GLOBAL RESEARCH ARCHIVE
FORCE Data @ASGCT Shows Impressive CNS Delivery; Z-ros BLA in 2Q:26
Research evidence excerpt
FORCE Data @ASGCT Shows Impressive CNS Delivery; Z-ros BLA in 2Q:26
q2W doses of 3mg/kg siRNA with a CNS- Shares Out (mil) 165.3
optimized TfR1 Fab decreased MAPT RNA by ~75% consistently across deep and Form 10Q as of May 8th
cortical brain regions. KD was stable for at least 4 weeks. MAPT KD translated into Market Cap. (mil) US$3,048.1
a >50% reduction in tau protein in both the entorhinal cortex and hippocampus, key Total Assets ($mil) 1,080
regions of tau accumulation in Alzheimer's disease. Dyne also showed that in humanized Avg Daily Vol (000) 2,053
Book Value/Share US$5.23 mice, subcutaneous siRNA could KD MAPT to a similar level as IV administration. We
Price/Book 353%
are impressed by the depth of MAPT KD with systemic delivery, especially in hard-to- Net Cash Per Share US$4.98
reach deep brain regions. We see blockbuster potential for tau-lowering therapies for 1Q:26 cash less Hercules debt
the treatment of tauopathies like Alzheimer's Disease, progressive supranuclear palsy, Debt to Total Capital 13.8%
and many cases of Parkinson's Disease. Div Yield 0.00%
Fiscal Year End Dec
• Z-rostudirsen BLA in 2Q:26; Potential Approval by 1Q:27. Dyne previously reported
Price Performance - 1 Year
that 20mg/kg q4W Z-rostudirsen (n=21) increased muscle-content adjusted dystrophin
by 4.63% to 5.46% vs. a 0.03% increase with placebo (n=7) to 0.95% at 6 months. USD
(p<0.0001) The safety profile remains favorable. Dyne will file the BLA for z-rostudirsen 25
in 2Q:26 and seek priority review. We anticipate accelerated approval and U.S. launch 20
in 1Q:27. Dyne plans to initiate a confirmatory Phase III trial in 2Q:26. Beyond the lead z-
rostudirsen (Exon 51) program, Dyne is also leveraging its FORCE platform to advance 15
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