Innovation focused on inherited retinal diseases
LuxFiat Therapeutics has a portfolio of potential gene therapies advancing to clinical trials for the treatment of inherited retinal diseases with unmet medical need.
Fueled by compelling proof of concept data generated at the University of Pennsylvania and having developed state of the art translational models, LuxFiat is advancing its pipeline to achieve transformational outcomes for patients with inherited blindness.
Fueled by compelling proof of concept data generated at the University of Pennsylvania and having developed state of the art translational models, LuxFiat is advancing its pipeline to achieve transformational outcomes for patients with inherited blindness.
How retinal gene therapies work
Retinal gene therapies are intended to work by delivering a normal copy of a gene directly into retinal cells, which may address deficits caused by a mutant gene. Alternatively, they silence a disease causing gene whose normal function damages the cell and replace it with a normal copy. Viral vectors are used to deliver the gene therapies due to their capability of delivering many copies of potentially therapeutic genes to the target cells. Application of gene therapies for retinal diseases is rapidly accelerating now that full mapping of the human genome has been achieved.
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Why some inherited retinal diseases cause blindness
Inherited Retinal Diseases (IRDs), or hereditary retinal dystrophies, are a diverse group of genetic disorders that can cause severe vision loss and even blindness. Together they have an estimated prevalence of 1:2,000 and are the leading cause of vision loss in people between 15 and 45 years of age. Disease can begin at birth (congenital) or later in life and may be stable (such as congenital stationary night blindness) or progress (such as retinitis pigmentosa).
Mutations in more than 270 genes are known to affect the structure and/or the function of key cell populations in the retina that include: photoreceptors (rods and cones), retinal pigment epithelial (RPE) cells, and bipolar cells. Dysfunction or loss of these cells prevents proper processing of the visual information that originates in the retina and is carried to the visual cortex of the brain
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