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Drug Development.

Jack Stahl1, Aswathy Joji1, Elianny Perozo Rojas1

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|December 25, 2025
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Summary
This summary is machine-generated.

Chemically optimized stringed modifiable oligonucleotides (COSMOs) offer a novel therapeutic approach for Alzheimer's Disease (AD), simultaneously reducing amyloid beta (A𝛣) and phosphorylated tau (p-Tau). This dual-action strategy shows promise for more effective AD treatment compared to single-target therapies.

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Area of Science:

  • Neuroscience
  • Molecular Biology
  • Pharmacology

Background:

  • Alzheimer's Disease (AD) is characterized by amyloid beta (A𝛣) and phosphorylated tau (p-Tau) accumulation.
  • Current A𝛣-targeting antibodies have limitations, including brain swelling and failure to reduce p-Tau.
  • Oligonucleotide therapeutics show promise, with siRNAs reducing A𝛣 and ASOs reducing p-Tau.

Purpose of the Study:

  • To develop a novel therapeutic approach for Alzheimer's Disease (AD).
  • To create multi-targeting siRNAs, termed COSMOs, capable of simultaneously reducing both A𝛣 and p-Tau.
  • To evaluate the efficacy and safety of COSMOs in vitro.

Main Methods:

  • Design, synthesis, and screening of siRNAs targeting amyloid precursor protein (APP) and microtubule-associated protein tau (MAPT).
  • Transfection of human SK-N-AS cells with COSMOs and individual siRNAs.
  • Quantification of mRNA and protein levels of APP, Tau, A𝛣42, and p-Tau181 using qPCR and ELISA.
  • Assessment of cellular viability and COSMO cleavability.

Main Results:

  • Top siRNAs and COSMOs demonstrated activity at picomolar concentrations.
  • Co-transfection and COSMO transfection simultaneously reduced A𝛣 and p-Tau levels.
  • APP- and MAPT-targeting COSMOs exhibited lower toxicity than individual siRNAs.
  • COSMOs can be engineered for tunable cleavability.

Conclusions:

  • No current AD therapeutics effectively reduce both A𝛣 and p-Tau.
  • COSMOs represent a promising new class of therapeutics for AD.
  • COSMOs have the potential to simultaneously target key pathologies in Alzheimer's Disease.