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RNA and Protein Degrader/Modulator Modalities of Small Molecules

In the evolving landscape of drug discovery, RNA and protein degrader/modulator modalities have emerged as revolutionary approaches in small-molecule therapeutics. These cutting-edge strategies offer a pathway to tackle previously “undruggable” targets and expand therapeutic horizons across multiple disease areas.

At Aragen Life Sciences, we empower innovation in Integrated Drug Discovery (IDD) by advancing cutting-edge RNA and protein degrader/modulator platforms. Our expertise transforms challenging drug targets into therapeutic realities, enabling breakthroughs across  oncology, CNS disorders, inflammation, metabolic diseases, and more.

RNA-Targeting Small Molecules

Small molecules targeting RNA are reshaping the therapeutic landscape by modulating gene expression at its source. These molecules can:

  • Inhibit RNA-protein interactions, disrupting pathways critical for disease progression.
  • Modulate RNA splicing to restore proper protein function or reduce the production of pathogenic variants.
  • Induce RNA degradation through innovative mechanisms like RIBOTACs (ribonuclease-targeting chimeras), selectively eliminating disease-driving RNA transcripts.

Applications span oncology, rare genetic disorders, metabolic diseases, and infectious diseases, offering precision intervention at the molecular level.

Protein Degraders and Modulators

Protein degraders, such as PROTACs (Proteolysis Targeting Chimeras) and molecular glues, have transformed how we approach challenging targets by harnessing the cell’s natural degradation machinery. These approaches:

  • Enable targeted protein degradation by recruiting E3 ligases to unwanted proteins, facilitating their elimination.
  • Provide an alternative to traditional inhibition, allowing dynamic modulation of protein levels rather than permanent binding.
  • Target elusive proteins, such as transcription factors or scaffolding proteins, previously considered “undruggable.”

Our capabilities encompass rational design, synthesis, and screening of small molecules that exploit these mechanisms, ensuring robust discovery and development pipelines.

Comprehensive Support for Next-Generation Modalities

As a Contract Research Organization (CRO), we offer end-to-end support in RNA and protein degrader/modulator development, including:

  • High-throughput screening to identify lead compounds.
  • Advanced biophysical and cellular assays for mechanistic validation.
  • Structure-based design leveraging cutting-edge technologies.
  • In vivo models to evaluate efficacy and safety profiles.

Partner with us to pioneer transformative therapies in RNA and protein modulation. Let’s redefine the boundaries of small-molecule drug discovery together.

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