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 to Sell Arvelle Stake as Part of Angelini Pharma’s Acquisition of Arvelle Therapeutics

Read more


Sio Gene Therapies Announces Positive Six-Month Follow-Up Data from Low-Dose Cohort of Phase 1/2 Trial of AXO-AAV-GM1 for GM1 Gangliosidosis

Read more


Sio Gene Therapies Announces First Patient Dosed in High-Dose Cohort of AXO-AAV-GM1 Clinical Trial in Patients with GM1 Gangliosidosis

Read more
Scroll to Top