Jun. 12 at 12:01 AM
$WVE — significant catalysts stacking into 2026 across editing + silencing:
🧬 AATD (006): RestorAATion-2 delivering — Z-AAT down ~71%, M-AAT restored to ~64% of total, durable monthly dosing, clean safety, no LNPs. FDA feedback on a potential accelerated-approval path expected mid-2026; 600mg multidose data mid/late ’26. First-ever clinical RNA editing translation.
💪 DMD (N531): NDA filing on track for 2026, exon-53 skipping via accelerated approval (dystrophin + function). Near-term regulatory inflection.
⚖️ Obesity (007/INHBE): Ph1 showed -14% placebo-adj visceral fat at 6mo with muscle preserved, potential 1–2x/yr dosing. The real catalyst — Ph2a multidose in higher-BMI (35–50) patients — on track to start Q2 2026.
🫀 Liver (008/PNPLA3): ~9M homozygous I148M individuals, CTA filing expected 2026. Quiet program, enormous TAM.
➕ HD (003): Huntington’s optionality on top.
~
$544M cash, runway into Q3 2028. RNA editing pioneer that’s shipping data. NFA / DYOR. 🌊
