Durable medicines at scale to treat common diseases.

Until now, durable medicines have reached only those affected by rare diseases because scaling them for common diseases has been too costly and complex. By integrating large structured datasets, industrial-scale man­u­fac­tur­ing infra­struc­ture, and world-class subject-matter expertise, we have collapsed the cost and scale barriers that have his­tor­i­cal­ly kept common diseases out of reach of trans­for­ma­tion­al therapies. 

Our clinical stage pipeline includes five inves­ti­ga­tion­al medicines, rep­re­sent­ing the output to date of our integrated product engine. Each one is designed to leverage validated biology to address diseases that affect millions of patients, delivering DNA directly to target cells that become a durable source of therapeutic protein following a single dose.

Our next wave of inves­ti­ga­tion­al medicines adheres to our core development principles: leveraging validated biology and focal delivery to address prevalent diseases. Each new program starts further ahead than the last – building on the data, expertise, and infra­struc­ture that contributed to each program that came before. We are committed to a future where durable medicines are the standard of care.