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The publisher's latest Pharmaceutical and Healthcare disease pipeline guide Rett Syndrome - Drugs In Development, 2022, provides an overview of the Rett Syndrome (Central Nervous System) pipeline landscape.Speak directly to the analyst to clarify any post sales queries you may have.
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
The publisher's Pharmaceutical and Healthcare latest pipeline guide Rett Syndrome - Drugs In Development, 2022, 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 Pre-Registration, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 1, 4, 4, 20 and 13 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 1 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 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.
Table of Contents
- Introduction
- 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
- 1st Bio Therapeutics Inc
- Acadia Pharmaceuticals Inc
- Adge Pharmaceuticals Inc
- Alcyone Therapeutics Inc
- Amicus Therapeutics Inc
- AMO Pharma Ltd
- Ananda Scientific Inc
- Anavex Life Sciences Corp
- ArmaGen Inc
- Biohaven Pharmaceutical Holding Company Ltd
- BioXcel Corp
- Connecta Therapeutics SL
- CuroNZ Ltd
- DepYmed Inc
- Echo Pharmaceuticals BV
- Epeius Pharma Co
- Epigen Biosciences Inc
- EuMentis Therapeutics Inc
- GEXVal Inc
- Herophilus
- Jazz Pharmaceuticals Plc
- LizarBio Therapeutics Inc
- Lucy Therapeutics Inc
- Neuren Pharmaceuticals Ltd
- Neurogene Inc
- Neurolixis Inc
- Novartis Gene Therapies
- OrganAI AB
- PharmatrophiX Inc
- Prilenia Therapeutics Development Ltd
- Q-State Biosciences Inc
- Sarepta Therapeutics Inc
- Shape Therapeutics Inc
- Starwise Therapeutics LLC
- StrideBio Inc
- Synaptogenix Inc
- Taysha Gene Therapies Inc
- Thiogenesis Therapeutics SARL
- Ultragenyx Pharmaceutical Inc
- Unravel Biosciences
- Vaccinex Inc
- Vico Therapeutics BV
- Vyant Bio Inc
- Rett Syndrome - Drug Profiles
- 1ST-301 - Drug Profile
- Product Description
- Mechanism Of Action
- ACTX-101 - Drug Profile
- Product Description
- Mechanism Of Action
- Antisense Oligonucleotide to Activate MECP2 for Rett Syndrome - Drug Profile
- Product Description
- Mechanism Of Action
- Astrocytes - Drug Profile
- Product Description
- Mechanism Of Action
- AVXS-201 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- BHV-5000 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- blarcamesine hydrochloride - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Bryostatin-1 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- BXCL-902 - Drug Profile
- Product Description
- Mechanism Of Action
- cannabidiol - Drug Profile
- Product Description
- Mechanism Of Action
- CTH-120 - Drug Profile
- Product Description
- Mechanism Of Action
- Drugs for Rett Syndrome - Drug Profile
- Product Description
- Mechanism Of Action
- ECP-012A - Drug Profile
- Product Description
- Mechanism Of Action
- elocalcitol - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- EM-036 - Drug Profile
- Product Description
- Mechanism Of Action
- Gene Therapies for Angelman Syndrome, Muscular Dystrophy and Rett Syndrome - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Gene Therapy to Activate MECP2 Protein for Rett Syndrome - Drug Profile
- Product Description
- Mechanism Of Action
- GWP-42006 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- GXV-001 - Drug Profile
- Product Description
- Mechanism Of Action
- LM-22A4 - Drug Profile
- Product Description
- Mechanism Of Action
- Neurodevelopmental Disease - Drug Profile
- Product Description
- Mechanism Of Action
- NGN-401 - Drug Profile
- Product Description
- Mechanism Of Action
- NLX-101 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- NRP-2945 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- pepinemab - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- pridopidine hydrochloride - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Rett Fusion Protein - Drug Profile
- Product Description
- Mechanism Of Action
- Rett Syndrome - Drug Profile
- Product Description
- Mechanism Of Action
- Rett Syndrome - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Rett Syndrome-MECP2 - Drug Profile
- Product Description
- Mechanism Of Action
- RVL-001 - Drug Profile
- Product Description
- Mechanism Of Action
- SHP-101 - Drug Profile
- Product Description
- Mechanism Of Action
- Small Molecule to Agonize mGlu7 for Rett Syndrome - Drug Profile
- Product Description
- Mechanism Of Action
- Small Molecules for Rett Syndrome - Drug Profile
- Product Description
- Mechanism Of Action
- Small Molecules to Agonize Sigma-1 Receptor for Metabolic and Central Nervous System Disorders - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Small Molecules to Inhibit HDAC6 for Rett Syndrome - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Small Molecules to Inhibit PTP1B for Rett Syndrome - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- STRX-230 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- tianeptine - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- triheptanoin - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- trofinetide - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- TSHA-102 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- TTI-0102 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- VYNT-0126 - Drug Profile
- Product Description
- Mechanism Of Action
- Rett Syndrome - Dormant Projects
- Rett Syndrome - Discontinued Products
- Rett Syndrome - Product Development Milestones
- Featured News & Press Releases
- Jul 18, 2022: Acadia Pharmaceuticals submits New Drug Application to the U.S. FDA for Trofinetide for the treatment of Rett Syndrome
- May 18, 2022: Neurogene announces new development program in Rett Syndrome utilizing novel EXACT technology platform
- May 03, 2022: DepYmed receives FDA Rare Pediatric Disease and Orphan Drug Designations for its lead clinical candidate for the treatment of Rett Syndrome
- Mar 29, 2022: Taysha Gene Therapies announces initiation of clinical development of TSHA-102 in Rett Syndrome
- Mar 22, 2022: Acadia Pharmaceuticals to present late-breaking data at the 2022 American Academy of Neurology Annual Meeting (AAN)
- Mar 10, 2022: DepYmed and Monash Biomedicine Discovery Institute to study the role of a new generation of PTP1B inhibitors against a novel intracellular checkpoint in cancer
- Feb 04, 2022: Anavex life sciences provides supplemental information on ANAVEX 2-73 (blarcamesine) AVATAR Phase 3 trial for the treatment of adult patients with Rett syndrome
- Feb 01, 2022: ANAVEX 2-73 (Blarcamesine) AVATAR phase 3 trial met primary and secondary efficacy endpoints
- Dec 07, 2021: Acadia reports positive data from Phase III Rett Syndrome drug trial
- Dec 06, 2021: Acadia Pharmaceuticals announces positive top-line results from the pivotal phase 3 lavender trial of Trofinetide in rett syndrome
- Sep 22, 2021: Taysha receives Orphan Drug Designation from the European Commission for TSHA-102 for the treatment of Rett Syndrome
- Sep 08, 2021: Taysha Gene Therapies to host key opinion leader webinar on TSHA-102 for the treatment of Rett syndrome
- Sep 01, 2021: DAFFODIL Rett syndrome clinical trial for trofinetide in girls aged 2 to 5 years
- Aug 05, 2021: Rett syndrome Phase 3 trial enrolment successfully completed - results in Q4 2021
- Jun 21, 2021: Anavex Life Sciences announces ANAVEX2-73 (Blarcamesine) biomarker correlated with efficacy endpoints in placebo-controlled U.S. phase 2 clinical trial for the treatment of adult patients with Rett syndrome
- Appendix
- Methodology
- Coverage
- Secondary Research
- Primary Research
- Expert Panel Validation
- Contact the Publisher
- Disclaimer
List of Tables
- Number of Products under Development for Rett Syndrome, 2022
- Number of Products under Development by Companies, 2022
- Number of Products under Development by Companies, 2022 (Contd..1)
- 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 Companies, 2022 (Contd..2)
- 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
- Rett Syndrome - Pipeline by 1st Bio Therapeutics Inc, 2022
- Rett Syndrome - Pipeline by Acadia Pharmaceuticals Inc, 2022
- Rett Syndrome - Pipeline by Adge Pharmaceuticals Inc, 2022
- Rett Syndrome - Pipeline by Alcyone Therapeutics Inc, 2022
- Rett Syndrome - Pipeline by Amicus Therapeutics Inc, 2022
- Rett Syndrome - Pipeline by AMO Pharma Ltd, 2022
- Rett Syndrome - Pipeline by Ananda Scientific Inc, 2022
- Rett Syndrome - Pipeline by Anavex Life Sciences Corp, 2022
- Rett Syndrome - Pipeline by ArmaGen Inc, 2022
- Rett Syndrome - Pipeline by Biohaven Pharmaceutical Holding Company Ltd, 2022
- Rett Syndrome - Pipeline by BioXcel Corp, 2022
- Rett Syndrome - Pipeline by Connecta Therapeutics SL, 2022
- Rett Syndrome - Pipeline by CuroNZ Ltd, 2022
- Rett Syndrome - Pipeline by DepYmed Inc, 2022
- Rett Syndrome - Pipeline by Echo Pharmaceuticals BV, 2022
- Rett Syndrome - Pipeline by Epeius Pharma Co, 2022
- Rett Syndrome - Pipeline by Epigen Biosciences Inc, 2022
- Rett Syndrome - Pipeline by EuMentis Therapeutics Inc, 2022
- Rett Syndrome - Pipeline by GEXVal Inc, 2022
- Rett Syndrome - Pipeline by Herophilus, 2022
- Rett Syndrome - Pipeline by Jazz Pharmaceuticals Plc, 2022
- Rett Syndrome - Pipeline by LizarBio Therapeutics Inc, 2022
- Rett Syndrome - Pipeline by Lucy Therapeutics Inc, 2022
- Rett Syndrome - Pipeline by Neuren Pharmaceuticals Ltd, 2022
- Rett Syndrome - Pipeline by Neurogene Inc, 2022
- Rett Syndrome - Pipeline by Neurolixis Inc, 2022
- Rett Syndrome - Pipeline by Novartis Gene Therapies, 2022
- Rett Syndrome - Pipeline by OrganAI AB, 2022
- Rett Syndrome - Pipeline by PharmatrophiX Inc, 2022
- Rett Syndrome - Pipeline by Prilenia Therapeutics Development Ltd, 2022
- Rett Syndrome - Pipeline by Q-State Biosciences Inc, 2022
- Rett Syndrome - Pipeline by Sarepta Therapeutics Inc, 2022
- Rett Syndrome - Pipeline by Shape Therapeutics Inc, 2022
- Rett Syndrome - Pipeline by Starwise Therapeutics LLC, 2022
- Rett Syndrome - Pipeline by StrideBio Inc, 2022
- Rett Syndrome - Pipeline by Synaptogenix Inc, 2022
- Rett Syndrome - Pipeline by Taysha Gene Therapies Inc, 2022
- Rett Syndrome - Pipeline by Thiogenesis Therapeutics SARL, 2022
- Rett Syndrome - Pipeline by Ultragenyx Pharmaceutical Inc, 2022
- Rett Syndrome - Pipeline by Unravel Biosciences, 2022
- Rett Syndrome - Pipeline by Vaccinex Inc, 2022
- Rett Syndrome - Pipeline by Vico Therapeutics BV, 2022
- Rett Syndrome - Pipeline by Vyant Bio Inc, 2022
- Rett Syndrome - Dormant Projects, 2022
- Rett Syndrome - Dormant Projects, 2022 (Contd..1)
- Rett Syndrome - Discontinued Products, 2022
List of Figures
- Number of Products under Development for Rett Syndrome, 2022
- Number of Products under Development by Companies, 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 Routes of Administration, 2022
- Number of Products by Stage and Routes of Administration, 2022
- Number of Products by Molecule Types, 2022
- Number of Products by Stage and Molecule Types, 2022
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- 1st Bio Therapeutics Inc
- Acadia Pharmaceuticals Inc
- Adge Pharmaceuticals Inc
- Alcyone Therapeutics Inc
- Amicus Therapeutics Inc
- AMO Pharma Ltd
- Ananda Scientific Inc
- Anavex Life Sciences Corp
- ArmaGen Inc
- Biohaven Pharmaceutical Holding Company Ltd
- BioXcel Corp
- Connecta Therapeutics SL
- CuroNZ Ltd
- DepYmed Inc
- Echo Pharmaceuticals BV
- Epeius Pharma Co
- Epigen Biosciences Inc
- EuMentis Therapeutics Inc
- GEXVal Inc
- Herophilus
- Jazz Pharmaceuticals Plc
- LizarBio Therapeutics Inc
- Lucy Therapeutics Inc
- Neuren Pharmaceuticals Ltd
- Neurogene Inc
- Neurolixis Inc
- Novartis Gene Therapies
- OrganAI AB
- PharmatrophiX Inc
- Prilenia Therapeutics Development Ltd
- Q-State Biosciences Inc
- Sarepta Therapeutics Inc
- Shape Therapeutics Inc
- Starwise Therapeutics LLC
- StrideBio Inc
- Synaptogenix Inc
- Taysha Gene Therapies Inc
- Thiogenesis Therapeutics SARL
- Ultragenyx Pharmaceutical Inc
- Unravel Biosciences
- Vaccinex Inc
- Vico Therapeutics BV
- Vyant Bio Inc
