Patients & Caregivers

Breaking boundaries today.
Building a lifetime of tomorrows.

Our Commitment

At Sio, our mission is simple: to combine cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. We are working tirelessly to advance our rare fatal pediatric and adult neurodegenerative programs into clinical development so that patients can access the therapies they need.

Our team is driven to challenge the status quo in the pursuit of delivering impactful gene therapies to patients in need. We commit to timely, clear, and transparent communications because we understand that every day for patients enduring these diseases is a day that matters. We care deeply about our partnerships with patients, families and patient advocacy groups, and we encourage you to reach out directly with any questions or feedback by contacting us at

Faces of the GM1 community

GM1 Gangliosidosis


We are developing a gene therapy candidate, AXO-AAV-GM1, to be potentially the first approved treatment for GM1 gangliosidosis. Learn more about AXO-AAV-GM1, which is currently in a registrational clinical trial here.

Cure GMI FoundationNational Tay-Sachs & Allied Diseases Association

Tay-Sachs/Sandhoff Disease

We are developing a gene therapy candidate, AXO-AAV-GM2, to be the first approved treatment for Tay-Sachs/Sandhoff Disease. Learn more about AXO-AAV-GM2 here.

Parkinson’s Disease

We are developing a gene therapy candidate, AXO-Lenti-PD, which is designed to replace the dopamine that is lost in the brains of those with Parkinson’s disease. Learn more about AXO-Lenti-PD and its registrational clinical trial here.

Downloadable Resources

Click below to download brochures containing clinical trial information, including details on our investigational gene therapies.

Detect Genetic Testing Program

At Sio, we believe that every child and family should be able to get the care they need. But too often, genetic testing is a barrier to care. That is why Sio is a sponsor of the Invitae Detect LSD program.

The Invitae Detect LSD program provides sponsored, no-charge genetic testing and counseling for children suspected of having a lysosomal storage disease (LSD). By increasing the availability of genetic testing, the program is designed to help bring more patients and families closer to answers, including a diagnosis.

To learn more about the Detect LSD program, click here.

Detect - Lysosomal Storage Diseases

Expanded Access Policy

Expanded access refers to the use of an investigational therapy, which has not yet obtained regulatory approval, for a patient outside of a clinical trial setting. By contrast, the use of investigational therapy in a clinical trial setting involves the collection and assessment of comprehensive safety and efficacy data in clinical trial subjects over a period of time in well controlled matter, generally to support an application for regulatory approval.

At this time, Sio believes that participation in one of its clinical trials is the most appropriate way to access it investigational therapies. Therefore, Sio does not have an expanded access program. If you have questions about participating in our clinical trials, please speak with your medical care team or contact us at

Consistent with the 21st Century Cures Act, Sio reserves the right to revise this policy at any time.

Contact Us

For questions about our ongoing clinical trials or the Detect LSD program, please contact us at

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