Long thread Sunday. I’m not able to make the trip to LA for #ASGCT23, but that didn’t stop me from combing through 1,700 abstracts to bring you just as many tweets on the highlights. Some key themes and tidbits once again (1/26) Before we dig in, a refresher on the themes I called out last year, as many are still relevant and highly represented this year. I’ll try to focus on new areas (2/26)

https://twitter.com/prof_oak_/status/1522587775014428673?s=46&t=mkJGGldDdQyuED4alkfLhw

Some Sunday musings on RT-PCR vs. ddPCR for exon skipping quantification in DMD, which has been a consistent debate over the years

Updates from $PEPG at MDA last week demonstrate the perils of RT-PCR, and call into question PGN-EDO51's differentiation vs. $SRPT's SRP-5051 (1/10) $PEPG's S-1 is essentially a hit piece on $SRPT, presenting multiple head-to-head data cuts for PGN-EDO51 vs. R6G-PMO, an SRP-5051 analogue

After single 30mg/kg dose, achieved >40% exon skipping *as measured by RT-PCR*

Looks pretty impressive, potl. best-in-class (2/10)

Recent Keystone pres from Epic Bio was intg. Epic is a @stanleyqilab spinout focused on epigenetic editing. Launched in '22 w/ $55M A

2 key aspects from Keystone:
(1) Novel effectors for fusion protein-based activation systems
(2) POC for *durable* "CRISPR-ON" activity in vitro (1) HTS of 45K effectors identified series of human/viral activators. DL used to identify and engineer minimally functional 'core' motifs. Resulting variants are on avg. smaller with similar or better function in vitro across a panel of genes vs. previously reported activators

Should get $ABEO Phase 3 RDEB data in the next couple of weeks. Stock is down >90% in the last year, trading at <$25M FD market cap, a fraction of the potential value of a PRV if EB-101 is approvable. Interesting set up? Let’s take a closer look (1/18) VIITAL is a controlled study in which patients serve as their own control. We know from 2Q22 ERN that 43 wounds with a mean body SA of 156cm^2 were treated across 11 patients. Per FDA requirements, the trial has co-primaries of (1) wound healing and (2) pain reduction (2/18)

Interrupting the #Omicron coverage flooding your feed for some under-the-radar ASH abstracts to which I'm looking forward. Collectively, these aren't the "stock moving" headliners that will get broader attention, but are sleepers with important & far-reaching implications (1/24) In the non-viral delivery world, first HSC-tropic LNP data in NHPs from $BEAM, showing up to ~19% "transduction" at doses up to 1mg/kg. Recall, we've seen directionally similar efficacy data from $NTLA, but doses were not disclosed (2/24)

ash.confex.com/ash/2021/webpr…

Have been eagerly anticipating the $BLUE / $TSVT Abecma launch as the first glimpse into the asset-level profitability of autologous CAR-T, as the CD19s have all been buried in big pharma income statements In 3Q21, Abecma generated $67M in US revenue. Product-level profit is split 50 / 50 with $BLUE / $TSVT

$BLUE / $TSVT recognized $13M in collaboration profit in 3Q21, implying a product-level operating margin of ~39%