+353-1-416-8900REST OF WORLD
+44-20-3973-8888REST OF WORLD
1-917-300-0470EAST COAST U.S
1-800-526-8630U.S. (TOLL FREE)

2019 Future of Rett Syndrome R&D Pipeline Drugs and Companies- Analysis of Pipeline Compounds, Phases, Mechanism of Action, Clinical Trials and Developments

  • PDF Icon


  • 80 Pages
  • February 2019
  • Region: Global
  • VPA Research
  • ID: 4749252
The global demand for Rett Syndrome treatment options is emerging rapidly driven by demand in major emerging and developed markets. An increasing number of companies are working on Rett Syndrome pipeline. Companies quickly adapting to the new technologies both independently and through collaborations and advancing their pipeline candidates to advanced phases will evolve as top performers. However, challenges of completing clinical trials on schedules and limited financial support are holding some of the Rett Syndrome pipeline companies from advancing their products into Phase 3 or Phase 4.

Rett Syndrome Report Description

The 2019 pipeline study on Rett Syndrome pipeline is a comprehensive research on the drug candidates across different phases. This research study evaluates each of the pipeline products in terms of their current status, regulatory progress and expected phase completion date. Focusing on the factors driving and challenging pipeline drug development, this strategic analysis review presents detailed analysis on all active Rett Syndrome pipeline compounds.

The Rett Syndrome pipeline guide presents information on all active drugs currently being developed for Rett Syndrome. The research work provides details of active therapeutic candidates in different phases including discovery, research, pre-clinical, phase I, Phase II, Phase III and pre-registration phases.

Details of current status, R&D progress and latest developments for every Rett Syndrome pipeline candidate are analyzed.

Further, current phase of development, mechanism of drug action, special designations such as orphan drug status, fast track designation, grants awarded, etc are presented for all Rett Syndrome drug candidates.

Drug development companies, collaborators, originating companies, license providers and universities participating in the Rett Syndrome product pipeline are provided. Company overview, snapshot and developments are provided for all participating companies.

The report assists in identifying potential upcoming companies and drugs in Rett Syndrome pipeline market along with progress of each drug. It also enables users to formulate effective strategies to stay ahead of competition.

The publisher has developed a robust methodology for assessing pharmaceutical drugs, market sizes and sound forecast tools. All our research reports are provided through intense and repetitive primary and secondary research methods. Further, these reports are validated with industry experts to ensure reliability in the current scenario. The report is presented in a user-friendly format and presents clear and actionable insights.

Scope of Rett Syndrome pipeline report includes
  • An overview of Rett Syndrome disease including symptoms, causes, diagnosis and available treatment options is provided.

  • Snapshot of pipeline detailing number of active compounds, mergers and acquisitions, phase wise compounds, target areas, mechanism of action, trials etc

  • Phase wise count of Rett Syndrome pipeline

  • Company wise list of Rett Syndrome pipeline

  • Mechanism of Action wise Rett Syndrome pipeline

For each pipeline candidate, the following details are provided:
  • Snapshot (Drug, Mechanism of Action, Phase, Co Developer, Originator, Synonym, Orphan Drug Status, New Molecular Entity, Area)

  • Current status of development

  • Drug overview

  • Mechanism of Action

  • Pre-clinical and Clinical Trials/Results

  • Company Overview and Recent Developments

Reasons to Buy
  • The report is designed to help industry executives promote the success and continued growth of their organizations

  • Get clear understanding of the entire Rett Syndrome pipeline, with details on active projects

  • Stay ahead of the competition through comprehensive knowledge of Rett Syndrome pipeline progress

  • Get in detail information of each product with updated information on each project along with key milestones

  • Gain clear insights into companies participating in Rett Syndrome pipeline along with progress of each of the company's products and information on associations/collaborations/licensing data

  • Get trial information for each pipeline product under development

  • Understand the pipeline structure in terms of mechanism of Action, phase and company

The report will be delivered in 2 working days.

Table of Contents

1.1 List of Tables

1.2 List of Figures

2. Global Rett Syndrome Pipeline Overview
2.1 Key Findings, 2019
2.2 Disease Overview
2.3 Research Methodology

3. Executive Summary
3.1 Rett Syndrome Drugs under active development, H1- 2019
3.2 Pipeline Drugs in Early Stage of Development
3.2.1 Pre-clinical
3.2.2 Discovery
3.2.3 Phase 1
3.2.4Phase 2
3.3 Pipeline Drugs in Advanced Stage of Development
3.3.1 Phase 3
3.3.2 Pre-registration
3.4 Companies involved in Rett Syndrome pipeline, H1- 2019
3.5 Mechanism of Action wise Rett Syndrome Pipeline Candidates

4 AMO Pharma Ltd Rett Syndrome Pipeline Details
4.1 AMO Pharma Ltd Business Profile
4.2 AMO Pharma Ltd Rett Syndrome Drug Details
4.3 Drug Snapshot
4.3.1 Originator
4.3.2 Collaborator/Co-Developer
4.3.3 Route of Administration
4.3.4 Orphan Drug/Fast Track/Special Designation
4.3.5 Geography
4.3.6 Type of Molecular Entity
4.3.7 Current Status
4.4 Drug Overview
4.5 Drug Mechanism of Action
4.6 Clinical/Pre-clinical Trial Details
4.7 Latest Drug Developments

5 Amorsa Therapeutics Inc Rett Syndrome Pipeline Details
5.1 Amorsa Therapeutics Inc Business Profile
5.2 Amorsa Therapeutics Inc Rett Syndrome Drug Details
5.3 Drug Snapshot
5.3.1 Originator
5.3.2 Collaborator/Co-Developer
5.3.3 Route of Administration
5.3.4 Orphan Drug/Fast Track/Special Designation
5.3.5 Geography
5.3.6 Type of Molecular Entity
5.3.7 Current Status
5.4 Drug Overview
5.5 Drug Mechanism of Action
5.6 Clinical/Pre-clinical Trial Details
5.7 Latest Drug Developments

6 Anavex Life Sciences Corp Rett Syndrome Pipeline Details
6.1 Anavex Life Sciences Corp Business Profile
6.2 Anavex Life Sciences Corp Rett Syndrome Drug Details
6.3 Drug Snapshot
6.3.1 Originator
6.3.2 Collaborator/Co-Developer
6.3.3 Route of Administration
6.3.4 Orphan Drug/Fast Track/Special Designation
6.3.5 Geography
6.3.6 Type of Molecular Entity
6.3.7 Current Status
6.4 Drug Overview
6.5 Drug Mechanism of Action
6.6 Clinical/Pre-clinical Trial Details
6.7 Latest Drug Developments

7 ArmaGen Inc Rett Syndrome Pipeline Details
7.1 ArmaGen Inc Business Profile
7.2 ArmaGen Inc Rett Syndrome Drug Details
7.3 Drug Snapshot
7.3.1 Originator
7.3.2 Collaborator/Co-Developer
7.3.3 Route of Administration
7.3.4 Orphan Drug/Fast Track/Special Designation
7.3.5 Geography
7.3.6 Type of Molecular Entity
7.3.7 Current Status
7.4 Drug Overview
7.5 Drug Mechanism of Action
7.6 Clinical/Pre-clinical Trial Details
7.7 Latest Drug Developments

8 AveXis Inc Rett Syndrome Pipeline Details
8.1 AveXis Inc Business Profile
8.2 AveXis Inc Rett Syndrome Drug Details
8.3 Drug Snapshot
8.3.1 Originator
8.3.2 Collaborator/Co-Developer
8.3.3 Route of Administration
8.3.4 Orphan Drug/Fast Track/Special Designation
8.3.5 Geography
8.3.6 Type of Molecular Entity
8.3.7 Current Status
8.4 Drug Overview
8.5 Drug Mechanism of Action
8.6 Clinical/Pre-clinical Trial Details
8.7 Latest Drug Developments

9 Biohaven Pharmaceutical Holding Company Ltd Rett Syndrome Pipeline Details
9.1 Biohaven Pharmaceutical Holding Company Ltd Business Profile
9.2 Biohaven Pharmaceutical Holding Company Ltd Rett Syndrome Drug Details
9.3 Drug Snapshot
9.3.1 Originator
9.3.2 Collaborator/Co-Developer
9.3.3 Route of Administration
9.3.4 Orphan Drug/Fast Track/Special Designation
9.3.5 Geography
9.3.6 Type of Molecular Entity
9.3.7 Current Status
9.4 Drug Overview
9.5 Drug Mechanism of Action
9.6 Clinical/Pre-clinical Trial Details
9.7 Latest Drug Developments

10 Cold Spring Harbor Laboratory Rett Syndrome Pipeline Details
10.1 Cold Spring Harbor Laboratory Business Profile
10.2 Cold Spring Harbor Laboratory Rett Syndrome Drug Details
10.3 Drug Snapshot
10.3.1 Originator
10.3.2 Collaborator/Co-Developer
10.3.3 Route of Administration
10.3.4 Orphan Drug/Fast Track/Special Designation
10.3.5 Geography
10.3.6 Type of Molecular Entity
10.3.7 Current Status
10.4 Drug Overview
10.5 Drug Mechanism of Action
10.6 Clinical/Pre-clinical Trial Details
10.7 Latest Drug Developments

11 Eloxx Pharmaceuticals Inc Rett Syndrome Pipeline Details
11.1 Eloxx Pharmaceuticals Inc Business Profile
11.2 Eloxx Pharmaceuticals Inc Rett Syndrome Drug Details
11.3 Drug Snapshot
11.3.1 Originator
11.3.2 Collaborator/Co-Developer
11.3.3 Route of Administration
11.3.4 Orphan Drug/Fast Track/Special Designation
11.3.5 Geography
11.3.6 Type of Molecular Entity
11.3.7 Current Status
11.4 Drug Overview
11.5 Drug Mechanism of Action
11.6 Clinical/Pre-clinical Trial Details
11.7 Latest Drug Developments

12 GW Pharmaceuticals Plc Rett Syndrome Pipeline Details
12.1 GW Pharmaceuticals Plc Business Profile
12.2 GW Pharmaceuticals Plc Rett Syndrome Drug Details
12.3 Drug Snapshot
12.3.1 Originator
12.3.2 Collaborator/Co-Developer
12.3.3 Route of Administration
12.3.4 Orphan Drug/Fast Track/Special Designation
12.3.5 Geography
12.3.6 Type of Molecular Entity
12.3.7 Current Status
12.4 Drug Overview
12.5 Drug Mechanism of Action
12.6 Clinical/Pre-clinical Trial Details
12.7 Latest Drug Developments

13 Mitochon Pharmaceuticals Inc Rett Syndrome Pipeline Details
13.1 Mitochon Pharmaceuticals Inc Business Profile
13.2 Mitochon Pharmaceuticals Inc Rett Syndrome Drug Details
13.3 Drug Snapshot
13.3.1 Originator
13.3.2 Collaborator/Co-Developer
13.3.3 Route of Administration
13.3.4 Orphan Drug/Fast Track/Special Designation
13.3.5 Geography
13.3.6 Type of Molecular Entity
13.3.7 Current Status
13.4 Drug Overview
13.5 Drug Mechanism of Action
13.6 Clinical/Pre-clinical Trial Details
13.7 Latest Drug Developments

14 Mitsubishi Tanabe Pharma Corp Rett Syndrome Pipeline Details
14.1 Mitsubishi Tanabe Pharma Corp Business Profile
14.2 Mitsubishi Tanabe Pharma Corp Rett Syndrome Drug Details
14.3 Drug Snapshot
14.3.1 Originator
14.3.2 Collaborator/Co-Developer
14.3.3 Route of Administration
14.3.4 Orphan Drug/Fast Track/Special Designation
14.3.5 Geography
14.3.6 Type of Molecular Entity
14.3.7 Current Status
14.4 Drug Overview
14.5 Drug Mechanism of Action
14.6 Clinical/Pre-clinical Trial Details
14.7 Latest Drug Developments

15 Neuren Pharmaceuticals Ltd Rett Syndrome Pipeline Details
15.1 Neuren Pharmaceuticals Ltd Business Profile
15.2 Neuren Pharmaceuticals Ltd Rett Syndrome Drug Details
15.3 Drug Snapshot
15.3.1 Originator
15.3.2 Collaborator/Co-Developer
15.3.3 Route of Administration
15.3.4 Orphan Drug/Fast Track/Special Designation
15.3.5 Geography
15.3.6 Type of Molecular Entity
15.3.7 Current Status
15.4 Drug Overview
15.5 Drug Mechanism of Action
15.6 Clinical/Pre-clinical Trial Details
15.7 Latest Drug Developments

16 Neurolixis Inc Rett Syndrome Pipeline Details
16.1 Neurolixis Inc Business Profile
16.2 Neurolixis Inc Rett Syndrome Drug Details
16.3 Drug Snapshot
16.3.1 Originator
16.3.2 Collaborator/Co-Developer
16.3.3 Route of Administration
16.3.4 Orphan Drug/Fast Track/Special Designation
16.3.5 Geography
16.3.6 Type of Molecular Entity
16.3.7 Current Status
16.4 Drug Overview
16.5 Drug Mechanism of Action
16.6 Clinical/Pre-clinical Trial Details
16.7 Latest Drug Developments

17 Neurotrope Bioscience Inc Rett Syndrome Pipeline Details
17.1 Neurotrope Bioscience Inc Business Profile
17.2 Neurotrope Bioscience Inc Rett Syndrome Drug Details
17.3 Drug Snapshot
17.3.1 Originator
17.3.2 Collaborator/Co-Developer
17.3.3 Route of Administration
17.3.4 Orphan Drug/Fast Track/Special Designation
17.3.5 Geography
17.3.6 Type of Molecular Entity
17.3.7 Current Status
17.4 Drug Overview
17.5 Drug Mechanism of Action
17.6 Clinical/Pre-clinical Trial Details
17.7 Latest Drug Developments

18 Newron Pharmaceuticals SpA Rett Syndrome Pipeline Details
18.1 Newron Pharmaceuticals SpA Business Profile
18.2 Newron Pharmaceuticals SpA Rett Syndrome Drug Details
18.3 Drug Snapshot
18.3.1 Originator
18.3.2 Collaborator/Co-Developer
18.3.3 Route of Administration
18.3.4 Orphan Drug/Fast Track/Special Designation
18.3.5 Geography
18.3.6 Type of Molecular Entity
18.3.7 Current Status
18.4 Drug Overview
18.5 Drug Mechanism of Action
18.6 Clinical/Pre-clinical Trial Details
18.7 Latest Drug Developments

19 OPKO Health Inc Rett Syndrome Pipeline Details
19.1 OPKO Health Inc Business Profile
19.2 OPKO Health Inc Rett Syndrome Drug Details
19.3 Drug Snapshot
19.3.1 Originator
19.3.2 Collaborator/Co-Developer
19.3.3 Route of Administration
19.3.4 Orphan Drug/Fast Track/Special Designation
19.3.5 Geography
19.3.6 Type of Molecular Entity
19.3.7 Current Status
19.4 Drug Overview
19.5 Drug Mechanism of Action
19.6 Clinical/Pre-clinical Trial Details
19.7 Latest Drug Developments

20 Orpheris Inc Rett Syndrome Pipeline Details
20.1 Orpheris Inc Business Profile
20.2 Orpheris Inc Rett Syndrome Drug Details
20.3 Drug Snapshot
20.3.1 Originator
20.3.2 Collaborator/Co-Developer
20.3.3 Route of Administration
20.3.4 Orphan Drug/Fast Track/Special Designation
20.3.5 Geography
20.3.6 Type of Molecular Entity
20.3.7 Current Status
20.4 Drug Overview
20.5 Drug Mechanism of Action
20.6 Clinical/Pre-clinical Trial Details
20.7 Latest Drug Developments

21 PharmatrophiX Inc Rett Syndrome Pipeline Details
21.1 PharmatrophiX Inc Business Profile
21.2 PharmatrophiX Inc Rett Syndrome Drug Details
21.3 Drug Snapshot
21.3.1 Originator
21.3.2 Collaborator/Co-Developer
21.3.3 Route of Administration
21.3.4 Orphan Drug/Fast Track/Special Designation
21.3.5 Geography
21.3.6 Type of Molecular Entity
21.3.7 Current Status
21.4 Drug Overview
21.5 Drug Mechanism of Action
21.6 Clinical/Pre-clinical Trial Details
21.7 Latest Drug Developments

22 Relmada Therapeutics Inc Rett Syndrome Pipeline Details
22.1 Relmada Therapeutics Inc Business Profile
22.2 Relmada Therapeutics Inc Rett Syndrome Drug Details
22.3 Drug Snapshot
22.3.1 Originator
22.3.2 Collaborator/Co-Developer
22.3.3 Route of Administration
22.3.4 Orphan Drug/Fast Track/Special Designation
22.3.5 Geography
22.3.6 Type of Molecular Entity
22.3.7 Current Status
22.4 Drug Overview
22.5 Drug Mechanism of Action
22.6 Clinical/Pre-clinical Trial Details
22.7 Latest Drug Developments

23 Starwise Therapeutics LLC Rett Syndrome Pipeline Details
23.1 Starwise Therapeutics LLC Business Profile
23.2 Starwise Therapeutics LLC Rett Syndrome Drug Details
23.3 Drug Snapshot
23.3.1 Originator
23.3.2 Collaborator/Co-Developer
23.3.3 Route of Administration
23.3.4 Orphan Drug/Fast Track/Special Designation
23.3.5 Geography
23.3.6 Type of Molecular Entity
23.3.7 Current Status
23.4 Drug Overview
23.5 Drug Mechanism of Action
23.6 Clinical/Pre-clinical Trial Details
23.7 Latest Drug Developments

24 Ultragenyx Pharmaceutical Inc Rett Syndrome Pipeline Details
24.1 Ultragenyx Pharmaceutical Inc Business Profile
24.2 Ultragenyx Pharmaceutical Inc Rett Syndrome Drug Details
24.3 Drug Snapshot
24.3.1 Originator
24.3.2 Collaborator/Co-Developer
24.3.3 Route of Administration
24.3.4 Orphan Drug/Fast Track/Special Designation
24.3.5 Geography
24.3.6 Type of Molecular Entity
24.3.7 Current Status
24.4 Drug Overview
24.5 Drug Mechanism of Action
24.6 Clinical/Pre-clinical Trial Details
24.7 Latest Drug Developments

25. Latest Rett Syndrome Drug Pipeline Developments, 2019

26. Appendix
26.1 About Us
26.2 Sources and Methodology
26.3 Contact Information

Companies Mentioned

  • AMO Pharma Ltd

  • Amorsa Therapeutics Inc

  • Anavex Life Sciences Corp

  • ArmaGen Inc

  • AveXis Inc

  • Biohaven Pharmaceutical Holding Company Ltd

  • Cold Spring Harbor Laboratory

  • Eloxx Pharmaceuticals Inc

  • GW Pharmaceuticals Plc

  • Mitochon Pharmaceuticals Inc

  • Mitsubishi Tanabe Pharma Corp

  • Neuren Pharmaceuticals Ltd

  • Neurolixis Inc

  • Neurotrope Bioscience Inc

  • Newron Pharmaceuticals SpA

  • OPKO Health Inc

  • Orpheris Inc

  • PharmatrophiX Inc

  • Relmada Therapeutics Inc

  • Starwise Therapeutics LLC

  • Ultragenyx Pharmaceutical Inc