Onasemnogene abeparvovec (brand name Zolgensma) is a gene therapy drug used to treat spinal muscular atrophy (SMA). It is an adeno-associated virus (AAV9) vector-based gene therapy that delivers a functional copy of the SMN1 gene to the patient's cells. It is the first gene therapy approved by the US Food and Drug Administration (FDA) to treat SMA. It is administered as a one-time intravenous (IV) infusion and is given to children under 2 years of age.
Onasemnogene abeparvovec, also known by the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy (SMA) [3]. SMA is a genetic disease that causes muscle weakness and wasting, primarily affecting young children.
Here's a breakdown of how Onasemnogene abeparvovec works:
- Targets the Root Cause: Unlike medications that manage symptoms, this gene therapy targets the underlying cause of SMA. SMA is caused by a mutation in the survival motor neuron 1 (SMN1) gene, which is responsible for producing a protein essential for motor neuron function.
- Delivers a Working Gene: Onasemnogene abeparvovec delivers a functional copy of the SMN1 gene into a person's motor neuron cells. This is achieved through a vector, which is a modified virus that carries the new gene.
- Increased Protein Production: Once inside the cells, the new gene instructs the cells to produce the missing SMN protein. This helps improve muscle function and development.
Key points about Onasemnogene abeparvovec:
- One-Time Treatment: This gene therapy is a single intravenous infusion.
- Age Limit: It is currently approved for use in children under 2 years of age with SMA.
- Effectiveness: Studies have shown improved motor function and survival rates in treated infants with SMA [2].