Batten Disease Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
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Batten disease, a rare genetic disorder, belongs to a group of progressive degenerative neurometabolic disorders known as neuronal ceroid lipofuscinoses. Symptoms include vision loss, lack of muscle coordination, mental retardation or decreasing mental function, emotional disturbances, seizures, muscle spasms, deterioration of muscle tone, and movement problems.
The Batten Disease Drugs in Development market research report provides an overview of the Batten Disease pipeline landscape. The report provides comprehensive information on the therapeutics under development for Batten Disease, 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 Batten Disease and features dormant and discontinued projects.
Batten Disease Pipeline Drugs Market Targets
Some of the targets of the Batten Disease pipeline drugs market are Palmitoyl Protein Thioesterase 1, Tripeptidyl Peptidase 1, Peroxisome Proliferator Activated Receptor Alpha, Battenin, Ceroid Lipofuscinosis Neuronal Protein 6, Polyadenylate Binding Protein 2 (Poly(A) Binding Protein 2 or Nuclear Poly, Retinoic Acid Receptor RXR Alpha, Cell Division Control Protein 42 Homolog, Dihydropyrimidinase Related Protein 2, and Galactocerebrosidase.
Batten Disease pipeline drugs market, by targets
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Mechanisms of Action of Batten Disease Pipeline Drugs Market
Some of the mechanisms of action of the Batten Disease pipeline drugs market are Palmitoyl Protein Thioesterase 1 Activator, Tripeptidyl Peptidase 1 Activator, Peroxisome Proliferator Activated Receptor Alpha Agonist, Battenin Activator, Ceroid Lipofuscinosis Neuronal Protein 6 Activator, Polyadenylate Binding Protein 2 (Poly(A) Binding Protein 2 or Nuclear PolyInhibitor, Retinoic Acid Receptor RXR Alpha Agonist, Cell Division Control Protein 42 Homolog Inhibitor, Dihydropyrimidinase Related Protein 2 Activator, and Galactocerebrosidase Replacement.
Batten Disease pipeline drugs market, by mechanisms of action
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Routes of Administration in Batten Disease Pipeline Drugs Market
The routes of administration in the Batten Disease pipeline drugs market are oral, intrathecal, parenteral, intravenous, intracerebral, intravitreal, intracisternal, and ophthalmic.
Batten Disease pipeline drugs market, by routes of administration
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Molecule Types in Batten Disease Pipeline Drugs Market
The molecule types in the Batten Disease pipeline drugs market are gene therapy, small molecule, biologic, cell therapy, oligonucleotide, protein, and recombinant enzyme.
Batten Disease pipeline drugs market, by molecule types
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Key Batten Disease Pipeline Drugs Market Companies
Some of the key companies in the Batten Disease pipeline drugs market are Neurogene Inc, Polaryx Therapeutics Inc, Amicus Therapeutics Inc, Recursion Pharmaceuticals Inc, Taysha Gene Therapies Inc, RegenxBio Inc, Retrotope Inc, Theranexus SAS, Abeona Therapeutics Inc, and Bioasis Technologies Inc.
Batten Disease pipeline drugs market, by key companies
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Market report overview
| Targets | Palmitoyl Protein Thioesterase 1, Tripeptidyl Peptidase 1, Peroxisome Proliferator Activated Receptor Alpha, Battenin, Ceroid Lipofuscinosis Neuronal Protein 6, Polyadenylate Binding Protein 2 (Poly(A) Binding Protein 2 or Nuclear Poly, Retinoic Acid Receptor RXR Alpha, Cell Division Control Protein 42 Homolog, Dihydropyrimidinase Related Protein 2, and Galactocerebrosidase |
| Mechanisms of Action | Palmitoyl Protein Thioesterase 1 Activator, Tripeptidyl Peptidase 1 Activator, Peroxisome Proliferator Activated Receptor Alpha Agonist, Battenin Activator, Ceroid Lipofuscinosis Neuronal Protein 6 Activator, Polyadenylate Binding Protein 2 (Poly(A) Binding Protein 2 or Nuclear PolyInhibitor, Retinoic Acid Receptor RXR Alpha Agonist, Cell Division Control Protein 42 Homolog Inhibitor, Dihydropyrimidinase Related Protein 2 Activator, and Galactocerebrosidase Replacement |
| Routes of Administration | Oral, Intrathecal, Parenteral, Intravenous, Intracerebral, Intravitreal, Intracisternal, and Ophthalmic |
| Molecule Types | Gene Therapy, Small Molecule, Biologic, Cell Therapy, Oligonucleotide, Protein, and Recombinant Enzyme |
| Key Companies | Neurogene Inc, Polaryx Therapeutics Inc, Amicus Therapeutics Inc, Recursion Pharmaceuticals Inc, Taysha Gene Therapies Inc, RegenxBio Inc, Retrotope Inc, Theranexus SAS, Abeona Therapeutics Inc, and Bioasis Technologies Inc |
Scope
This report provides:
- A snapshot of the global therapeutic landscape of Batten Disease (Central Nervous System).
- Reviews of pipeline therapeutics for Batten Disease (Central Nervous System) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- Pipeline products based on several stages of development ranging from pre-registration to discovery and undisclosed stages.
- Descriptive drug profiles for the pipeline products which include product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities.
- Reviews of key companies involved in Batten Disease (Central Nervous System) therapeutics and enlists all their major and minor projects.
- Evaluation of Batten Disease (Central Nervous System) targeted therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA), and molecule type.
- All the dormant and discontinued pipeline projects.
- Reviews of the latest news and deals related to pipeline therapeutics for Batten Disease (Central Nervous System).
Reasons to Buy
- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Batten Disease (Central Nervous System).
- 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 it’s most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Batten Disease (Central Nervous System) 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 pipeline.
Amicus Therapeutics Inc
Bioasis Technologies Inc
Blue Turtle Bio Technologies Inc
Circumvent Pharmaceuticals Inc
Collaborations Pharmaceuticals Inc
Exicure Inc
LEXEO Therapeutics LLC
Neurogene Inc
Polaryx Therapeutics Inc
Recursion Pharmaceuticals Inc
RegenxBio Inc
Retrotope Inc
Seneb BioSciences Inc
Spark Therapeutics Inc
Taysha Gene Therapies Inc
Theranexus SAS
Xonovo Inc
Table of Contents
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Frequently asked questions
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What are the targets of the Batten Disease pipeline drugs market?
Some of the targets of the Batten Disease pipeline drugs market are Palmitoyl Protein Thioesterase 1, Tripeptidyl Peptidase 1, Peroxisome Proliferator Activated Receptor Alpha, Battenin, Ceroid Lipofuscinosis Neuronal Protein 6, Polyadenylate Binding Protein 2 (Poly(A) Binding Protein 2 or Nuclear Poly, Retinoic Acid Receptor RXR Alpha, Cell Division Control Protein 42 Homolog, Dihydropyrimidinase Related Protein 2, and Galactocerebrosidase.
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What are the mechanisms of action of the Batten Disease pipeline drugs market?
Some of the mechanisms of action of the Batten Disease pipeline drugs market are Palmitoyl Protein Thioesterase 1 Activator, Tripeptidyl Peptidase 1 Activator, Peroxisome Proliferator Activated Receptor Alpha Agonist, Battenin Activator, Ceroid Lipofuscinosis Neuronal Protein 6 Activator, Polyadenylate Binding Protein 2 (Poly(A) Binding Protein 2 or Nuclear PolyInhibitor, Retinoic Acid Receptor RXR Alpha Agonist, Cell Division Control Protein 42 Homolog Inhibitor, Dihydropyrimidinase Related Protein 2 Activator, and Galactocerebrosidase Replacement.
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What are the routes of administration in the Batten Disease pipeline drugs market?
The routes of administration in the Batten Disease pipeline drugs market are oral, intrathecal, parenteral, intravenous, intracerebral, intravitreal, intracisternal, and ophthalmic.
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What are the molecule types in the Batten Disease pipeline drugs market?
The molecule types in the Batten Disease pipeline drugs market are gene therapy, small molecule, biologic, cell therapy, oligonucleotide, protein, and recombinant enzyme.
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Which are the key companies in the Batten Disease pipeline drugs market?
Some of the key companies in the Batten Disease pipeline drugs market are Neurogene Inc, Polaryx Therapeutics Inc, Amicus Therapeutics Inc, Recursion Pharmaceuticals Inc, Taysha Gene Therapies Inc, RegenxBio Inc, Retrotope Inc, Theranexus SAS, Abeona Therapeutics Inc, and Bioasis Technologies Inc.
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