Leber Congenital Amaurosis Market Is Expected To Grow During The Forecast Period (2019-2032), DelveInsight | Major Companies- ProQR Therapeutics, Editas Medicine, and Several Others

DelveInsight's LEBER CONGENITAL AMAUROSIS Market Insights, Epidemiology, and Market Forecast 2032" report delivers an in-depth understanding of the disease, historical and forecasted epidemiology as well as the LEBER CONGENITAL AMAUROSIS Market Size and Share in the 7MM (i.e. the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan).

The LEBER CONGENITAL AMAUROSIS Market report covers emerging drugs, current treatment practices, market share of individual therapies, and the current forecasted market size from 2019 to 2032. It also evaluates the current treatment practice/algorithm, market drivers & barriers, and unmet medical needs to curate the best opportunities and assess the underlying potential of the market.

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LEBER CONGENITAL AMAUROSIS: An Overview
Leber Congenital Amaurosis (LCA) is a rare genetic eye disorder. Affected infants are often blind at birth. Other symptoms may include crossed eyes (strabismus); rapid, involuntary eye movements (nystagmus); unusual sensitivity to light (photophobia); clouding of the lenses of the eyes (cataracts); and/or a cone shape to the front of the eye (keratoconus). LCA is usually inherited as an autosomal recessive genetic condition. Children born with LCA have light-gathering cells (rods and cones) of the retina that do not function properly. Absence or reduction of the electrical activity of the retina is always observed and is necessary for the diagnosis of LCA. A decrease in visual responsiveness at birth is the first sign of the disease. Often the child will poke, press and rub the eyes to stimulate the retina to produce light (Franceschetti's oculo-digital sign).This activity may cause the eyes to become sunken or deep set (enophthalmos). LCA is a monogenic disease and at least 27 genes are implicated. Changes (mutations) in these genes can account for about 80-90% of diagnosed cases of LCA. The genes responsible for the remaining 10-20% of diagnoses are not known. LCA is usually inherited as an autosomal recessive genetic condition.

LEBER CONGENITAL AMAUROSIS Market
The LEBER CONGENITAL AMAUROSIS market size is anticipated to increase during the study period owing to the increasing incident population and rising awareness of the LEBER CONGENITAL AMAUROSIS Market in the 7MM. The expected launch of emerging therapies and the research and development activities of pharmaceutical companies will also fuel the market growth during the forecast period.
The market outlook section of the report helps to build a detailed comprehension of the historic, current, and forecasted LEBER CONGENITAL AMAUROSIS Market size by analyzing the impact of current and emerging therapies in the market. It also provides a detailed assessment of the market drivers & barriers, unmet needs, and emerging technologies.
The report gives a thorough detail of the LEBER CONGENITAL AMAUROSIS Market trend for each marketed drug and late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action (MoA), competition with other therapies, brand value, their impact on the market and view of the key opinion leaders.

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LEBER CONGENITAL AMAUROSIS Emerging Drugs

Sepofarsen (QR-110): ProQR Therapeutics
Sepofarsen (QR-110) is an RNA therapy that aims to restore vision in Leber Congenital Amaurosis 10 (LCA10) due to the most common p.Cys998X mutation in the CEP290 gene. Sepofarsen aims to restore vision loss in people with Leber Congenital Amaurosis due to a specific mutation in the CEP290 gene. This p.Cys998X mutation, also known as c.2991+1655A>G, is the most common mutation causing LCA10. The p.Cys998X mutation causes a mistake in the CEP290 RNA and therefore a process called splicing is not done properly. The cell can therefore not use the RNA to produce a working CEP290 protein that is essential for vision. Currently, it is in Phase II/III stage of development to treat Leber Congenital Amaurosis.

EDIT-101: Editas Medicine, Inc.
The company's approach to Leber Congenital Amaurosis 10 (LCA10) is to target a disease-causing mutation in the CEP290 gene that causes degeneration in ocular photoreceptor cells-cells critical for experiencing normal vision. They have developed an experimental CRISPR medicine called EDIT-101 designed to remove the CEP290 mutation. This approach may restore normal protein expression, photoreceptor function, and ultimately, vision.

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LEBER CONGENITAL AMAUROSIS Market Outlook
The Leber Congenital Amaurosis market outlook of the report helps to build a detailed comprehension of the historic, current, and forecasted Leber Congenital Amaurosis market trends by analyzing the impact of current Leber Congenital Amaurosis therapies on the market, unmet needs, drivers and barriers, and demand for better technology. This segment gives a thorough detail of Leber Congenital Amaurosis market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria's, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated Leber Congenital Amaurosis market data are presented with relevant tables and graphs to give a clear view of the market at first sight

LEBER CONGENITAL AMAUROSIS Epidemiology
The epidemiology section covers insights into the historical and current LEBER CONGENITAL AMAUROSIS Market patient pool and forecasted trends for every seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides the diagnosed patient pool and their trends along with assumptions undertaken.

LEBER CONGENITAL AMAUROSIS Market Drugs & Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the LEBER CONGENITAL AMAUROSIS Market or expected to get launched in the market during the study period. The analysis covers LEBER CONGENITAL AMAUROSIS market uptake by drugs; patient uptake by therapies; and sales of each drug.

Report's Drugs Uptake section helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allow the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

The report also covers the LEBER CONGENITAL AMAUROSIS Market Pipeline Development Activities and provides valuable insights about different therapeutic candidates in Phase II, and Phase III stages and the key companies in the market involved in developing targeted therapeutics. It also analyses the recent Development such as collaborations, acquisitions, and mergers, licensing patent details, and other information for emerging therapies. Unmet needs in the LEBER CONGENITAL AMAUROSIS include therapies that are oral, anthracycline sparing and deliverable in resource-limited settings. Another major unmet need is the relapse and refractory cases in the patients given treatment with Doxil.

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LEBER CONGENITAL AMAUROSIS Companies
• ProQR Therapeutics
• Editas Medicine
• Coave Therapeutics
• MeiraGTx
• Novelion Therapeutics
• Applied Genetic Technologies Corporation
• Oxford BioMedica
• IVERIC bio, and Several Others

Table of content

• Key Insights
• Executive Summary
• Competitive Intelligence Analysis
• Market Overview at a Glance
• Disease Background and Overview
• Patient Journey
• Epidemiology and Patient Population
• Treatment Algorithm, Current Treatment, and Medical Practices
• Unmet Needs
• Key Endpoints of LEBER CONGENITAL

AMAUROSIS Market Treatment
• Marketed Products
• Emerging Therapies
• Seven Major Market Analysis
• Attribute analysis
• 7MM: Market Outlook
• Access and Reimbursement Overview
• KOL Views
• Market Drivers
• Market Barriers
• Appendix
• DelveInsight Capabilities
• Disclaimer
• About DelveInsight

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