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@Hall8Jack "@TTRAmyloid But yet $rna insiders dumped $20M the last month. And data is far from slam dunk for stat sig in P3: $arwr $dyn $pepg"
X Link @Hall8Jack 2025-09-04T23:24Z 1585 followers, XXX engagements

"@ShafferBiotech And why no functional data similar to Avidity (which imo was pretty mediocre for efficacy and safety) if splicing data so superior"
X Link @Hall8Jack 2025-09-24T23:30Z 1585 followers, XXX engagements

"@BiotechAnalysst So thats X Imlifidase studies from $hnsa - one with Genethon and this from $srpt :"
X Link @Hall8Jack 2025-10-14T10:21Z 1584 followers, 1314 engagements

"@blippd @scamsweeper Early data- $pepg clearly better splicing than $rna at single dose; rna saw DLT at 4mg/kg. Pepg has gotten beyond 4mg/kg but need to see MAD studies- that is very key. Pepg has had multiple clinical holds (1/2 contd):"
X Link @Hall8Jack 2025-09-24T21:05Z 1585 followers, 8956 engagements

"@yachmod Think you mean Genethon $srpt used to own US rights to this. Not sure where that stands now:"
X Link @Hall8Jack 2025-10-11T01:01Z 1586 followers, XXX engagements

"@semodough I think $srpt only saw hypoMG in their MAD study for Srp-5051. So the sad study doesnt mean much no $pepg already seen X clinical holds"
X Link @Hall8Jack 2025-09-24T22:08Z 1584 followers, XXX engagements

"@cal89511029 $srpt indicated they would file BLA for Srp-9003 before year end:"
X Link @Hall8Jack 2025-09-23T17:42Z 1584 followers, XXX engagements

"@TTRAmyloid (2/2) $rna $srpt $dyn $wve $pepg #NS Exon skip tough from a regulatory standpt in terms of grind it out piecemeal approvals; many skippers have small TAMs (ie exon 44); v crowded in terms of competition. Good summary on DMD exon skipping field/competition:"
X Link @Hall8Jack 2025-09-05T06:19Z 1584 followers, XXX engagements

"@Pharmdca If you look at what happened to PGN-EDO51 after it went from sad to mad studies (clinical hold; discontinued) not a big shocker where $pepg market cap is. PGN-EDODM1 about to go from sad to mad. $rna $dyn $arwr $srpt . Also this:"
X Link @Hall8Jack 2025-09-25T15:24Z 1584 followers, 1162 engagements

"@blippd @scamsweeper Also only seen splicing data from $pepg If so really need to see DM1 functional data in MAD $rna $dyn $arwr"
X Link @Hall8Jack 2025-09-24T21:11Z 1585 followers, XXX engagements

"This is true with one time GTxs. But key is an overall fatality rate of X% for $srpt; a one time tx. Juxtapose this vs chronic (lifetime) RNA txs that are seeing discontinuations (and related clinical holds) for $rna $dyn $pepg etc in multiple muscular dystrophy dxs"
X Link @Hall8Jack 2025-09-22T14:33Z 1585 followers, 1452 engagements

"@TTRAmyloid There are less than 1k x44 patients. Small TAM. DelZota has had a discontinuation in small N trial so there are worrisome safety signals. Also was a discontinuation in $rna DM1 program. And the data isnt even clearly better let alone 10x better lol"
X Link @Hall8Jack 2025-09-16T18:08Z 1585 followers, XXX engagements

"@MauriceOnTW $rgnx has presented limited functional data (which if I recall not differentiated from $srpt Elevidys). Has $sldb presented any functional data at all thus far And is it fair to summarize that sldb has disclosed more safety events (multiple thrombocytopenias) compared to rgnx"
X Link @Hall8Jack 2025-09-03T01:51Z 1583 followers, XXX engagements

"@blippd @scamsweeper (2/2) so safety is in question for their platform and all comes down to MAD. But imo $pepg has a chance as $rna most advanced in DM1 but efficacy+safety pretty mediocre. Also $arwr DM1 data imminent and also has chance at BIC as think theyve gotten well beyond 4mg/kg"
X Link @Hall8Jack 2025-09-24T21:08Z 1584 followers, 1061 engagements

"@MauriceOnTW Well N=5/6. Really need P3 data to truly compare sldb vs rgnx; srpt"
X Link @Hall8Jack 2025-09-03T01:59Z 1583 followers, XXX engagements

"@newerraa Do you have any expectations from the ARO-MMP7 IPF trial $arwr has proven they can get lung exposure with ARO-RAGE but $srpt doesnt mention ARO-MMP7 anymore"
X Link @Hall8Jack 2025-10-07T16:47Z 1585 followers, XXX engagements

"@richtrades100 If you look at recent pps performance and regulatory updates $tsha and $ngne have gotten for their Rett GTx or $qure for their HD GTx- $srpt should have very similar path for their LGMD2e GTx"
X Link @Hall8Jack 2025-10-11T01:10Z 1584 followers, XXX engagements

"@TTRAmyloid Has $rna released any functional data on its exon XX skipper to show that it works (1/2)"
X Link @Hall8Jack 2025-09-05T06:02Z 1585 followers, XXX engagements

"@ShafferBiotech $pepg $rna $dyn :"
X Link @Hall8Jack 2025-09-25T10:22Z 1584 followers, XXX engagements