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 Appoints Gene Therapy Pioneer Guangping Gao, Ph.D., as Chief AAV Scientific Advisor

Read more


Sio Gene Therapies Announces Corporate Updates and Financial Results for Second Fiscal Quarter Ended September 30, 2020

Read more


Axovant Gene Therapies Announces Name Change to Sio Gene Therapies

Read more
Scroll to Top