|
PRODUCT
DESCRIPTION
StarGen is a novel gene-based therapeutic for the treatment of
Stargardt’s disease. The disease is caused by a mutation of the ABCR
gene which leads to the degeneration of photoreceptors in the retina
and vision loss. The product is designed to deliver a corrected
version of the ABCR gene.
INDICATION
Autosomal
recessive Stargardt’s disease is a juvenile-onset macular dystrophy.
Age of onset is typically 8-12 years with severely decreased visual
acuities developing within nine years of onset. Presentation includes
increased blind spots, and reduced ability to adapt to dark after
sunlight exposure. Impact is confined to loss of central vision
analogous to age-related macular degeneration.
MARKET
Stargardt’s
disease is the most common juvenile degenerative retinal disease
with a US and EU population of approx. 50,000 cases and an incidence
of 1/10,000 (600 new cases /year). Based on prevalence, the market
could be US$75 million.
TECHNICAL
DESIGN
A LentiVector
system carrying the normal functional ABCR gene administered to
photoreceptors in the retina.
DEVELOPMENT
STATUS
- Preclinical
efficacy has been demonstrated in an in vivo model of Stargardt’s
disease. Efficacy continued to the end of the experiments at
approximately 9 months
- Further
preclinical development is ongoing at Columbia University with
financial support from the US charity, Foundation Fighting
Blindness
COMMERCIALISATION
STRATEGY
Commercial partners sought to co-develop and
market the product.
PARTNERS/FUNDING
Foundation
Fighting Blindness, USA
Website by College Hill - Life Sciences |
