We develop product candidates from proprietary AAV Capsids with high potency, specificity and manufacturability. By screening tens of millions of novel capsid variants, we’re seeking to advance the standard for precision gene therapy.
“I really hope we can find a way to slow down this disease. No parent wants to see their kid go through endless hospital visits, the pain of treatments, or spend their days in a hospital bed. We just want more happy, normal days at home, more laughter, and less tears—more time being a family without the constant stress of medical issues.”
“Huntngton's Disease remains a devastating, incurable genetic brain disease, for which we desperately need effective interventions. A breakthrough would be transformative for affected patients and families worldwide.”
