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

Child Smiling

GM1 Gangliosidosis Resources

child smiling

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 Resources

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 Resources

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.

Detect Genetic Testing Program

Sio is a sponsor of the Invitae Detect LSD program, which provides sponsored, no-charge genetic testing and counseling for people suspected of having a lysosomal storage disease (LSD).

This program breaks down many barriers to genetic testing for LSDs. Through the program, individuals suspected of having a lysosomal disease have access to genetic testing and counseling to help bring them one step closer to a diagnosis and the appropriate clinical management.

For more information 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

Scroll to Top