What We Bring to People Affected by CNS Diseases
A bold vision to fulfill the promise of gene therapy by delivering life-transforming medicines that provide a cure for previously incurable diseases.
A differentiated approach to gene therapy development through our innovative research partnership with the world-renowned University of Pennsylvania’s Gene Therapy Program (GTP) and Orphan Disease Center.
A focused pipeline of optimized gene therapies for CNS disorders, including two clinical-stage programs focused on the following devastating diseases:
Our Critical Core Values
We are driven to deliver on our vision by core values that guide our everyday work.
We:
- Put patients at the center of every decision
- Apply leading-edge science, based on a commitment to excellence
- Make an impact that will transform the lives of patients
- Act with integrity and make decisions based on what is right
- Succeed together because we are all part of the solution
Our Commitment to the Community
Building lasting relationships with the communities we serve is a foundational element of our corporate mission. Our commitment includes the following activities:
- Facilitate improved and earlier patient identification
- Increase access to resources such as genetic testing and counseling
- Amplify the voices of community members
- Partner with advocacy groups on fundraising efforts and initiatives