Arexera Therapeutics, Inc.
Arexera Therapeutics is a clinical-stage biotechnology company developing RNA interference (siRNA) medicines for severe neurological diseases. The company develops divalent and bispecific siRNA architectures intended to enable broad CNS biodistribution, robust target knockdown and prolonged durability, and is advancing a pipeline beginning with Huntington’s disease (HTT- and MSH3-targeted programs) toward IND-enabling studies and first-in-human trials.
Industries
Nr. of Employees
medium (51-250)
Arexera Therapeutics, Inc.
Products
ATL-101 (HTT-targeting di-siRNA)
Investigational di-siRNA therapeutic designed to reduce huntingtin (HTT) expression across CNS regions affected by Huntington’s disease; advanced through IND-enabling studies with plans to initiate Phase 1.
ARX-MSH3 (MSH3-targeting di-siRNA)
Investigational di-siRNA program targeting MSH3, a genetic modifier associated with somatic CAG repeat expansion and disease progression in Huntington’s disease; being advanced in parallel to HTT-lowering approaches.
ARX-MAPT (MAPT-targeting program)
Discovery-stage siRNA program targeting MAPT for tauopathies.
ARX-SNCA (SNCA-targeting program)
Discovery-stage siRNA program targeting SNCA for synucleinopathies.
Next-generation bispecific siRNA platform and Brain Shuttle concepts
Platform R&D focused on bispecific siRNA therapeutics (bi-siRNA) and next-generation delivery concepts to expand multi-target CNS therapies.
ATL-101 (HTT-targeting di-siRNA)
Investigational di-siRNA therapeutic designed to reduce huntingtin (HTT) expression across CNS regions affected by Huntington’s disease; advanced through IND-enabling studies with plans to initiate Phase 1.
ARX-MSH3 (MSH3-targeting di-siRNA)
Investigational di-siRNA program targeting MSH3, a genetic modifier associated with somatic CAG repeat expansion and disease progression in Huntington’s disease; being advanced in parallel to HTT-lowering approaches.
ARX-MAPT (MAPT-targeting program)
Discovery-stage siRNA program targeting MAPT for tauopathies.
ARX-SNCA (SNCA-targeting program)
Discovery-stage siRNA program targeting SNCA for synucleinopathies.
Next-generation bispecific siRNA platform and Brain Shuttle concepts
Platform R&D focused on bispecific siRNA therapeutics (bi-siRNA) and next-generation delivery concepts to expand multi-target CNS therapies.
Services
Strategic collaborations to co-develop CNS-targeted siRNA programs with biopharma partners.
Strategic collaborations to co-develop CNS-targeted siRNA programs with biopharma partners.
Expertise Areas
- siRNA therapeutics
- CNS drug delivery and biodistribution
- Neurodegenerative disease drug development
- Preclinical safety and efficacy studies
Key Technologies
- Divalent siRNA (di-siRNA)
- Bispecific siRNA (bi-siRNA)
- RNA interference (RNAi)
- Deep brain delivery platforms
News & Updates
Press release describing the company rebrand, platform capabilities (di-siRNA and bi-siRNA), preclinical ATL-101 data, plans to initiate Phase 1 by end of 2026, and strategic overview.
Company reports having raised approximately $150 million to advance its siRNA platform and pipeline.
Press release describing the company rebrand, platform capabilities (di-siRNA and bi-siRNA), preclinical ATL-101 data, plans to initiate Phase 1 by end of 2026, and strategic overview.
Company reports having raised approximately $150 million to advance its siRNA platform and pipeline.