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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
Website by College Hill - Life Sciences |
