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Our Progress

We are committed to ensuring patients and families remain at the heart of our work as we strive to advance potential one-time gene therapies for people living with neurodegenerative diseases. Here we will provide updates on our progress.


From Promise to Practice: Reflections on Our Commitment to Rare Disease Communities in 2023

2023 was a busy and exciting year for Prevail as we continued our mission to develop novel gene therapies for rare and neurodegenerative diseases and provide vital support for patient communities. Striving towards this mission would be impossible without the support of our exceptional patient advocacy partners. We deeply appreciate the invaluable work of patient…


Prevail Therapeutics features on Behind the Mystery, a recurring series on The Balancing Act devoted to advocating for rare and genetic diseases.

The episode, “Behind the Mystery of Gaucher Disease,” was launched on October 02, 2023. This ground-breaking episode unites experts from patient advocacy, healthcare, and industry, alongside someone living with Gaucher disease who has an inspiring story, offering hope to the patient community.


Prevail partners with the International Gaucher Alliance to sponsor the GARDIAN Registry for Gaucher disease

Prevail is proud to sponsor the International Gaucher Alliance’s GARDIAN registry for people and families affected by neuronopathic Gaucher disease (nGD), a resource built to help advance research for new treatment options by tracking personal experiences with this diagnosis.


Reflecting on our commitment to the communities we serve

As we enter a new year, Prevail is reflecting on our 2021-2022 community collaborations that fostered important conversations as we continued our commitment toward new and improved treatments for rare diseases.


PROCEED trial evaluating PR001 for GD1 announced

At Prevail, our team strives to develop disease-modifying gene therapies for patients with neurodegenerative diseases. Learn about our PROCEED trial which will evaluate PR001 for Type 1 Gaucher disease (GD1).

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