Architects
We are a biotechnology company focused on delivering transformational therapies for patients with serious, genetically defined diseases.
Our proprietary chemistry platform enables the precise design, optimization and production of stereopure oligonucleotides: molecules optimized to potentially deliver greater efficacy, safety and tolerability than stereorandom oligonucleotides. Through the eyes of our world-leading scientists, we see the future of medicine.
Explore our scienceArchitects
of transformation
Vast potential impact
We are creating a powerful genetic toolbox with the potential to impact many serious conditions for which there are currently few or no treatment options.
Discover our pipelineCommitment is our bridge to innovation
Our team shares a commitment to deliver on the promise of precision medicine for the many patients and families battling devastating diseases. These include clinical programs for Huntington’s disease, amyotrophic lateral sclerosis and frontotemporal dementia.
Our lead programsPatients and families
Learn about our commitment to patients and families and access information on our research programs.
Learn morePatients and families
Learn about our commitment to patients and families and access information on our research programs.
Learn more