PGN-EDODM1
Nucleic Acid Target: DMPK mRNA, CUG repeat expansion, MBNL1, DMPK
Most recent annual filing: 2026-03-04
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Enhanced Delivery Oligonucleotide platform leveraging cell-penetrating peptides (CPPs) conjugated to oligonucleotide therapeutics (PPMOs) to optimize tissue penetration, cellular uptake, and nuclear delivery; focus on neuromuscular and neurologic diseases including myotonic dystrophy type 1
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