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

The 2020 Rett Syndrome pipeline report presents a comprehensive overview of the research and development of Rett Syndrome drug candidates. It presents drugs in development that could potentially reach the market in the next 5 to 10 years: One drug in Research phase, nine drugs in pre-clinical phase, four drugs in Phase 1, six drugs in Phase 2, and three drugs in Phase 3.

As of February 2020, the Rett Syndrome pipeline remains robust with 27 therapeutic candidates under development. An increasing number of companies are actively participating in the development of Rett Syndrome treatment. Diverse types of targeted therapies are being explored through clinical trials including 5-HT1A receptor agonist; Cannabinoid receptor modulators; Fatty acid replacements; Glutamate modulators; G-protein coupled receptors (GPR55) antagonist; HDAC6 protein inhibitors; M1 muscarinic agonist; Methyl-CpG-binding protein 2 expression stimulants; Mitochondrial protein modulators; NADPH quinone oxidase 1 (NQO1) modulators; Neuropeptide receptor modulators; NMDA receptor antagonists; Protein kinase C stimulants; protein tyrosine phosphatase 1B (PTP 1B) inhibitor; Ribosomal protein modulators; Sigma-1 receptor agonists; Sphingosine 1-phosphate receptor antagonist; Triglyceride replacements; TrkB receptor.

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 Rett Syndrome 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

The publisher's 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 Rett Syndrome development
Rett Syndrome pipeline compounds and molecules under study by both large scale and small companies are included in the report
Rett Syndrome 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 Rett Syndrome pipeline candidates included
Business overview and snapshot of all companies involved in Rett Syndrome pipeline are included
Latest market and pipeline developments are provided in the report

Rett Syndrome pipeline companies included in the report are: AMO Pharma Ltd, Amorsa Therapeutics Inc, Anavex Life Sciences Corp, ArmaGen Inc, AveXis Inc, BioElectron Technology Corp, Biohaven Pharmaceutical Holding Company Ltd, BioXcel Corp, Cold Spring Harbor Laboratory, DepYmed Inc, Eloxx Pharmaceuticals Inc, Epigen Biosciences Inc, GW Pharmaceuticals Plc, Lucy Therapeutics Inc, Mitochon Pharmaceuticals Inc, Neuren Pharmaceuticals Ltd, Neurolixis Inc, Neurotrope Bioscience Inc, Newron Pharmaceuticals SpA, Novartis AG, PharmatrophiX Inc, Relmada Therapeutics Inc, Sarepta Therapeutics, Starwise Therapeutics LLC, Thiogenesis Therapeutics SARL, Ultragenyx Pharmaceutical Inc.

Rett Syndrome pipeline drugs profiled in the report include: AMO-04, NMDA receptor antagonists for Rett Syndrome, ANAVEX 2-73 (blarcamesine hydrochloride), Rett fusion protein, AVXS-201, vatiquinone (EPI-743), BHV-5000, BXCL902, CPT157633, PTP1B inhibitor for Rett Syndrome, ELX-02, Sigma 1 receptor for Rett Syndrome, Epidiolex (cannabidiol), GWP42006, Drugs for Rett Syndrome by Lucy Therapeutics Inc, MP101, FTY720 (fingolimod hydrochloride), trofinetide (NNZ-2566), NLX-101, Bryostatin-1, sarizotan hydrochloride, LM 22A4, REL-1017 (dextromethadone), HDAC6 inhibitors for Rett Syndrome, TTI-0102, triheptanoin, AAV based gene therapy.

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 Rett Syndrome Condition
2.3 Rett Syndrome Pipeline Snapshot, 2020
2.4 Companies investing in Rett Syndrome pipeline therapeutics
2.5 Phase wise Rett Syndrome Pipeline Candidates
2.6 Most Researched Mechanism of Action of Rett Syndrome Pipeline Products
2.7 Route of Administration of Rett Syndrome Pipeline Drugs

3. Companies Active in Pipeline Development

3.1 AMO Pharma Ltd Overview, Contacts and ASD Pipeline Drugs
3.2 Amorsa Therapeutics Inc Overview, Contacts and ASD Pipeline Drugs
3.3 Anavex Life Sciences Corp Overview, Contacts and ASD Pipeline Drugs
3.4 ArmaGen Inc Overview, Contacts and ASD Pipeline Drugs
3.5 AveXis Inc Overview, Contacts and ASD Pipeline Drugs
3.6 BioElectron Technology Corp Overview, Contacts and ASD Pipeline Drugs
3.7 Biohaven Pharmaceutical Holding Company Ltd Overview, Contacts and ASD Pipeline Drugs
3.8 BioXcel Corp Overview, Contacts and ASD Pipeline Drugs
3.9 Cold Spring Harbor Laboratory Overview, Contacts and ASD Pipeline Drugs
3.10 DepYmed Inc Overview, Contacts and ASD Pipeline Drugs
3.11 Eloxx Pharmaceuticals Inc Overview, Contacts and ASD Pipeline Drugs
3.12 Epigen Biosciences Inc Overview, Contacts and ASD Pipeline Drugs
3.13 GW Pharmaceuticals Plc Overview, Contacts and ASD Pipeline Drugs
3.14 Lucy Therapeutics Inc Overview, Contacts and ASD Pipeline Drugs
3.15 Mitochon Pharmaceuticals Inc Overview, Contacts and ASD Pipeline Drugs
3.16 Neuren Pharmaceuticals Ltd Overview, Contacts and ASD Pipeline Drugs
3.17 Neurolixis Inc Overview, Contacts and ASD Pipeline Drugs
3.18 Neurotrope Bioscience Inc Overview, Contacts and ASD Pipeline Drugs
3.19 Newron Pharmaceuticals SpA Overview, Contacts and ASD Pipeline Drugs
3.20 Novartis AG Overview, Contacts and ASD Pipeline Drugs
3.21 PharmatrophiX Inc Overview, Contacts and ASD Pipeline Drugs
3.22 Relmada Therapeutics Inc Overview, Contacts and ASD Pipeline Drugs
3.23 Sarepta Therapeutics Overview, Contacts and ASD Pipeline Drugs
3.24 Starwise Therapeutics LLC Overview, Contacts and ASD Pipeline Drugs
3.25 Thiogenesis Therapeutics SARL Overview, Contacts and ASD Pipeline Drugs
3.26 Ultragenyx Pharmaceutical Inc Overview, Contacts and ASD Pipeline Drugs

4. Active Pipeline Drug Details, 2020

4.1 AMO-04 Drug Details
4.1.1 AMO-04 Current Status
4.1.2 AMO-04 Drug Overview
4.1.3 AMO-04 Mechanism of Action
4.1.4 AMO-04 Licensing/Collaboration Companies
4.1.5 AMO-04 Clinical Trials
4.2 NMDA receptor antagonists for Rett Syndrome Drug Details
4.2.1 NMDA receptor antagonists for Rett Syndrome Current Status
4.2.2 NMDA receptor antagonists for Rett Syndrome Drug Overview
4.2.3 NMDA receptor antagonists for Rett Syndrome Mechanism of Action
4.2.4 NMDA receptor antagonists for Rett Syndrome Licensing/Collaboration Companies
4.2.5 NMDA receptor antagonists for Rett Syndrome Clinical Trials
4.3 ANAVEX 2-73 (blarcamesine hydrochloride) Drug Details
4.3.1 ANAVEX 2-73 (blarcamesine hydrochloride) Current Status
4.3.2 ANAVEX 2-73 (blarcamesine hydrochloride) Drug Overview
4.3.3 ANAVEX 2-73 (blarcamesine hydrochloride) Mechanism of Action
4.3.4 ANAVEX 2-73 (blarcamesine hydrochloride) Licensing/Collaboration Companies
4.3.5 ANAVEX 2-73 (blarcamesine hydrochloride) Clinical Trials
4.4 Rett fusion protein Drug Details
4.4.1 Rett fusion protein Current Status
4.4.2 Rett fusion protein Drug Overview
4.4.3 Rett fusion protein Mechanism of Action
4.4.4 Rett fusion protein Licensing/Collaboration Companies
4.4.5 Rett fusion protein Clinical Trials
4.5 AVXS-201 Drug Details
4.5.1 AVXS-201 Current Status
4.5.2 AVXS-201 Drug Overview
4.5.3 AVXS-201 Mechanism of Action
4.5.4 AVXS-201 Licensing/Collaboration Companies
4.5.5 AVXS-201 Clinical Trials
4.6 vatiquinone (EPI-743) Drug Details
4.6.1 vatiquinone (EPI-743) Current Status
4.6.2 vatiquinone (EPI-743) Drug Overview
4.6.3 vatiquinone (EPI-743) Mechanism of Action
4.6.4 vatiquinone (EPI-743) Licensing/Collaboration Companies
4.6.5 vatiquinone (EPI-743) Clinical Trials
4.7 BHV-5000 Drug Details
4.7.1 BHV-5000 Current Status
4.7.2 BHV-5000 Drug Overview
4.7.3 BHV-5000 Mechanism of Action
4.7.4 BHV-5000 Licensing/Collaboration Companies
4.7.5 BHV-5000 Clinical Trials
4.8 BXCL902 Drug Details
4.8.1 BXCL902 Current Status
4.8.2 BXCL902 Drug Overview
4.8.3 BXCL902 Mechanism of Action
4.8.4 BXCL902 Licensing/Collaboration Companies
4.8.5 BXCL902 Clinical Trials
4.9 CPT157633 Drug Details
4.9.1 CPT157633 Current Status
4.9.2 CPT157633 Drug Overview
4.9.3 CPT157633 Mechanism of Action
4.9.4 CPT157633 Licensing/Collaboration Companies
4.9.5 CPT157633 Clinical Trials
4.10 PTP1B inhibitor for Rett Syndrome Drug Details
4.10.1 PTP1B inhibitor for Rett Syndrome Current Status
4.10.2 PTP1B inhibitor for Rett Syndrome Drug Overview
4.10.3 PTP1B inhibitor for Rett Syndrome Mechanism of Action
4.10.4 PTP1B inhibitor for Rett Syndrome Licensing/Collaboration Companies
4.10.5 PTP1B inhibitor for Rett Syndrome Clinical Trials
4.11 ELX-02 Drug Details
4.11.1 ELX-02 Current Status
4.11.2 ELX-02 Drug Overview
4.11.3 ELX-02 Mechanism of Action
4.11.4 ELX-02 Licensing/Collaboration Companies
4.11.5 ELX-02 Clinical Trials
4.12 Sigma 1 receptor for Rett Syndrome Drug Details
4.12.1 Sigma 1 receptor for Rett Syndrome Current Status
4.12.2 Sigma 1 receptor for Rett Syndrome Drug Overview
4.12.3 Sigma 1 receptor for Rett Syndrome Mechanism of Action
4.12.4 Sigma 1 receptor for Rett Syndrome Licensing/Collaboration Companies
4.12.5 Sigma 1 receptor for Rett Syndrome Clinical Trials
4.13 Epidiolex (cannabidiol) Drug Details
4.13.1 Epidiolex (cannabidiol) Current Status
4.13.2 Epidiolex (cannabidiol) Drug Overview
4.13.3 Epidiolex (cannabidiol) Mechanism of Action
4.13.4 Epidiolex (cannabidiol) Licensing/Collaboration Companies
4.13.5 Epidiolex (cannabidiol) Clinical Trials
4.14 GWP42006 Drug Details
4.14.1 GWP42006 Current Status
4.14.2 GWP42006 Drug Overview
4.14.3 GWP42006 Mechanism of Action
4.14.4 GWP42006 Licensing/Collaboration Companies
4.14.5 GWP42006 Clinical Trials
4.15 Drugs for Rett Syndrome by Lucy Therapeutics Inc Drug Details
4.15.1 Drugs for Rett Syndrome by Lucy Therapeutics Inc Current Status
4.15.2 Drugs for Rett Syndrome by Lucy Therapeutics Inc Drug Overview
4.15.3 Drugs for Rett Syndrome by Lucy Therapeutics Inc Mechanism of Action
4.15.4 Drugs for Rett Syndrome by Lucy Therapeutics Inc Licensing/Collaboration Companies
4.15.5 Drugs for Rett Syndrome by Lucy Therapeutics Inc Clinical Trials
4.16 MP101 Drug Details
4.16.1 MP101 Current Status
4.16.2 MP101 Drug Overview
4.16.3 MP101 Mechanism of Action
4.16.4 MP101 Licensing/Collaboration Companies
4.16.5 MP101 Clinical Trials
4.17 FTY720 (fingolimod hydrochloride) Drug Details
4.17.1 FTY720 (fingolimod hydrochloride) Current Status
4.17.2 FTY720 (fingolimod hydrochloride) Drug Overview
4.17.3 FTY720 (fingolimod hydrochloride) Mechanism of Action
4.17.4 FTY720 (fingolimod hydrochloride) Licensing/Collaboration Companies
4.17.5 FTY720 (fingolimod hydrochloride) Clinical Trials
4.18 trofinetide (NNZ-2566) Drug Details
4.18.1 trofinetide (NNZ-2566) Current Status
4.18.2 trofinetide (NNZ-2566) Drug Overview
4.18.3 trofinetide (NNZ-2566) Mechanism of Action
4.18.4 trofinetide (NNZ-2566) Licensing/Collaboration Companies
4.18.5 trofinetide (NNZ-2566) Clinical Trials
4.19 NLX-101 Drug Details
4.19.1 NLX-101 Current Status
4.19.2 NLX-101 Drug Overview
4.19.3 NLX-101 Mechanism of Action
4.19.4 NLX-101 Licensing/Collaboration Companies
4.19.5 NLX-101 Clinical Trials
4.20 Bryostatin-1 Drug Details
4.20.1 Bryostatin-1 Current Status
4.20.2 Bryostatin-1 Drug Overview
4.20.3 Bryostatin-1 Mechanism of Action
4.20.4 Bryostatin-1 Licensing/Collaboration Companies
4.20.5 Bryostatin-1 Clinical Trials
4.21 sarizotan hydrochloride Drug Details
4.21.1 sarizotan hydrochloride Current Status
4.21.2 sarizotan hydrochloride Drug Overview
4.21.3 sarizotan hydrochloride Mechanism of Action
4.21.4 sarizotan hydrochloride Licensing/Collaboration Companies
4.21.5 sarizotan hydrochloride Clinical Trials
4.22 LM 22A4 Drug Details
4.22.1 LM 22A4 Current Status
4.22.2 LM 22A4 Drug Overview
4.22.3 LM 22A4 Mechanism of Action
4.22.4 LM 22A4 Licensing/Collaboration Companies
4.22.5 LM 22A4 Clinical Trials
4.23 REL-1017 (dextromethadone) Drug Details
4.23.1 REL-1017 (dextromethadone) Current Status
4.23.2 REL-1017 (dextromethadone) Drug Overview
4.23.3 REL-1017 (dextromethadone) Mechanism of Action
4.23.4 REL-1017 (dextromethadone) Licensing/Collaboration Companies
4.23.5 REL-1017 (dextromethadone) Clinical Trials
4.24 HDAC6 inhibitors for Rett Syndrome Drug Details
4.24.1 HDAC6 inhibitors for Rett Syndrome Current Status
4.24.2 HDAC6 inhibitors for Rett Syndrome Drug Overview
4.24.3 HDAC6 inhibitors for Rett Syndrome Mechanism of Action
4.24.4 HDAC6 inhibitors for Rett Syndrome Licensing/Collaboration Companies
4.24.5 HDAC6 inhibitors for Rett Syndrome Clinical Trials
4.25 TTI-0102 Drug Details
4.25.1 TTI-0102 Current Status
4.25.2 TTI-0102 Drug Overview
4.25.3 TTI-0102 Mechanism of Action
4.25.4 TTI-0102 Licensing/Collaboration Companies
4.25.5 TTI-0102 Clinical Trials
4.26 triheptanoin Drug Details
4.26.1 triheptanoin Current Status
4.26.2 triheptanoin Drug Overview
4.26.3 triheptanoin Mechanism of Action
4.26.4 triheptanoin Licensing/Collaboration Companies
4.26.5 triheptanoin Clinical Trials
4.27 AAV based gene therapy Drug Details
4.27.1 AAV based gene therapy Current Status
4.27.2 AAV based gene therapy Drug Overview
4.27.3 AAV based gene therapy Mechanism of Action
4.27.4 AAV based gene therapy Licensing/Collaboration Companies
4.27.5 AAV based gene therapy Clinical Trials

5. Latest Rett Syndrome Pipeline News and Deals

6. Appendix

6.1 Our Databases and Reports
6.2 Research Methodolgy