Advancing the practice of genetic medicine
Toward more
life-changing therapies
By leveraging the power of getting just the right gene expression, packaged and delivered to the body in just the right way, we can develop highly effective genetic medicines for more people.
How genetic medicines can alter the course of diseases—and people’s lives.
Finding opportunities to set new standards of care
The potential of gene therapy isn’t limited to genetic diseases. Many conditions that result from deficient or missing proteins in the body’s cells could benefit from genetic medicines that help improve protein production.
That’s why we’re looking beyond genetic conditions to severe, chronic diseases where the consistent delivery of a therapeutic protein is likely to improve outcomes—beyond what’s possible with current treatments.
To not only provide the convenience of a one-time gene therapy infusion but also set a new standard of care for patients who need it most.
Elevating our product candidates
By optimizing every component of our product candidates, we’re designing therapies that are tailored to a disease, where every aspect of the therapy works together to potentially realize improved clinical outcomes.
The adeno-associated virus (AAV) is a proven capsid, able to deliver genetic material to targeted cells with a single infusion.
We’re tapping into this potential, developing AAV capsids with improved tissue targeting, more efficient transfers of genetic material, and reduced immunogenicity to ensure optimal safety and efficacy.
The packaged genetic material inside the capsid is the transgene, a piece of DNA that enters the cells to enable the production of the proteins of interest.
We’re working on engineering the proteins we wish to deliver by optimizing exposure. This can mean increasing half-life, stability, activity, and targeting of the therapeutic protein, ensuring that it provides the desired therapeutic benefit.
Optimized and selective promoters and gene sequences ensure the genetic material is expressed in the right way and in the right tissues and cells.
We’re designing our promoters and gene sequences to improve tissue selectivity and to advance the synthesis of the therapeutic protein, allowing for greater benefit at lower doses.
Producing our therapies
Early in the research and development process, we have an eye on manufacturing, with a robust process to produce our products at scale.
Our in-house labs have advanced chemistry, manufacturing, and control (CMC) capabilities for preclinical drug development.
For our clinical and commercial supplies, we partner with contract development and manufacturing organizations (CDMOs) with quality systems compliant with FDA and EMA requirements.
Doing more to impact more lives
As we advance our rare disease programs, we’re expanding our focus to more widespread chronic conditions to help us bring the promise of gene therapy to more people around the world.
Learn more about our programs, pipeline, and results.
Partner with us
Join us in creating the next generation of genetic medicine. Contact us about collaboration opportunities to build on our breakthroughs and advance life-changing therapies.