VY-SMN101

VY-SMN101 is an investigational gene therapy developed for the treatment of **spinal muscular atrophy** (SMA). It is designed as an adeno-associated virus (AAV)-mediated gene replacement therapy targeting the **SMN1 gene** to restore normal levels of survival motor neuron (SMN) protein, which is deficient in patients with SMA. The therapy seeks to deliver a functional copy of the SMN1 gene to motor neurons, aiming for long-term correction of the genetic defect underlying the disease. VY-SMN101 was developed by **Voyager Therapeutics** as part of its gene therapy pipeline addressing monogenic neurological disorders[13][5].