Designed to treat the genetic root cause of SMA. Designed to work continuously to increase the amount of SMN protein
ITVISMA is a gene replacement therapy for people with spinal muscular atrophy (SMA). It delivers a new, working survival motor neuron 1 (SMN1) gene to replace the function of the missing or nonworking SMN1 gene in the body with only one dose.
Here’s how:
The new, working SMN1 gene is placed inside a delivery vehicle called a vector.
This vector delivers the new, working gene to motor neuron cells, enabling continuous production of survival motor neuron (SMN) protein.
By delivering this new, working gene, ITVISMA can stop the progressive loss of motor neurons, which are essential to muscle function.
ITVISMA replaces the function of the SMN1 gene and is designed to continuously produce this essential protein.
ITVISMA is not a cure and can't reverse the damage that SMA has already caused to motor neurons.