Clinical Trials

Overview

Onasemnogene abeparvovec-xioi (Zolgensma™)

Novartis Gene Therapies

Onasemnogene abeparvovec-xioi, previously AVXS-101 ( Zolgensma™) is a gene therapy developed by Novartis Gene Therapies (previously AveXis), designed to address the genetic root cause of SMA by replacing the function of the missing or faulty SMN1 gene.

Administered during a single, intravenous (IV) infusion, Zolgensma delivers a new working copy of the SMN1 gene into a patient’s cells, halting disease progression.

Method of Action

Zolgensma™ delivers a new, working copy of the human SMN gene inside a vector. The vector carries this gene into cells throughout the body, including motor neurons, where it enables production of functional SMN protein.

Zolgensma™ treatment

In Focus

The Vector

The vector that delivers the SMN gene is made from a virus called adeno-associated virus 9, or AAV9. This type of virus is harmless. To make the vector, the DNA of the virus is removed and the new SMN gene inserted in its place. Vectors are used because they can travel throughout the body and deliver the new, working gene to the cells where it is needed.

When the new gene reaches its destination, it is ready to tell the motor neurons to start making SMN protein. This happens throughout the body, with many vectors delivering a new, working copy of the SMN gene to motor neurons.

Motor neurons now make sufficient SMN protein to survive, function, and be maintained.

More about Zolgensma™

Trials of onasemnogene abeparvovec-xioi/ AVXS-101

Trial Name
SMART
Age:
Up to 17 years old
Aim:
To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 in people with SMA with bi-allelic mutations in the SMN1 gene weighing ≥ 8.5 kg and ≤ 21 kg, over a 12 month period.
Status:
Completed
Locations:
Belgium, France, Portugal, Australia & Canada.
Identifier:
NCT04089566
Trial Name
START
Age:
Child, Adult, Older Adult
Aim:
A long term follow-up safety study of people in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering AVXS-101
Status:
Active, not recruiting
Locations:
USA
Identifier:
NCT03421977
Trial Name
STR1VE
Age:
Up to 180 days old
Aim:
Phase 3, open-label, single-arm, single-dose gene replacement therapy clinical trial for infants with SMA Type 1 with one or two SMN2 copies delivering AVXS-101 by intravenous infusion
Status:
Completed
Locations:
USA
Identifier:
NCT03306277
Trial Name
SPRINT
Age:
Up to 42 days
Aim:
A global study of a single, one-time dose of AVXS-101 delivered to infants with genetically diagnosed and pre-symptomatic SMA with multiple copies of SMN2
Status:
Completed
Locations:
Belgium, Germany,Italy, Spain, UK, Israel, Australia, Canada, Japan, Korea, Taiwan, USA
Identifier:
NCT03505099

Community updates

Related pages & resources