Rett Syndrome - Pipeline Review, H2 2019

Publisher Name :
Date: 16-Dec-2019
No. of pages: 165
Inquire Before Buying

Rett Syndrome - Pipeline Review, H2 2019

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Rett Syndrome - Pipeline Review, H2 2019, provides an overview of the Rett Syndrome (Central Nervous System) pipeline landscape.

Rett syndrome is a neurodevelopmental disorder. This is caused by genetic mutation on MECP2 gene. Rett syndrome is described in four stages: Stage I or early onset, Stage II or rapid destruction, Stage III, or the plateau and Stage IV, or the late motor deterioration stage. Symptoms include slowed growth, loss of normal movement and coordination, loss of communication abilities, breathing problems, pain, irregular heartbeat and seizures. Risk factors include family history and gender. Treatment includes disease modifying drugs.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Rett Syndrome - Pipeline Review, H2 2019, provides comprehensive information on the therapeutics under development for Rett Syndrome (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Rett Syndrome (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Rett Syndrome and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 3, 6, 4, 8 and 4 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 5 molecules, respectively.

Rett Syndrome (Central Nervous System) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Rett Syndrome (Central Nervous System).

- The pipeline guide reviews pipeline therapeutics for Rett Syndrome (Central Nervous System) by companies and universities/research institutes based on information derived from company and industry-specific sources.

- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.

- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.

- The pipeline guide reviews key companies involved in Rett Syndrome (Central Nervous System) therapeutics and enlists all their major and minor projects.

- The pipeline guide evaluates Rett Syndrome (Central Nervous System) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.

- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.

- The pipeline guide reviews latest news related to pipeline therapeutics for Rett Syndrome (Central Nervous System)

Reasons to buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.

- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.

- Find and recognize significant and varied types of therapeutics under development for Rett Syndrome (Central Nervous System).

- Classify potential new clients or partners in the target demographic.

- Develop tactical initiatives by understanding the focus areas of leading companies.

- Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.

- Formulate corrective measures for pipeline projects by understanding Rett Syndrome (Central Nervous System) pipeline depth and focus of Indication therapeutics.

- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.

- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Rett Syndrome - Pipeline Review, H2 2019

Table of Contents
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Rett Syndrome - Overview
Rett Syndrome - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Rett Syndrome - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Rett Syndrome - Companies Involved in Therapeutics Development
AMO Pharma Ltd
Amorsa Therapeutics Inc
Anavex Life Sciences Corp
ArmaGen Inc
AveXis Inc
BioElectron Technology Corp
Biohaven Pharmaceutical Holding Company Ltd
BioXcel Corp
DepYmed Inc
Eloxx Pharmaceuticals Inc
Epigen Biosciences Inc
GW Pharmaceuticals Plc
Lucy Therapeutics Inc
Mitochon Pharmaceuticals Inc
Mitsubishi Tanabe Pharma Corp
Neuren Pharmaceuticals Ltd
Neurolixis Inc
Neurotrope Bioscience Inc
Newron Pharmaceuticals SpA
PharmatrophiX Inc
Relmada Therapeutics Inc
Starwise Therapeutics LLC
Thiogenesis Therapeutics SARL
Ultragenyx Pharmaceutical Inc
Rett Syndrome - Drug Profiles
AVXS-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BHV-5000 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
blarcamesine hydrochloride - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Bryostatin-1 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BXCL-902 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
cannabidiol - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CPT-157633 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Drugs for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ELX-02 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
fingolimod hydrochloride - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Fusion Protein for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene therapy to Activate MECP2 for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate MECP2 Protein for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
GWP-42006 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LM-22A4 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
MP-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NLX-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
REL-1017 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
sarizotan hydrochloride - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecule to Agonize mGlu5 for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecule to Agonize mGlu7 for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Agonize Sigma-1 Receptor for Metabolic and entral Nervous System Disorders - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Antagonize NMDA Receptor for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Inhibit HDAC6 for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Inhibit PTP1B for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
tianeptine - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
triheptanoin - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
trofinetide - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
TTI-0102 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
vatiquinone - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Rett Syndrome - Dormant Projects
Rett Syndrome - Product Development Milestones
Featured News & Press Releases
Nov 19, 2019: Newron receives FDA Rare Pediatric Disease Designation for Sarizotan for the treatment of Rett Syndrome
Nov 14, 2019: Anavex Life Sciences receives Rare Pediatric Disease Designation from FDA for ANAVEX2-73 (blarcamesine) for the treatment of Rett Syndrome
Nov 06, 2019: Anavex Life Sciences announces publication of foundational data for ANAVEX2-73 (blarcamesine) in Rett Syndrome
Oct 31, 2019: Acadia initiates Phase III study of trofinetide to treat Rett syndrome
Oct 02, 2019: Anavex Life Sciences Issued New U.S. Patent for Composition of Matter Covering ANAVEX2-73 for the Treatment of Alzheimer's Disease
Sep 27, 2019: Anavex Life Sciences presented at the 6th Annual European Rett Syndrome Conference preliminary clinical efficacy data from the U.S. Rett Syndrome Phase 2 ANAVEX2-73 Study
Sep 16, 2019: Anavex Life Sciences announces preliminary clinical efficacy data of its U.S. phase 2 clinical trial of ANAVEX 2-73 in patients with Rett Syndrome
Sep 04, 2019: Anavex Life Sciences announces initiation of the EXCELLENCE ANAVEX 2-73-RS-003 clinical study in pediatric patients with Rett Syndrome
Jul 31, 2019: Anavex Life Sciences reports recent data review by the independent data safety monitoring board for its US phase 2 clinical trial of ANAVEX 2-73 in patients with Rett syndrome
Jul 29, 2019: Anavex Life Sciences receives positive opinion for orphan designation from the European Medicines Agency for ANAVEX2-73 for the treatment of rett syndrome
Jul 17, 2019: Anavex Life Sciences presents new clinical data identifying gut microbiota biomarkers asociated with improved clinical response in patients treated with ANAVEX2-73 at 2019 Alzheimer's Association International Conference (AAIC)
Jun 25, 2019: Anavex Life Sciences announces first patient dosed in extension study to its U.S. phase 2 clinical trial of ANAVEX2-73 in patients for Rett Syndrome
Jun 06, 2019: Anavex Life Sciences announces first patient dosed in Phase 2 clinical trial of ANAVEX2-73 for the treatment of Rett Syndrome (AVATAR)
May 08, 2019: Rett Syndrome Association of Australia and Anavex Life Sciences announce initiation of the phase 2 AVATAR clinical study in patients with Rett Syndrome
Mar 27, 2019: Positive phase 2 study results of Trofinetide in pediatric Rett Syndrome published in Neurology, the medical journal of the American Academy of Neurology
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Number of Products under Development for Rett Syndrome, H2 2019
Number of Products under Development by Companies, H2 2019
Number of Products under Development by Companies, H2 2019 (Contd..1), H2 2019
Number of Products under Development by Universities/Institutes, H2 2019
Products under Development by Companies, H2 2019
Products under Development by Companies, H2 2019 (Contd..1), H2 2019
Products under Development by Universities/Institutes, H2 2019
Number of Products by Stage and Target, H2 2019
Number of Products by Stage and Mechanism of Action, H2 2019
Number of Products by Stage and Route of Administration, H2 2019
Number of Products by Stage and Molecule Type, H2 2019
Rett Syndrome - Pipeline by AMO Pharma Ltd, H2 2019
Rett Syndrome - Pipeline by Amorsa Therapeutics Inc, H2 2019
Rett Syndrome - Pipeline by Anavex Life Sciences Corp, H2 2019
Rett Syndrome - Pipeline by ArmaGen Inc, H2 2019
Rett Syndrome - Pipeline by AveXis Inc, H2 2019
Rett Syndrome - Pipeline by BioElectron Technology Corp, H2 2019
Rett Syndrome - Pipeline by Biohaven Pharmaceutical Holding Company Ltd, H2 2019
Rett Syndrome - Pipeline by BioXcel Corp, H2 2019
Rett Syndrome - Pipeline by DepYmed Inc, H2 2019
Rett Syndrome - Pipeline by Eloxx Pharmaceuticals Inc, H2 2019
Rett Syndrome - Pipeline by Epigen Biosciences Inc, H2 2019
Rett Syndrome - Pipeline by GW Pharmaceuticals Plc, H2 2019
Rett Syndrome - Pipeline by Lucy Therapeutics Inc, H2 2019
Rett Syndrome - Pipeline by Mitochon Pharmaceuticals Inc, H2 2019
Rett Syndrome - Pipeline by Mitsubishi Tanabe Pharma Corp, H2 2019
Rett Syndrome - Pipeline by Neuren Pharmaceuticals Ltd, H2 2019
Rett Syndrome - Pipeline by Neurolixis Inc, H2 2019
Rett Syndrome - Pipeline by Neurotrope Bioscience Inc, H2 2019
Rett Syndrome - Pipeline by Newron Pharmaceuticals SpA, H2 2019
Rett Syndrome - Pipeline by PharmatrophiX Inc, H2 2019
Rett Syndrome - Pipeline by Relmada Therapeutics Inc, H2 2019
Rett Syndrome - Pipeline by Starwise Therapeutics LLC, H2 2019
Rett Syndrome - Pipeline by Thiogenesis Therapeutics SARL, H2 2019
Rett Syndrome - Pipeline by Ultragenyx Pharmaceutical Inc, H2 2019
Rett Syndrome - Dormant Projects, H2 2019

List of Figures
Number of Products under Development for Rett Syndrome, H2 2019
Number of Products under Development by Companies, H2 2019
Number of Products by Top 10 Targets, H2 2019
Number of Products by Stage and Top 10 Targets, H2 2019
Number of Products by Top 10 Mechanism of Actions, H2 2019
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2019
Number of Products by Routes of Administration, H2 2019
Number of Products by Stage and Routes of Administration, H2 2019
Number of Products by Molecule Types, H2 2019
Number of Products by Stage and Molecule Types, H2 2019
  • Central Nervous System Disorder Drugs Industry Forecasts - China Focus
    Published: 27-Nov-2023        Price: US 1800 Onwards        Pages: 116
    This study focuses on China's Central Nervous System Disorder Drugs industry forecasts. In the two past decades, the industry has been growing at a fast pace. The dramatic expansions of the manufacturing capabilities and rising consumer consumptions in China have transformed China's society and economy. China is one of the world's major producers for industrial and consumer products. Far outpacing other economies in the world, China is the world's fastest growing market for the consumptions of g......
  • Central Nervous System Disorder Drugs Companies in China
    Published: 27-Nov-2023        Price: US 1800 Onwards        Pages: 48
    This study focuses on China's Central Nervous System Disorder Drugs industry assessments and company profiles. In the two past decades, the industry has been growing at a fast pace. The dramatic expansions of the manufacturing capabilities and rising consumer consumptions in China have transformed China's society and economy. China is one of the world's major producers for industrial and consumer products. Far outpacing other economies in the world, China is the world's fastest growing market fo......
  • Central Nervous System Disorder Drugs Markets in China
    Published: 27-Nov-2023        Price: US 4000 Onwards        Pages: 214
    China's demand for Central Nervous System Disorder Drugs has grown at a fast pace in the past decade. In the next decade, both production and demand will continue to grow. The Chinese economy maintains a high speed growth which has been stimulated by the consecutive increases of industrial output, imports & exports, consumer consumption and capital investment for over two decades. This new study examines China's economic trends, investment environment, industry development, supply and demand, in......
  • Global Drugs of Nervous System Industry Research Report, In-depth Analysis of Current Status and Outlook of Key Countries 2023-2028
    Published: 14-Nov-2023        Price: US 3380 Onwards        Pages: 108
    Market Overview of Global Drugs of Nervous System market: According to our latest research, the global Drugs of Nervous System market looks promising in the next 5 years. As of 2022, the global Drugs of Nervous System market was estimated at USD XX million, and it's anticipated to reach USD XX million in 2028, with a CAGR of XX% during the forecast years. Drugs of Nervous System are drugs used for the treatment of diseases affecting the brain or spinal cord such as epilepsy, ......
  • Central Nervous System Collaboration and Licensing Deals 2016-2023
    Published: 01-Oct-2023        Price: US 3995 Onwards        Pages: 650
    Central Nervous System Collaboration and Licensing Deals provides a comprehensive understanding and unprecedented access to the central nervous system deals entered into by the worlds leading biopharma companies. Fully revised and updated, the report provides details of central nervous system deals from 2016 to 2023. The report provides access to deal payment terms as announced between the parties. This data provides useful insight into the payment and other deal te......
  • Global Drugs of Nervous System Market Insights: Impact of COVID-19 and Future Expectations to 2028
    Published: 24-Feb-2023        Price: US 3260 Onwards        Pages: 123
    Drugs of Nervous System are drugs used for the treatment of diseases affecting the brain or spinal cord such as epilepsy, Parkinson's disease, Alzheimer's disease, depression, anxiety and bipolar disorders. Epilepsy is a chronic brain disorder characterized by recurring seizures. Parkinson's disease is a degenerative CNS disorder associated with symptoms including tremor, rigidity, akinesia and bradykinesia. The Drugs of Nervous System market revenue was XX Million USD in 2022, and will r......
  • Global Drugs of Nervous System Market Research Report 2022 - Market Size, Current Insights and Development Trends
    Published: 26-Jan-2023        Price: US 3450 Onwards        Pages: 111
    Drugs of Nervous System are drugs used for the treatment of diseases affecting the brain or spinal cord such as epilepsy, Parkinson's disease, Alzheimer's disease, depression, anxiety and bipolar disorders. Epilepsy is a chronic brain disorder characterized by recurring seizures. Parkinson's disease is a degenerative CNS disorder associated with symptoms including tremor, rigidity, akinesia and bradykinesia. The report focuses on the Drugs of Nervous System market size, segment si......
  • Encephalopathy Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
    Published: 30-Nov-2022        Price: US 2000 Onwards        Pages: 71
    Encephalopathy Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Encephalopathy - Drugs In Development, 2022, provides an overview of the Encephalopathy (Central Nervous System) pipeline landscape. Encephalopathy is a term for any diffuse disease of the brain that alters brain function or structure. Symptoms include seizures......
  • Metachromatic Leukodystrophy (MLD) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
    Published: 30-Nov-2022        Price: US 2000 Onwards        Pages: 40
    Metachromatic Leukodystrophy (MLD) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Metachromatic Leukodystrophy (MLD) - Drugs In Development, 2022, provides an overview of the Metachromatic Leukodystrophy (MLD) (Central Nervous System) pipeline landscape. Metachromatic leukodystrophy is an inherited disorder characterized ......
  • SERVICES
    Value for Money
    We believe in "optimum utilization of available budget and resources". While servicing our clients' (your) market research requirements, we keep the same approach in focus to help you get the best value for your $$s.
    Ever Growing Inventory
    Ranging from the smallest feasible / required data (datasheets, data facts, SWOT analysis, company profiles, etc) to full research reports that help you make decisions, our inventory is updated almost on a daily basis with the latest industry reports from domain experts that track more than 5000 niche sectors.
    One Stop Solution
    Need a custom research report on medical devices market? Require all available business intelligence on 3D printing industry? Exploring F&B sector of a particular country/region? RnRMarketResearch.com is your one-stop-solution to all market intelligence needs. We not only offer custom research and consulting services, we also "bundle" reports to meet your needs and help you fetch the data analysis you require for your business.
    Dedicated Client Engagement
    Not limited to only "finding" relevant reports for you, our client engagement team dedicates its efforts to understand your "business need" and accordingly maps available research data to help you move forward. Call "your" client engagement executive any time of your day and get your questions answered in order to make the correct business decision.
    Saving Time and Efforts
    Simply share your research requirement details with us and let us do all the hard work to find required intelligence for you. When you add up our "one stop solution" and "dedicated client engagement" services mentioned above, you obviously know the time and effort saving you do by working with us.
    Payment Flexibility
    Working with Fortune 500 organizations, we understand the importance of being flexible for payments. Share your payment terms with us and we will surely match up to them to ensure you get access to required business intelligence data without having to wait for the payment to be done.
    Post-Purchase Research Support
    Have questions after reading a report / datasheet bought through us? Not sure about the methodology used for data available in the research? Talk to us / Share your questions with us and if required, we will connect you with the analyst(s)/author(s) of the report(s) and ensure you get satisfactory answers for the same. Need more data / analysis / report(s) on the topic of your research/project? The RnRMarketResearch.com team is here for you 24X7 to support you with your post-purchase requirements. Subscription Offers & Packages (Get in touch with us for more details - [email protected] / +1 888 391 5441 )
    • Ad Hoc
    • Pay - as - you - go / Bucket Subscriptions
    • Fixed Cost for #of reports
    • Customize / Personalize as per your needs