$SGMO Some nice receptor identification data showing STAC-BBB cross-reactivity between cyno and human receptors.

Three years later, $RNA is announcing initial clinical data from their FSHD program tomorrow morning. Management has guided to expect changes in DUX4 target gene expression, but potentially not for all patients due technical challenges in the biopsy. $ARWR $DYN $FULC

Antibody-Oligo conjugates continue to impress in FSHD $RNA data show, for the first time, convincing measures of DUX4 target engagement in humans $ARWR $DYN not far behind

$RNA vs $FULC reduction in DUX-4 regulated genes

$RNA RWS data look comparable to that of $FULC, but at an earlier time point (16w vs 48w). While Avidity likely has the more potent drug, this data derisks RWS as a more reliable endpoint for the Fulcrum readout in Q4.

Some folks have been asking why companies like $RNA don't report DUX4 knockdown. Short thread on why. DUX4 expressed in a development-dependent manner and silenced afterwards.

$DYN officially transitions to siRNA payload for their FSHD program. Always made sense given the significantly lower potency of ASOs. Original guidance for FSHD IND filing was 2021-2022, so looking at a delay of at least 3 years. $RNA $ARWR

$SRPT $SLDB $PFE Interesting preclinical study comparing µ-dystrophin vectors. insight.jci.org/articles/view/…

$RNA $DYN trading like BO Monday

$SGMO finally executes on BD for STAC-BBB. Gave up exclusive license for most advanced Tau program to only extend runway for 2 Qs, but bridges to potential $PFE milestone payments.

Incredible DMD44 data from $RNA, and quite surprising considering their preclinical data. Management stated on the call that they're observing encouraging trends on functional endpoints already. Gene therapies will be in trouble if DMD44 is not a special case.

Past 5 years we've seen the emergence and value creation of muscle delivery with $RNA $DYN. Optimistic that the next 5 will be in CNS delivery. $LLY $DNLI $VYGR