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

Fulfilling the promise of genetic medicine by developing transformative therapies for people with devastating CNS diseases.

Learn more about our company

Advancing Lead Clinical Programs in FTD and GM1

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Our Phase 1/2 Study in Frontotemporal Dementia (FTD)

The upliFT-D study is a multinational Phase 1/2 clinical trial evaluating the safety and efficacy of PBFT02, a novel and potentially transformative gene therapy for patients with FTD with progranulin gene mutations.

Learn about PBFT02

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Our Phase 1/2 Study in GM1 Gangliosidosis (GM1)

The Imagine-1 study is a multinational Phase 1/2 clinical trial evaluating the safety and efficacy of PBGM01, a novel and potentially transformative gene therapy for patients with infantile GM1 gangliosidosis.

Learn about PBGM01

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Transforming the Lives of People Affected by CNS Diseases

People living or born with CNS diseases 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.

Learn about our commitment