Research into Leber Congenital Amaurosis (LCA) has significant implications in the field of gene therapy. LCA is a rare genetic disorder that causes severe vision loss or blindness from birth due to mutations in genes crucial for retinal function. Gene therapy approaches aim to introduce functional copies of these genes into retinal cells, potentially restoring vision. Luxturna (voretigene neparvovec), an FDA-approved gene therapy, targets RPE65 mutations, a common cause of LCA, and has shown promising results in clinical trials by improving vision in affected individuals. This breakthrough underscores the potential of gene therapy to treat LCA and other genetic retinal disorders, paving the way for innovative treatments in ophthalmology.
Global Leber Congenital Amaurosis Market is expected to grow at a CAGR of 4.8% during the forecast period 2022 to 2029.
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Below are the Top Leber Congenital Amaurosis Companies with a Significant Market Share:
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Rank
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Company
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Overview
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Product Portfolio
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Sales Geographical Coverage
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Developments
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1.
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Spark Therapeutics, Inc.
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Spark Therapeutics is known for developing Luxturna (voretigene neparvovec), the first FDA-approved gene therapy for inherited retinal diseases, including Leber Congenital Amaurosis (LCA). Their focus is on pioneering gene therapies to address genetic disorders, including those affecting the retina, aiming to provide transformative treatments for patients with LCA.
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U.S.
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In July 2017, Spark Therapeutics, Inc submitted the Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for LUXTURNA, the proposed trade name for voretigene neparvovec. This has increased the company’s revenue and product portfolio.
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2.
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Optos
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Optos is a medical technology company specializing in ultra-widefield retinal imaging. Their devices are used in the diagnosis and management of retinal conditions, including LCA. Optos' imaging technologies enable clinicians to capture wide-angle views of the retina, aiding in the early detection and monitoring of retinal diseases such as LCA.
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South America, North America, Europe, Middle East and Africa, and Asia-Pacific
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In October 2019, Optos launched Silverstone, a Combined Ultra-widefield Retinal Imaging Device, and UWF-Guided Swept Source OCT for the Ophthalmic Market. This has increased the company’s sales and product portfolio.
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3.
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CD Genomics
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CD Genomics offers genetic testing services, including next-generation sequencing (NGS) and other molecular diagnostic tools. They play a crucial role in genetic testing for LCA, providing accurate and comprehensive genetic analyses to identify mutations responsible for the disease and aiding in personalized treatment approaches and genetic counseling.
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Europe
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CD Genomics offers comprehensive genetic testing and analysis for Leber Congenital Amaurosis (LCA), a rare and severe inherited retinal dystrophy. It includes identification of genetic mutations in over 30 genes associated with LCA, enabling early diagnosis and informed treatment strategies. Our expert team provides detailed reports and recommendations for genetic counseling and family planning. With CD Genomics, you can gain a deeper understanding of the genetic causes of LCA and make informed decisions for your patients.
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4.
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Blueprint Genetics Oy.
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Blueprint Genetics is a company specializing in genetic diagnostics for rare inherited diseases, including LCA. They provide comprehensive genetic testing panels that include genes associated with LCA, facilitating accurate diagnosis and genetic counseling for patients and their families.
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Europe, North America, and the Middle East
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In May 2021, Blueprint Genetics welcome Spark Therapeutics as a new collaborator to the My Retina Tracker Genetic Testing Program to broaden access for individuals living with an inherited retinal degeneration (IRD). This will help in broadening the sale and engagement of the population on the program
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5.
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LKC TECHNOLOGIES, INC.
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LKC Technologies is a medical device company focused on diagnostic equipment for ophthalmology, including electrophysiology testing. Their devices are used in assessing retinal function, which is crucial for evaluating the progression and severity of retinal diseases such as LCA. LKC Technologies products support clinical trials and research efforts aimed at developing and evaluating new treatments for LCA.
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U.S., Canada, Finland
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In July 2021, LKC Technologies, Inc. announced that the National Health Surveillance Agency (Anvisa) has approved the UTAS device, making it a significant advance in the preservation and improvement of patients' vision in Brazil which has increased their distribution channel
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Conclusion
In the leber congenital amaurosis (LCA) market, companies such as Spark Therapeutics, Optos, CD Genomics, Blueprint Genetics Oy, and LKC Technologies, Inc. play pivotal roles in advancing diagnosis, treatment, and research for this rare genetic disorder. Spark Therapeutics leads with Luxturna, the pioneering gene therapy approved for treating LCA, highlighting the transformative potential of gene therapies in ophthalmology. Optos contributes through advanced retinal imaging technologies crucial for early detection and monitoring of LCA progression. CD Genomics and Blueprint Genetics provide essential genetic testing services, enabling precise diagnosis and personalized treatment strategies. LKC Technologies supports clinical assessments with specialized electrophysiology equipment, facilitating research into new therapeutic interventions. Together, these companies drive innovation and progress in the LCA market, promising improved outcomes and quality of life for affected individuals.
