• Pipeline
  • ASO

Antisense Oligonucleotide (ASO)

ATB-320

Immune-Modulating Anti-Cancer ASO
  • Next-generation TGF-β2-targeting ASO enhanced through chemical modifications, demonstrating improved mRNA binding affinity and plasma stability for cancer treatment.
  • Sequence-specific mRNA binding enables selective suppression of TGF-β2 expression, offering therapeutic potential in tumors with TGF-β2 overexpression while minimizing adverse effects associated with broad TGF-β pathway inhibition.
  • Demonstrated safety in non-clinical studies and cleared to initiate a Phase 1 clinical trial in Australia.

ATB-720

Immune-Modulating ASO for Skin Diseases
  • First-in-class CHI3L1-targeting ASO for inflammatory skin diseases and skin cancer.
  • Discovered using the AI-driven ASODE™ platform, enabling rapid and efficient ASO hit and lead generation.
  • High-permeability topical formulation established, with further optimization in progress.
  • Demonstrated superior efficacy and safety in animal models of atopic dermatitis and psoriasis, outperforming standard-of-care treatments.
  • Engineered for topical delivery to minimize systemic and off-target exposure, particularly in the liver and kidney, often seen with injectable ASOs.

ATB-810

Immune-Modulating ASO for Neurodegenerative Diseases
  • First-in-class CHI3L1-targeting ASO in development for neurodegenerative diseases.
  • Utilizes C-Duplex™ technology, which improves brain distribution of ASOs and extends dosing intervals.
  • Inhibits CHI3L1 mRNA expression and protein secretion in brain cells.
  • Demonstrated reduced CHI3L1 expression and decreased p-TDP-43 levels, a key pathological marker of amyotrophic lateral sclerosis (ALS), in an ALS organoid model.
  • Improved brain distribution of ASOs through C-Duplex™ technology confirmed in animal studies.