Oligonucleotide Therapeutics

Modulate protein expression and splicing with RNA-binding ASOs and RNAi

Submit a project inquiry

Support for the discovery and development of antisense therapeutics

Oligonucleotide therapeutics, including antisense oligonucleotides (ASOs) and RNA interference (RNAi), represent a powerful modality for the treatment of many diseases, particularly genetic disorders associated with aberrant protein production. The most famous example of oligonucleotide-based intervention is in the treatment of the neuromuscular disorder spinal muscular atrophy (SMA), associated with mutations in the SMN1 gene. Treatment with an ASO induces alternate splicing of the SMN2 transcript, leading to the generation of SMN protein that functionally replaces the missing SMN1 product.

The efficacy of this approach has led to continuing interest in this therapeutic modality. Our services can support the selection of optimal ASO/RNAi targets, the analysis of oligo species, and the comprehensive characterization of oligonucleotide treatment effects.

  • Identify targets amenable to ASO/RNAi intervention
  • Validate oligo identity and quality
  • Evaluate the effects of ASO/RNAi treatment on a proteome-wide level (e.g., measure protein level changes and protein synthesis rates, identify off-targets, and unambiguously determine splicing isoforms on protein level)

Explore Other Modalities

We offer support for the development of a broad range of therapeutic modalities.

  • Kinase Inhibitors

    Develop selective and potent protein kinase inhibitors

  • ADCs

    Targeted immunotherapeutics for cancer treatment and beyond

  • Gene Therapies

    Modify genetic content and expression to enable new frontiers of disease treatment

  • Cell Therapies

    Harness the power of cell therapy to overcome unmet disease challenges

  • Radiotherapeutics

    Harness the ultra-sensitive analytic power of ICP-MS for radioligand discovery and development

  • Molecular Glues

    Induce or stabilize protein-protein interactions for therapeutic applications

  • Targeted Degraders

    Induce target degradation with precision and potency

  • Covalent Drugs

    Unlock potent and selective therapeutics through covalent binding

  • DNA- and RNA-Targeting Drugs

    Accelerate the development of DNA- and RNA-targeted small-molecule therapeutics

Submit a Project Inquiry

To connect with our scientific team, submit a project inquiry. Our team will be in touch shortly to schedule an introductory meeting.

Newsletter Signup

Sign up for News & Updates