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2020 Batten Disease Drug Pipeline Report- Current Status, Phase, Mechanism, Route of Administration, and Companies, of Pre-Clinical And Clinical Drugs

February 2020 | 82 pages | ID: 221A7E6F7C67EN
VPAResearch

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The 2020 Batten Disease pipeline report presents a comprehensive overview of the research and development of Batten Disease drug candidates. It presents drugs in development that could potentially reach the market in the next 5 to 10 years: 16 drugs in pre-clinical phase and three drugs in Phase 2

As of February 2020, the Batten Disease pipeline remains robust with 19 therapeutic candidates under development. An increasing number of companies are actively participating in the development of Batten Disease treatment. Diverse types of targeted therapies are being explored through clinical trials including CLN1 Gene Therapy; CLN3 Gene Therapy; CLN6 Gene Therapy; CLN8 Gene Therapy; Lipid peroxidation inhibitors; Palmitoyl-protein thioesterase replacements; peroxisome proliferator activated receptor alpha (PPAR?) agonist; Tripeptidyl peptidase 1 replacements.

The report provides complete details of pipeline drugs including the development phase, mechanism of action, companies involved, clinical trial developments, molecule type, and other details. Further, the report also provides Batten Disease drug development history, latest news, and other developments.

It assists companies, governments, investors and research organizations to understand the current status in 2020 and possible development in the next 10 years. Further, it enables readers to track new companies in the market and their developments. The product portfolio of different companies and their growth strategies are also detailed in the report.

PUBLISHER EXPERTISE

VPAResearch online databases analyze pipeline drugs and developments for over 2,000 diseases worldwide. All our reports and databases are developed through intensive primary and secondary research methods. The insights and data presented in the databases are validated through industry experts and represent completely unbiased opinions.

SCOPE:
  • The report scope comprises of both pre-clinical phase and clinical phase development drugs for Batten Disease development
  • Batten Disease pipeline compounds and molecules under study by both large scale and small companies are included in the report
  • Batten Disease pipeline across different phases including discovery, research, and pre-clinical stage, phase 1, phase 2, phase 3 and pre-registration phases are covered
  • Drug profile comprising of current development status, regulatory progress, companies, sponsors, mechanism of action, route of administration, molecule, and discovery details are covered
  • Further, orphan drug status, fast track designation, different grants awarded and special status for Batten Disease pipeline candidates included
  • Business overview and snapshot of all companies involved in Batten Disease pipeline are included
  • Latest market and pipeline developments are provided in the report
Batten Disease pipeline companies included in the report are- Abeona Therapeutics Inc, Amicus Therapeutics Inc, Circumvent Pharmaceuticals Inc, Collaborations Pharmaceuticals Inc, Exicure Inc, Polaryx Therapeutics Inc, Recursion Pharmaceuticals Inc, RegenxBio Inc, Retrotope Inc, Seneb BioSciences Inc, Spark Therapeutics Inc, Xonovo Inc

Batten Disease pipeline drugs profiled in the report include- ABO-201, ABO-202, CLN1 Gene Therapy for Batten Disease, CLN3 Gene Therapy for Batten Disease, CLN6 Gene Therapy for Batten Disease, CLN8 Gene Therapy for Batten Disease, Small Molecule for Batten Disease, Recombinant palmitoyl-protein thioesterase-1 for Batten Disease, Oligonucleotides for Neurology, PLX-100, PLX-200, PLX-300, preclinical programs for Batten disease, AAVRh.10, RGX-181, RT001, SNB-4050, SPK-TPP1, XN001
1. TABLE OF CONTENTS

1.1 List of Tables
1.2 List of Figures

2. EXECUTIVE SUMMARY

2.1 Report Scope and Research Methodology
2.2 Introduction to Batten Disease Condition
2.3 Batten Disease Pipeline Snapshot, 2020
2.4 Companies investing in Batten Disease pipeline therapeutics
2.5 Phase wise Batten Disease Pipeline Candidates
2.6 Most Researched Mechanism of Action of Batten Disease Pipeline Products
2.7 Route of Administration of Batten Disease Pipeline Drugs

3. COMPANIES ACTIVE IN PIPELINE DEVELOPMENT

3.1 Abeona Therapeutics Inc Overview, Contacts and ASD Pipeline Drugs
3.2 Amicus Therapeutics Inc Overview, Contacts and ASD Pipeline Drugs
3.3 Circumvent Pharmaceuticals Inc Overview, Contacts and ASD Pipeline Drugs
3.4 Collaborations Pharmaceuticals Inc Overview, Contacts and ASD Pipeline Drugs
3.5 Exicure Inc Overview, Contacts and ASD Pipeline Drugs
3.6 Polaryx Therapeutics Inc Overview, Contacts and ASD Pipeline Drugs
3.7 Recursion Pharmaceuticals Inc Overview, Contacts and ASD Pipeline Drugs
3.8 RegenxBio Inc Overview, Contacts and ASD Pipeline Drugs
3.9 Retrotope Inc Overview, Contacts and ASD Pipeline Drugs
3.10 Seneb BioSciences Inc Overview, Contacts and ASD Pipeline Drugs
3.11 Spark Therapeutics Inc Overview, Contacts and ASD Pipeline Drugs
3.12 Xonovo Inc Overview, Contacts and ASD Pipeline Drugs

4. ACTIVE PIPELINE DRUG DETAILS, 2020

4.1 ABO-201 Drug Details
  4.1.1 ABO-201 Current Status
  4.1.2 ABO-201 Drug Overview
  4.1.3 ABO-201 Mechanism of Action
  4.1.4 ABO-201 Licensing/Collaboration Companies
  4.1.5 ABO-201 Clinical Trials
4.2 ABO-202 Drug Details
  4.2.1 ABO-202 Current Status
  4.2.2 ABO-202 Drug Overview
  4.2.3 ABO-202 Mechanism of Action
  4.2.4 ABO-202 Licensing/Collaboration Companies
  4.2.5 ABO-202 Clinical Trials
4.3 CLN1 Gene Therapy for Batten Disease Drug Details
  4.3.1 CLN1 Gene Therapy for Batten Disease Current Status
  4.3.2 CLN1 Gene Therapy for Batten Disease Drug Overview
  4.3.3 CLN1 Gene Therapy for Batten Disease Mechanism of Action
  4.3.4 CLN1 Gene Therapy for Batten Disease Licensing/Collaboration Companies
  4.3.5 CLN1 Gene Therapy for Batten Disease Clinical Trials
4.4 CLN3 Gene Therapy for Batten Disease Drug Details
  4.4.1 CLN3 Gene Therapy for Batten Disease Current Status
  4.4.2 CLN3 Gene Therapy for Batten Disease Drug Overview
  4.4.3 CLN3 Gene Therapy for Batten Disease Mechanism of Action
  4.4.4 CLN3 Gene Therapy for Batten Disease Licensing/Collaboration Companies
  4.4.5 CLN3 Gene Therapy for Batten Disease Clinical Trials
4.5 CLN6 Gene Therapy for Batten Disease Drug Details
  4.5.1 CLN6 Gene Therapy for Batten Disease Current Status
  4.5.2 CLN6 Gene Therapy for Batten Disease Drug Overview
  4.5.3 CLN6 Gene Therapy for Batten Disease Mechanism of Action
  4.5.4 CLN6 Gene Therapy for Batten Disease Licensing/Collaboration Companies
  4.5.5 CLN6 Gene Therapy for Batten Disease Clinical Trials
4.6 CLN8 Gene Therapy for Batten Disease Drug Details
  4.6.1 CLN8 Gene Therapy for Batten Disease Current Status
  4.6.2 CLN8 Gene Therapy for Batten Disease Drug Overview
  4.6.3 CLN8 Gene Therapy for Batten Disease Mechanism of Action
  4.6.4 CLN8 Gene Therapy for Batten Disease Licensing/Collaboration Companies
  4.6.5 CLN8 Gene Therapy for Batten Disease Clinical Trials
4.7 Small Molecule for Batten Disease Drug Details
  4.7.1 Small Molecule for Batten Disease Current Status
  4.7.2 Small Molecule for Batten Disease Drug Overview
  4.7.3 Small Molecule for Batten Disease Mechanism of Action
  4.7.4 Small Molecule for Batten Disease Licensing/Collaboration Companies
  4.7.5 Small Molecule for Batten Disease Clinical Trials
4.8 Recombinant palmitoyl-protein thioesterase-1 for Batten Disease Drug Details
  4.8.1 Recombinant palmitoyl-protein thioesterase-1 for Batten Disease Current Status
  4.8.2 Recombinant palmitoyl-protein thioesterase-1 for Batten Disease Drug Overview
  4.8.3 Recombinant palmitoyl-protein thioesterase-1 for Batten Disease Mechanism of Action
  4.8.4 Recombinant palmitoyl-protein thioesterase-1 for Batten Disease Licensing/Collaboration Companies
  4.8.5 Recombinant palmitoyl-protein thioesterase-1 for Batten Disease Clinical Trials
4.9 Oligonucleotides for Neurology Drug Details
  4.9.1 Oligonucleotides for Neurology Current Status
  4.9.2 Oligonucleotides for Neurology Drug Overview
  4.9.3 Oligonucleotides for Neurology Mechanism of Action
  4.9.4 Oligonucleotides for Neurology Licensing/Collaboration Companies
  4.9.5 Oligonucleotides for Neurology Clinical Trials
4.10 PLX-100 Drug Details
  4.10.1 PLX-100 Current Status
  4.10.2 PLX-100 Drug Overview
  4.10.3 PLX-100 Mechanism of Action
  4.10.4 PLX-100 Licensing/Collaboration Companies
  4.10.5 PLX-100 Clinical Trials
4.11 PLX-200 Drug Details
  4.11.1 PLX-200 Current Status
  4.11.2 PLX-200 Drug Overview
  4.11.3 PLX-200 Mechanism of Action
  4.11.4 PLX-200 Licensing/Collaboration Companies
  4.11.5 PLX-200 Clinical Trials
4.12 PLX-300 Drug Details
  4.12.1 PLX-300 Current Status
  4.12.2 PLX-300 Drug Overview
  4.12.3 PLX-300 Mechanism of Action
  4.12.4 PLX-300 Licensing/Collaboration Companies
  4.12.5 PLX-300 Clinical Trials
4.13 preclinical programs for Batten disease Drug Details
  4.13.1 preclinical programs for Batten disease Current Status
  4.13.2 preclinical programs for Batten disease Drug Overview
  4.13.3 preclinical programs for Batten disease Mechanism of Action
  4.13.4 preclinical programs for Batten disease Licensing/Collaboration Companies
  4.13.5 preclinical programs for Batten disease Clinical Trials
4.14 AAVRh.10 Drug Details
  4.14.1 AAVRh.10 Current Status
  4.14.2 AAVRh.10 Drug Overview
  4.14.3 AAVRh.10 Mechanism of Action
  4.14.4 AAVRh.10 Licensing/Collaboration Companies
  4.14.5 AAVRh.10 Clinical Trials
4.15 RGX-181 Drug Details
  4.15.1 RGX-181 Current Status
  4.15.2 RGX-181 Drug Overview
  4.15.3 RGX-181 Mechanism of Action
  4.15.4 RGX-181 Licensing/Collaboration Companies
  4.15.5 RGX-181 Clinical Trials
4.16 RT001 Drug Details
  4.16.1 RT001 Current Status
  4.16.2 RT001 Drug Overview
  4.16.3 RT001 Mechanism of Action
  4.16.4 RT001 Licensing/Collaboration Companies
  4.16.5 RT001 Clinical Trials
4.17 SNB-4050 Drug Details
  4.17.1 SNB-4050 Current Status
  4.17.2 SNB-4050 Drug Overview
  4.17.3 SNB-4050 Mechanism of Action
  4.17.4 SNB-4050 Licensing/Collaboration Companies
  4.17.5 SNB-4050 Clinical Trials
4.18 SPK-TPP1 Drug Details
  4.18.1 SPK-TPP1 Current Status
  4.18.2 SPK-TPP1 Drug Overview
  4.18.3 SPK-TPP1 Mechanism of Action
  4.18.4 SPK-TPP1 Licensing/Collaboration Companies
  4.18.5 SPK-TPP1 Clinical Trials
4.19 XN001 Drug Details
  4.19.1 XN001 Current Status
  4.19.2 XN001 Drug Overview
  4.19.3 XN001 Mechanism of Action
  4.19.4 XN001 Licensing/Collaboration Companies
  4.19.5 XN001 Clinical Trials

5. LATEST BATTEN DISEASE PIPELINE NEWS AND DEALS

6. APPENDIX

6.1 Our Databases and Reports
6.2 Research Methodolgy


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