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unknown.

The power of gene therapy can change more than words.
It can transform the future.

Our Company Our Science Patients and Caregivers

Fulfilling the promise of gene therapy using a distinctive research and development model to transform the lives of patients with central nervous system (CNS) disorders

Fulfilling the promise of gene therapy

The potential of gene therapy is unlimited, but potential doesn’t save lives—medicines do. Our focus is on producing medicines that will transform the lives of patients and their families.

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A groundbreaking research and development model

Through our partnership with the University of Pennsylvania’s Gene Therapy Program (GTP), we have enhanced access to a wealth of cutting-edge science, expertise, and resources. We are well positioned to turn research into real treatments.

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Transforming the lives of patients

Patients are at the center of every decision we make. We strive to turn disbelief into belief, disease into ease, and the incurable into the curable. That’s what it means to transform lives.

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for patients and families

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