We are a genetic medicines company focused on delivering life-changing treatments for people battling devastating diseases.
Using PRISMTM, our proprietary discovery and drug development platform that enables the precise design, optimization and production of novel, stereopure oligonucleotides, we are developing potential medicines to reduce, restore or modulate proteins responsible for genetic diseases.
Explore our scienceRealizing a brighter futurefor patients and families
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 pipelineInnovation by design
Our team shares a commitment to deliver on the promise of genetic medicine for people and families battling devastating diseases. Our clinical programs are assessing investigational compounds for Huntington’s disease, amyotrophic lateral sclerosis, frontotemporal dementia and Duchenne muscular dystrophy.
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