Sio Gene Therapies Announces Reorganization of R&D Group Read more

Breaking the Boundaries of Gene Therapy

At Sio, we operate with a sense of urgency to develop and deliver gene therapies that transform the treatment of serious neurodegenerative diseases.

Our aim is to combine cutting-edge science with bold imagination to fill unmet medical needs for patients with one-time therapies that deliver lifelong benefits.


Rare fatal pediatric diseases:

Sio is developing AXO-AAV-GM1 and AXO-AAV-GM2 as the first potential treatments for rare and fatal pediatric diseases of GM1 gangliosidosis and GM2 gangliosidosis (also known as Tay-Sachs/Sandhoff disease).

Adult neurodegenerative diseases:

Sio is developing AXO-Lenti-PD, the only in vivo lentiviral gene therapy in development for Parkinson’s disease.

Patients & Caregivers

Patients and families are our priority. We will work as partners to enhance their well-being and quality of life through the tight collaboration between patients, healthcare providers, and our team.

patients and caregivers

Breaking boundaries today.
Building a lifetime of tomorrows.



Sio Gene Therapies Announces Reorganization of R&D Group

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Sio Gene Therapies Announces Granting of FDA Fast Track Designation for Investigational AXO-AAV-GM1 (AAV9-GLB1) Gene Therapy in Patients with GM1 Gangliosidosis

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Sio Gene Therapies Announces Positive Interim Safety and Biomarker Data from Ongoing Phase 1/2 Clinical Study of AXO-AAV-GM1 Gene Therapy in GM1 Gangliosidosis

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