Leber Congenital Amaurosis (LCA) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
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Leber Congenital Amaurosis (LCA) is the most common cause of inherited blindness in childhood. Its symptoms include vision loss, roving eye movements, deep-set eyes, developmental delay, epilepsy, and motor skill impairment.
The Leber Congenital Amaurosis pipeline market research report provides comprehensive information on the therapeutics under development for Leber Congenital Amaurosis, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history, and the latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Leber Congenital Amaurosis and features dormant and discontinued projects.
Key Targets in the Leber Congenital Amaurosis Pipeline Products Market
The key targets in the Leber Congenital Amaurosis pipeline products market are Centrosomal Protein Of 290 kDa, Retinoid Isomerohydrolase, Aryl Hydrocarbon Interacting Protein Like 1, Cone Rod Homeobox Protein, Inward Rectifier Potassium Channel 13 Inward Rectifier K, Lebercilin, Nicotinamide/Nicotinic Acid Mononucleotide Adenylyltransferase 1, Retinal Guanylyl Cyclase 1, Retinol Dehydrogenase 12, and X Linked Retinitis Pigmentosa GTPase Regulator Interacting Protein 1.
Leber Congenital Amaurosis Pipeline Products Market Analysis by Targets
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Key Mechanisms of Action in the Leber Congenital Amaurosis Pipeline Products Market
The key mechanisms of action in the Leber Congenital Amaurosis pipeline products market are Centrosomal Protein Of 290 kDa Activator, Retinoid Isomerohydrolase Activator, Aryl Hydrocarbon Interacting Protein Like 1 Activator, Cone Rod Homeobox Protein Activator, Inward Rectifier Potassium Channel 13 Inward Rectifier KActivator, Lebercilin Activator, Nicotinamide/Nicotinic Acid Mononucleotide Adenylyltransferase 1 Activator, Retinal Guanylyl Cyclase 1 Inhibitor, Retinol Dehydrogenase 12 Activator, and X Linked Retinitis Pigmentosa GTPase Regulator Interacting Protein 1 Activator.
Leber Congenital Amaurosis Pipeline Products Market Analysis by Mechanisms of Action
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Key Routes of Administration in the Leber Congenital Amaurosis Pipeline Products Market
The key routes of administration in the Leber Congenital Amaurosis pipeline products market are intraocular, intravitreal, and ophthalmic.
Leber Congenital Amaurosis Pipeline Products Market Analysis by Routes of Administration
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Key Molecule Types in the Leber Congenital Amaurosis Pipeline Products Market
The key molecule types in the Leber Congenital Amaurosis pipeline products market are gene therapy, antisense oligonucleotide, antisense RNAi oligonucleotide, and gene-modified cell therapy.
Leber Congenital Amaurosis Pipeline Products Market Analysis by Molecule Types
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Major Companies in the LCA Pipeline Products Market
Some of the major companies in the LCA pipeline products market are MeiraGTx Holdings Plc, Opus Genetics Inc, ProQR Therapeutics NV, Variant, Atsena Therapeutics Inc, Editas Medicine Inc, Generation Bio Co, Hubble Therapeutics Inc, Huida (Shanghai) Biotechnology Co Ltd, and IVERIC bio Inc.
Leber Congenital Amaurosis Pipeline Products Market Analysis by Companies
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Leber Congenital Amaurosis Pipeline Products Market Report Overview
Key Targets | Centrosomal Protein Of 290 kDa, Retinoid Isomerohydrolase, Aryl Hydrocarbon Interacting Protein Like 1, Cone Rod Homeobox Protein, Inward Rectifier Potassium Channel 13 Inward Rectifier K, Lebercilin, Nicotinamide/Nicotinic Acid Mononucleotide Adenylyltransferase 1, Retinal Guanylyl Cyclase 1, Retinol Dehydrogenase 12, and X Linked Retinitis Pigmentosa GTPase Regulator Interacting Protein 1 |
Key Mechanisms of Action | Centrosomal Protein Of 290 kDa Activator, Retinoid Isomerohydrolase Activator, Aryl Hydrocarbon Interacting Protein Like 1 Activator, Cone Rod Homeobox Protein Activator, Inward Rectifier Potassium Channel 13 Inward Rectifier KActivator, Lebercilin Activator, Nicotinamide/Nicotinic Acid Mononucleotide Adenylyltransferase 1 Activator, Retinal Guanylyl Cyclase 1 Inhibitor, Retinol Dehydrogenase 12 Activator, and X Linked Retinitis Pigmentosa GTPase Regulator Interacting Protein 1 Activator |
Key Routes of Administration | Intraocular, Intravitreal, and Ophthalmic |
Key Molecule Type | Gene Therapy, Antisense Oligonucleotide, Antisense RNAi Oligonucleotide, and Gene-Modified Cell Therapy |
Major Companies | MeiraGTx Holdings Plc, Opus Genetics Inc, ProQR Therapeutics NV, Variant, Atsena Therapeutics Inc, Editas Medicine Inc, Generation Bio Co, Hubble Therapeutics Inc, Huida (Shanghai) Biotechnology Co Ltd, and IVERIC bio Inc |
Scope
- The pipeline guide provides a snapshot of the global therapeutic landscape of Leber Congenital Amaurosis (LCA) (Genetic Disorders).
- The pipeline guide reviews pipeline therapeutics for Leber Congenital Amaurosis (LCA) (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Leber Congenital Amaurosis (LCA) (Genetic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Leber Congenital Amaurosis (LCA) (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA), and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews the latest news related to pipeline therapeutics for Leber Congenital Amaurosis (LCA) (Genetic Disorders)
Reasons to Buy
- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolios and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Leber Congenital Amaurosis (LCA) (Genetic Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and their most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Leber Congenital Amaurosis (LCA) (Genetic Disorders) pipeline depth and focus of Indication therapeutics.
- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from the pipeline.
Editas Medicine Inc
Generation Bio Co
Hubble Therapeutics Inc
Huida (Shanghai) Biotechnology Co Ltd
IVERIC bio Inc
LuxFiat Therapeutics LLC
MeiraGTx Holdings Plc
Ocugen Inc
Odylia Therapeutics Inc
Opus Genetics Inc
ProQR Therapeutics NV
Variant
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Frequently asked questions
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What are the key targets in the Leber Congenital Amaurosis products market?
The key targets in the Leber Congenital Amaurosis pipeline products market are Centrosomal Protein Of 290 kDa, Retinoid Isomerohydrolase, Aryl Hydrocarbon Interacting Protein Like 1, Cone Rod Homeobox Protein, Inward Rectifier Potassium Channel 13 Inward Rectifier K, Lebercilin, Nicotinamide/Nicotinic Acid Mononucleotide Adenylyltransferase 1, Retinal Guanylyl Cyclase 1, Retinol Dehydrogenase 12, and X Linked Retinitis Pigmentosa GTPase Regulator Interacting Protein 1.
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What are the key mechanisms of action in the Leber Congenital Amaurosis pipeline products market?
The key mechanisms of action in the Leber Congenital Amaurosis pipeline products market are Centrosomal Protein Of 290 kDa Activator, Retinoid Isomerohydrolase Activator, Aryl Hydrocarbon Interacting Protein Like 1 Activator, Cone Rod Homeobox Protein Activator, Inward Rectifier Potassium Channel 13 Inward Rectifier KActivator, Lebercilin Activator, Nicotinamide/Nicotinic Acid Mononucleotide Adenylyltransferase 1 Activator, Retinal Guanylyl Cyclase 1 Inhibitor, Retinol Dehydrogenase 12 Activator, and X Linked Retinitis Pigmentosa GTPase Regulator Interacting Protein 1 Activator.
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What are the key routes of administration in the LCA pipeline products market?
The key routes of administration in the LCA pipeline products market are intraocular, intravitreal, and ophthalmic.
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What are the key molecule types in the Leber Congenital Amaurosis pipeline products market?
The key molecule types in the Leber Congenital Amaurosis pipeline products market are gene therapy, antisense oligonucleotide, antisense RNAi oligonucleotide, and gene-modified cell therapy.
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Which are the major companies in the LCA pipeline products market?
Some of the major companies in the LCA pipeline products market are MeiraGTx Holdings Plc, Opus Genetics Inc, ProQR Therapeutics NV, Variant, Atsena Therapeutics Inc, Editas Medicine Inc, Generation Bio Co, Hubble Therapeutics Inc, Huida (Shanghai) Biotechnology Co Ltd, and IVERIC bio Inc.
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