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
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
Batten Disease - Drug Profiles
(gemfibrozil + tretinoin) - Drug Profile
Product Description
Mechanism Of Action
History of Events
ABO-201 - Drug Profile
Product Description
Mechanism Of Action
History of Events
ATGTX-502 - Drug Profile
Product Description
Mechanism Of Action
History of Events
Biologic to Activate CLN6 for Batten Disease - Drug Profile
Product Description
Mechanism Of Action
CIRC-825 - Drug Profile
Product Description
Mechanism Of Action
History of Events
DUOC-01 - Drug Profile
Product Description
Mechanism Of Action
gemfibrozil - Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene Therapy for Batten Diseases - Drug Profile
Product Description
Mechanism Of Action
Gene Therapy to Activate CLN1 for Batten Disease - Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene Therapy to Activate CLN6 for Batten Disease - Drug Profile
Product Description
Mechanism Of Action
Gene Therapy to Activate CLN7 for Batten Disease - Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene Therapy to Activate CLN7 for Batten Disease - Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene Therapy to Activate CLN8 for Batten Disease - Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene Therapy to Activate Palmitoyl Protein Thioesterase 1 for Batten Disease - Drug Profile
Product Description
Mechanism Of Action
Gene Therapy to Activate TPP1 for Batten Disease - Drug Profile
Product Description
Mechanism Of Action
INI-0602 - Drug Profile
Product Description
Mechanism Of Action
LX-1004 - Drug Profile
Product Description
Mechanism Of Action
History of Events
miglustat - Drug Profile
Product Description
Mechanism Of Action
History of Events
miglustat + trehalose - Drug Profile
Product Description
Mechanism Of Action
History of Events
NGN-101 - Drug Profile
Product Description
Mechanism Of Action
History of Events
Oligonucleotides to Target CLN3 For Batten Disease - Drug Profile
Product Description
Mechanism Of Action
PLX-300 - Drug Profile
Product Description
Mechanism Of Action
REC-259618 - Drug Profile
Product Description
Mechanism Of Action
REC-648190 - Drug Profile
Product Description
Mechanism Of Action
REC-648647 - Drug Profile
Product Description
Mechanism Of Action
Recombint Palmitoyl-Protein Thioesterase-1 Replacement for Batten Disease - Drug Profile
Product Description
Mechanism Of Action
History of Events
RGX-181 - Drug Profile
Product Description
Mechanism Of Action
History of Events
RGX-381 - Drug Profile
Product Description
Mechanism Of Action
History of Events
RT-001 - Drug Profile
Product Description
Mechanism Of Action
RT-002 - Drug Profile
Product Description
Mechanism Of Action
Small Molecules for Batten Disease - Drug Profile
Product Description
Mechanism Of Action
Small Molecules to Inhibit CDC42 for Batten Disease - Drug Profile
Product Description
Mechanism Of Action
SNB-4050 - Drug Profile
Product Description
Mechanism Of Action
History of Events
SPK-1001 - Drug Profile
Product Description
Mechanism Of Action
History of Events
TSHA-118 - Drug Profile
Product Description
Mechanism Of Action
History of Events
xB3-Progranulin - Drug Profile
Product Description
Mechanism Of Action
XN-001 - Drug Profile
Product Description
Mechanism Of Action
History of Events
Batten Disease - Dormant Projects
Batten Disease - Discontinued Products
Batten Disease - Product Development Milestones
Featured News & Press Releases
Feb 09, 2022: Taysha Gene Therapies announces positive safety data from UT Southwestern-sponsored clinical trial for the treatment of CLN7 batten disease at 18th annual WORLDSymposium
Dec 16, 2021: Taysha Gene Therapies announces initiation of clinical development of TSHA-118 for the treatment of CLN1 disease
Sep 14, 2021: Neurogene announces FDA clearance of IND for NGN-101 Gene Therapy to Treat CLN5 Batten Disease
Aug 17, 2021: Taysha Gene Therapies to host key opinion leader webir on TSHA-118 for the treatment of CLN1 disease
Jun 29, 2021: Neurogene announces EMA grants orphan drug desigtion to CLN5 Batten disease gene therapy
Nov 19, 2020: The Food and Drug Administration (FDA) issues a favorable opinion on the preclinical development plan submitted by Theranexus and BBDF
Nov 12, 2020: Neurogene announces first patient enrolled in tural History study evaluating two subtypes of Batten Disease including CLN5
Nov 12, 2020: Neurogene announces first patient enrolled in tural History Study evaluating two subtypes including CLN7 subtype of Batten Disease
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
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures

List of Figures
Number of Products under Development for Batten Disease, 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Universities/Institutes, 2022
Number of Products by Top 10 Targets, 2022
Number of Products by Stage and Top 10 Targets, 2022
Number of Products by Top 10 Mechanism of Actions, 2022
Number of Products by Stage and Top 10 Mechanism of Actions, 2022
Number of Products by Top 10 Routes of Administration, 2022
Number of Products by Stage and Top 10 Routes of Administration, 2022
Number of Products by Molecule Types, 2022
Number of Products by Stage and Molecule Types, 2022

List of Tables

List of Tables
Number of Products under Development for Batten Disease, 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Universities/Institutes, 2022
Products under Development by Companies, 2022
Products under Development by Companies, 2022 (Contd..1)
Products under Development by Universities/Institutes, 2022
Number of Products by Stage and Target, 2022
Number of Products by Stage and Mechanism of Action, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Batten Disease - Pipeline by Abeona Therapeutics Inc, 2022
Batten Disease - Pipeline by Amicus Therapeutics Inc, 2022
Batten Disease - Pipeline by Bioasis Technologies Inc, 2022
Batten Disease - Pipeline by Blue Turtle Bio Technologies Inc, 2022
Batten Disease - Pipeline by Circumvent Pharmaceuticals Inc, 2022
Batten Disease - Pipeline by Collaborations Pharmaceuticals Inc, 2022
Batten Disease - Pipeline by Exicure Inc, 2022
Batten Disease - Pipeline by LEXEO Therapeutics LLC, 2022
Batten Disease - Pipeline by Neurogene Inc, 2022
Batten Disease - Pipeline by Polaryx Therapeutics Inc, 2022
Batten Disease - Pipeline by Recursion Pharmaceuticals Inc, 2022
Batten Disease - Pipeline by RegenxBio Inc, 2022
Batten Disease - Pipeline by Retrotope Inc, 2022
Batten Disease - Pipeline by Seneb BioSciences Inc, 2022
Batten Disease - Pipeline by Spark Therapeutics Inc, 2022
Batten Disease - Pipeline by Taysha Gene Therapies Inc, 2022
Batten Disease - Pipeline by Theranexus SAS, 2022
Batten Disease - Pipeline by Xonovo Inc, 2022
Batten Disease - Dormant Projects, 2022
Batten Disease - Discontinued Products, 2022