Table of Content


List of Tables
List of Figures
Introduction
Global Markets Direct 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
Abeo 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 Desigtion to PLX-200 for the treatment of patients with Juvenile Neurol Ceroid Lipofuscinosis
Aug 11, 2020: Theranexus and BBDF obtain orphan drug desigtion (ODD) and rare pediatric disease desigtion (RPDD) from the Food and Drug Administration (FDA) for BBDF-101 for Batten Disease
Aug 11, 2020: Neurogene announces FDA Orphan Drug Desigtion for CLN7 Batten Disease Gene Therapy
Jul 07, 2020: FDA grants Orphan Drug Desigtion 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 Neurol Ceroid Lipofuscinosis
Jan 20, 2020: Polaryx Therapeutics receives IND approval for PLX-200 From the FDA for the treatment of late infantile neurol ceroid lipofuscinosis
Oct 17, 2019: REGENXBIO Announces Presentations at the European Society of Gene & Cell Therapy 27th Annual Congress
Jun 18, 2019: Abeo Therapeutics receives FDA fast track desigtion for ABO-202 AAV9 gene therapy in CLN1 disease
May 21, 2019: Abeo Therapeutics announces FDA clearance of Investigatiol New Drug Application for ABO-202 Gene Therapy in CLN1 Disease
Apr 15, 2019: Abeo 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 Desigtion for RGX-181 Gene Therapy for the Treatment of CLN2 Form of Batten Disease
Dec 06, 2018: Abeo 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 Us
Disclaimer



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


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