

$dyn revisiting shift of primary endpoint from CASI (splicing score) to vHOT (functional endpoint) as demanded by new #FDA: what happened to 'plausible biological pathway'? Myotonia affects many, but not all DM1 patients + various degrees, mis-splicing is common to all forms of

#biotech summer doldrums. Only bad data will be announced by September. Good data shortly thereafter.

$prme interesting keynote talk by David Liu, including disease-agnostic suppressor tRNAs enabled by prime editing. youtube.com/watch?v=Z3OM5c…

So $repl had to meet higher bar because their target population 10-20x higher than $pgen's?

Is $PGEN approval sign of...

$repl this whole Pazdur versus Prasad driving the CRL, an idea propagated by Adam Feuerstein ✡️...who everybody should know by now cannot be trusted. This might allow for a reversal of decision 'by Prasad' without him losing face. Prasad may have commissioned Adam's Stat piece 😜

$pgen FULL approval following accelerated approval pathway. Almost like giving AA to #Elevidys when $srpt not even asked for it. Has Vinay been to a CCP-style re-education camp or what?

$prme after $pgen, full approval and PRV for CGD any time soon? CNPV would help here, too.

$unh Warren's way of saying serious PBM reform wont happen.

$pgen why hold conference call on Monday, not today?

Nice. Makary calls it a 'gene therapy', not an immunotherapy. He's the regulatory flexibility guy that should be very positive to #CRISPR in particular (another 'novel tech'). Hope he finds a way to hang out with Prasad without endangering his own job. $pgen $qure $sgmo

#portfolio update: took some $ off table ahead of 3 September clinical data read-outs I'm playing: 1) $ions sHTG p3s. huge underserved market w/ lots of off-label potential, too. 50%+ trig reduction, good safety. Stat sig pancreatitis would be home-run. Long. Also watch next