PRODUCT DESCRIPTION
SMN-1G is a specific treatment for spinal muscular atrophy (SMA), which restores levels of survival motor neuron (SMN) protein by delivering the corrected version of the SMN1 gene using the LentiVector system. Deficiency in SMN protein has a severe effect on motor neurons, leading to muscle deficiency throughout the body.

INDICATION
Spinal muscular atrophy, a motor neuron disease.

MARKET
SMA is one of the most common inherited causes of death in childhood. The incidence of SMA is one in every 6,000-10,000 live births, similar to Duchenne muscular dystrophy.

TECHNICAL DESIGN
LentiVector delivering the SMN1 gene.

DEVELOPMENT STATUS

• Preclinical efficacy has been demonstrated in an industry standard in vivo model of SMA
• Further preclinical studies are ongoing to optimise the SMN-1G product configuration
• A clinical development strategy is currently being planned

COMMERCIALISATION STRATEGY
Commercial partners sought to co-develop and market the product.

PARTNERS/FUNDING
FightSMA, USA
The Ohio State University, USA

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