Novartis announces plans for new Zolgensma gene therapy trial for SMA

Published Date
Research Team
Scientist observing a DNA molecule

Last month the pharmaceutical company Novartis announced plans to start a new, international clinical trial of the spinal muscular atrophy (SMA) gene therapy Zolgensma.

Zolgensma, also known as onasemnogene abeparvovec, addresses the genetic cause of SMA by delivering a functional copy of the SMN1 gene into nerve cells. This gene is needed to make a protein called the survival motor neuron (SMN) protein, which is critical for the function of the nerves that control muscles.

The phase 3b clinical trial, called SMART, will investigate the safety and efficacy of the treatment in young children with SMA who weigh between 8.5kg and 21kg.

The study aims to enroll 24 participants at global sites including in Europe, North America, Australia and Taiwan, and will follow participants over 12 months. The study locations are still being finalised and it is expected that a small number of children will be recruited in each country. Recruitment will be led by each site and is expected to begin in September 2021. More information about the trial, including inclusion criteria, can be found here.

The SMART study will produce new clinical data to expand the clinical evidence on the use of Zolgensma beyond the population studied in clinical trials to date. It will supplement emerging real-world evidence on the use of Zolgensma in babies and young children up to 21kg.

For more information read Novartis’ press release.

If you have any research questions, please contact our Research Line on

Keep in touch