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Batten Disease - Pipeline Review, H2 2020

  • ID: 5146854
  • Drug Pipelines
  • August 2020
  • Region: Global
  • 93 pages
  • Global Markets Direct

FEATURED COMPANIES

  • Abeona Therapeutics Inc
  • Amicus Therapeutics Inc
  • Exicure Inc
  • Neurogene Inc
  • Polaryx Therapeutics Inc
  • RegenxBio Inc
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Summary

The publisher's latest Pharmaceutical and Healthcare disease pipeline guide Batten Disease - Pipeline Review, H2 2020, provides an overview of the Batten Disease (Central Nervous System) pipeline landscape.

Batten disease, a rare genetic disorder, belongs to a group of progressive degenerative neurometabolic disorders known as the neuronal ceroid lipofuscinoses. Symptoms of Batten disease usually become apparent between five and 15 years of age. 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.

Report Highlights

The publisher's Pharmaceutical and Healthcare latest pipeline guide Batten Disease - Pipeline Review, H2 2020, provides comprehensive information on the therapeutics under development for Batten Disease (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Batten Disease (Central Nervous System) pipeline guide also reviews the key players involved in therapeutic development for Batten Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, Phase I, IND/CTA Filed, Preclinical and Discovery stages are 2, 1, 2, 18 and 1 respectively. Similarly, the Universities portfolio in Phase I, Preclinical and Discovery stages comprises 1, 4 and 2 molecules, respectively.

Batten Disease (Central Nervous System) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from the publisher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope
  • The pipeline guide provides a snapshot of the global therapeutic landscape of Batten Disease (Central Nervous System).
  • The pipeline guide reviews pipeline therapeutics for Batten Disease (Central Nervous System) 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 Batten Disease (Central Nervous System) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Batten Disease (Central Nervous System) 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 latest news 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 the 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 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 the pipeline.
Note: Product cover images may vary from those shown

FEATURED COMPANIES

  • Abeona Therapeutics Inc
  • Amicus Therapeutics Inc
  • Exicure Inc
  • Neurogene Inc
  • Polaryx Therapeutics Inc
  • RegenxBio Inc
Introduction

Report Coverage
  • Batten Disease - Overview
  • Batten Disease - Therapeutics Development
Pipeline Overview
  • Pipeline by Companies
  • Pipeline by Universities/Institutes
  • Products under Development by Companies
  • Products under Development by Universities/Institutes
Batten Disease - Therapeutics Assessment
  • Assessment by Target
  • Assessment by Mechanism of Action
  • Assessment by Route of Administration
  • Assessment by Molecule Type
Batten Disease - Companies Involved in Therapeutics Development
  • Abeona Therapeutics Inc
  • Amicus Therapeutics Inc
  • Blue Turtle Bio Technologies Inc
  • Circumvent Pharmaceuticals Inc
  • Collaborations Pharmaceuticals Inc
  • Exicure Inc
  • Neurogene Inc
  • Polaryx Therapeutics Inc
  • Recursion Pharmaceuticals Inc
  • RegenxBio Inc
  • Retrotope Inc
  • Seneb BioSciences Inc
  • Spark Therapeutics Inc
  • Theranexus SAS
  • Xonovo Inc
Batten Disease - Drug Profiles
(gemfibrozil + tretinoin) - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • ABO-201 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
Batten Disease - Dormant Projects
  • Batten Disease - Discontinued Products
  • Batten Disease - Product Development Milestones
Featured News & Press Releases
  • Aug 20, 2020: Polaryx Therapeutics receives FDA Fast Track Designation to PLX-200 for the treatment of patients with Juvenile Neuronal Ceroid Lipofuscinosis
  • Aug 11, 2020: Theranexus and BBDF obtain orphan drug designation (ODD) and rare pediatric disease designation (RPDD) from the Food and Drug Administration (FDA) for BBDF-101 for Batten Disease
  • Aug 11, 2020: Neurogene announces FDA Orphan Drug Designation for CLN7 Batten Disease Gene Therapy
  • Jul 07, 2020: FDA grants Orphan Drug Designation to Neurogene’s gene therapy for the treatment of CLN5 Batten Disease
  • Jun 24, 2020: Theranexus provides update on its Batten disease drug candidate BBDF-101
  • May 05, 2020: Amicus Therapeutics announces presentation on its CLN8 batten disease gene therapy at the American Society of Gene & Cell Therapy 23rd Annual Meeting
  • Apr 08, 2020: Polaryx Therapeutics receives IND approval from the FDA to study PLX-200 treatment for patients with Juvenile Neuronal Ceroid Lipofuscinosis
  • Jan 20, 2020: Polaryx Therapeutics receives IND approval for PLX-200 From the FDA for the treatment of late infantile neuronal ceroid lipofuscinosis
  • Oct 17, 2019: REGENXBIO Announces Presentations at the European Society of Gene & Cell Therapy 27th Annual Congress
  • Jun 18, 2019: Abeona Therapeutics receives FDA fast track designation for ABO-202 AAV9 gene therapy in CLN1 disease
  • May 21, 2019: Abeona Therapeutics announces FDA clearance of Investigational New Drug Application for ABO-202 Gene Therapy in CLN1 Disease
  • Apr 15, 2019: Abeona Therapeutics to present data for ABO-202 in CLN1 disease at the American Society of Gene and Cell Therapy Annual Meeting
  • Jan 31, 2019: Spark Therapeutics announces presentation of preclinical data in CLN2 disease at 15th annual WORLDSymposium
  • Jan 31, 2019: REGENXBIO receives Rare Pediatric Disease Designation for RGX-181 Gene Therapy for the Treatment of CLN2 Form of Batten Disease
  • Dec 06, 2018: Abeona Therapeutics provides update on Infantile Batten Disease drug candidate ABO-202 at 2018 R&D Day
Appendix
  • Methodology
  • Coverage
  • Secondary Research
  • Primary Research
  • Expert Panel Validation
  • Contact
  • Disclaimer
List of Tables
  • Number of Products under Development for Batten Disease, H2 2020
  • Number of Products under Development by Companies, H2 2020
  • Number of Products under Development by Universities/Institutes, H2 2020
  • Products under Development by Companies, H2 2020
  • Products under Development by Companies, H2 2020 (Contd..1), H2 2020
  • Products under Development by Universities/Institutes, H2 2020
  • Number of Products by Stage and Target, H2 2020
  • Number of Products by Stage and Mechanism of Action, H2 2020
  • Number of Products by Stage and Route of Administration, H2 2020
  • Number of Products by Stage and Molecule Type, H2 2020
  • Batten Disease - Pipeline by Abeona Therapeutics Inc, H2 2020
  • Batten Disease - Pipeline by Amicus Therapeutics Inc, H2 2020
  • Batten Disease - Pipeline by Blue Turtle Bio Technologies Inc, H2 2020
  • Batten Disease - Pipeline by Circumvent Pharmaceuticals Inc, H2 2020
  • Batten Disease - Pipeline by Collaborations Pharmaceuticals Inc, H2 2020
  • Batten Disease - Pipeline by Exicure Inc, H2 2020
  • Batten Disease - Dormant Projects, H2 2020
  • Batten Disease - Discontinued Products, H2 2020
List of Figures
  • Number of Products under Development for Batten Disease, H2 2020
  • Number of Products under Development by Companies, H2 2020
  • Number of Products under Development by Universities/Institutes, H2 2020
  • Number of Products by Top 10 Targets, H2 2020
  • Number of Products by Stage and Top 10 Targets, H2 2020
  • Number of Products by Top 10 Mechanism of Actions, H2 2020
  • Number of Products by Stage and Top 10 Mechanism of Actions, H2 2020
  • Number of Products by Routes of Administration, H2 2020
  • Number of Products by Stage and Routes of Administration, H2 2020
  • Number of Products by Molecule Types, H2 2020
  • Number of Products by Stage and Molecule Types, H2 2020
Note: Product cover images may vary from those shown
  • Abeona Therapeutics Inc
  • Amicus Therapeutics Inc
  • Blue Turtle Bio Technologies Inc
  • Circumvent Pharmaceuticals Inc
  • Collaborations Pharmaceuticals Inc
  • Exicure Inc
  • Neurogene Inc
  • Polaryx Therapeutics Inc
  • Recursion Pharmaceuticals Inc
  • RegenxBio Inc
  • Retrotope Inc
  • Seneb BioSciences Inc
  • Spark Therapeutics Inc
  • Theranexus SAS
  • Xonovo Inc
Note: Product cover images may vary from those shown
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