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PRODUCT
DESCRIPTION
RetinoStat is a gene-based product that is designed to halt the aberrant blood vessel growth that causes age-related macular degeneration and proliferative diabetic retinopathy.
The product is comprised of a LentiVector®
system expressing angiostatin and endostatin genes. The Company has an exclusive license to the angiostatics, endostatin and angiostatin, for the treatment of vision-loss via localised gene delivery. The LentiVector technology preferentially targets and delivers the therapeutic gene using a tissue-specific switch (VMD2) to target gene expression to the retinal pigment epithelial cells.
INDICATION
Age-related
macular degeneration (AMD) and proliferative diabetic retinopathy
(PDR).
MARKET
Together, AMD
and PDR are the leading causes of vision loss in the developed world.
Estimate of market size is ~ US$1 billion.
TECHNICAL
DESIGN
A LentiVector
system delivering genes encoding anti-angiogenic proteins.
DEVELOPMENT
STATUS
- Preclinical data from an industry standard in vivo model of neovascular AMD have shown statistically significant efficacy
- Treatment
with RetinoStat was safe and well tolerated, reduced the area
of the
eye with abnormal blood vessel growth and
reduced
blood vessel leakage in the eye, which leads to the distortion
and loss of central vision in AMD
- Further preclinical
studies are being conducted in the USA with Johns Hopkins University
and financial support from the US charity,
Foundation Fighting Blindness
- Additional
preclinical data have shown that RetinoStat achieves specific
gene expression in target
cells of the retina
- The manufacturing
process is being optimised for clinical material and preparations
are underway for a submission
to start trials
COMMERCIALISATION STRATEGY
Commercial partners
sought to co-develop and market the product.
PARTNERS/FUNDING
Foundation Fighting
Blindness, USA
Institute
of Ophthalmology, UK
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