Leber Congenital Amaurosis Detailed Pipeline Analysis for H1 2017


Published on : May 15, 2017

Albany, New York, May 15, 2017: Leber Congenital Amaurosis (LCA) is a genetic disorder that is detected at the time of birth or in the first few months. Market Research Hub (MRH) has recently added an analyzed research titled as “Leber Congenital Amaurosis - Pipeline Review, H1 2017” to its vast database. The report provides an all-inclusive information of the LCA therapeutics under development with comprehensive analysis by stage of development, mechanism of action, drug target, route of administration and molecule type.

Leber Congenital Amaurosis is genetic eye disorder which affects the retina that is the specialized tissue at the back of the eye for detecting light and color. Being the main cause of inherited blindness in childhood, its treatment, knowledge and various therapeutic development are of immense importance. The various symptoms that signify LCA include vision loss, roving eye movements, deep-set eyes, developmental delay, epilepsy, and motor skill impairment. The analyzed genetic problem is also associated with other vision problems that comprise involuntary movements of the eyes (nystagmus), increased sensitivity to light (photophobia), and extreme farsightedness (hyperopia). The pupils, which usually expand and contract in response to the amount of light entering the eye, do not react normally to light in the case of LCA rather they expand and contract more slowly than normal, or they may not respond to light at all.

The detailed report starts with the Leber Congenital Amaurosis (LCA) overview, therapeutics development and ongoing clinical trials. Further, the pipeline overview, pipeline by companies & universities/institutes and product under development by them are also listed. The LCA therapeutic assessment is done by the target, mechanism of action, route of administration, and molecule type. The report has also listed the companies involved in the therapeutic development of LCA, the top players included are AmpliPhi Biosciences Corp, Editas Medicine Inc., Novelion Therapeutics Inc., ProQR Therapeutics NV and Spark Therapeutics Inc.

Moving further, the drugs for Leber Congenital Amaurosis (LCA) are also profiled in the study with detailed product description, mechanism of action and R&D progress. The main drugs described are Gene Therapy to Activate Retinal Guanylyl Cyclase-1 for Leber Congenital Amaurosis-1, Gene Therapy to Activate Retinoid Isomerohydrolase for Leber Congenital Amaurosis, Gene Therapy to Activate RPE65 for Leber Congenital Amaurosis, HORARPE-65, QR-110, voretigene neparvovec, and zuretinol acetate. Later, the last segment of the report describes the LCA dormant projects and product development milestone. The featured news and press releases are also linked with the report that gives detail instinct on the Leber Congenital Amaurosis latest news and therapeutics developments at the global level.

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