Leber congenital amaurosis (LCA) is a disorder of eye mainly affects the retina. Retina is the specific tissue at the back of the eye responsible for detection of light and color. People, primarily babies suffering with this disorder have severe visual impairment from the first year of life. The visual impairment because of this disorder likely to be stable for the rest of the life, whereas is some cases it may worsen very slowly over time. LCA is a congenital disease and usually inherited from the parents where both the parents carry a copy of a recessive gene even though they are normal sighted.
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The prime symptom of LCA includes lack of visual reactions and unusual eye movements which collectively termed as nystagmus, where it can be observed within the first few months of life, the other secondary symptoms include glaucoma, cataract and cornea problems. Recently companies got experimental successes regarding treatment of LCA using gene therapy, but research and development work on this therapy is still in the research phase.
The disease leber congenital amaurosis is very rare and identified at early stage of the life. According to report published by American Association for Pediatric Ophthalmology and Strabismus, approximately 3 cases per 100,000 births have LCA and out of all congenital blindness or severely reduced vision cases in children only 10% are caused by LCA. According to United States Food and Drug Administration, LCA represents more than 5% of all retinal dystrophies.
All these facts represent demand for the various diagnosis and treatment options for leber congenital amaurosis globally in coming future. The technological advancements in diagnosis of LCA and suitable reimbursement circumstances may drive the growth of the market.
On the other hand, high cost involved and limited awareness may hinder the growth of the overall market. Currently there is a gene therapy under clinical trial phase for LCA caused genetic mutation in gene RPE65 whereas the clinical study has been completed by QLT Inc. for LCA Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin: Retinol Acyltransferase (LRAT) Mutations and Nantes University Hospital for genetic study of patients suffering from LCA. According to American Society of Gene and Cell Therapy report in June 2014, Spark Therapeutics is planning to commercialize gene therapies for Leber congenital amaurosis in coning future.
Leber congenital amaurosis market can be segmented according to different categories such as regional geography and market for diagnosis and treatment. Geographically, this market can be segmented in four regions namely North American, Europe, Asia-Pacific and Rest of the World, out of all these segments North America region will highest contribution in terms of value because of more awareness and higher percentage of total income spent on healthcare compared to other economies.
Followed by this, Europe, Asia-Pacific and Rest of the World respectively could be major segments of the market. It is notable that all the companies which are catering in this segment are based out of either in North America or European region, which represents the one sided growth in these particular countries in the near future.
Currently very few established players in biotechnology market which are on the way to develop gene therapy products for leber congenital amaurosis. Out of these companies, QLT, Inc., ReGenX Biosciences, LLC, Applied Genetic Technologies Corporation, BBB technologies BV and Spark Therapeutics, LLC have upper hand in this market.
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