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PRODUCT
DESCRIPTION
ReQuinate
is a novel gene-based therapeutic for the treatment of haemophilia.
Current treatment is intravenous injection of Factor VIII either
on demand or prophylactically. However, this presents a number
of
problems and risks that could be addressed by a gene therapy product.
ReQuinate has the potential to cure this disease with infrequent
administration via insertion of the correct gene into long-lived
cells in the recipient.
INDICATION
Haemophilia
A, a bleeding disorder resulting from a deficiency in functional
coagulation Factor VIII.
MARKET
Haemophilia
A occurs in 1:5000 males worldwide. In North America there are
approximately
20,000 people with the disease. The current market is dominated
by recombinant Factor VIII products, which eliminate the risk
of
contamination associated with blood-derived products, but are significantly
more expensive.
TECHNICAL
DESIGN
A LentiVector
system carrying the Factor VIII gene administered to the
liver via intravenous injection.
DEVELOPMENT
STATUS
- Preclinical
in vitro data showed that ReQuinate produced potentially therapeutic
levels of the Factor VIII protein in liver cells
- Further
preclinical development is ongoing with financial support from
the UK Department
of Health
COMMERCIALISATION
STRATEGY
Commercial partners
sought to co-develop and market the product.
PARTNERS/FUNDING
Department of
Health, UK
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