Rett Syndrome Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Rett Syndrome 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 Rett Syndrome - Drugs In Development, 2022, 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 - 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 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.


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
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
ACTX-101 – Drug Profile
Antisense Oligonucleotide to Activate MECP2 for Rett Syndrome – Drug Profile
Astrocytes – Drug Profile
AVXS-201 – Drug Profile
BHV-5000 – Drug Profile
blarcamesine hydrochloride – Drug Profile
Bryostatin-1 – Drug Profile
BXCL-902 – Drug Profile
cannabidiol – Drug Profile
CTH-120 – Drug Profile
Drugs for Rett Syndrome – Drug Profile
ECP-012A – Drug Profile
elocalcitol – Drug Profile
EM-036 – Drug Profile
Gene Therapies for Angelman Syndrome, Muscular Dystrophy and Rett Syndrome – Drug Profile
Gene Therapy to Activate MECP2 Protein for Rett Syndrome – Drug Profile
GWP-42006 – Drug Profile
GXV-001 – Drug Profile
LM-22A4 – Drug Profile
Neurodevelopmental Disease – Drug Profile
NGN-401 – Drug Profile
NLX-101 – Drug Profile
NRP-2945 – Drug Profile
pepinemab – Drug Profile
pridopidine hydrochloride – Drug Profile
Rett Fusion Protein – Drug Profile
Rett Syndrome – Drug Profile
Rett Syndrome-MECP2 – Drug Profile
RVL-001 – Drug Profile
SHP-101 – Drug Profile
Small Molecule to Agonize mGlu7 for Rett Syndrome – Drug Profile
Small Molecules for Rett Syndrome – Drug Profile
Small Molecules to Agonize Sigma-1 Receptor for Metabolic and Central Nervous System Disorders – Drug Profile
Small Molecules to Inhibit HDAC6 for Rett Syndrome – Drug Profile
Small Molecules to Inhibit PTP1B for Rett Syndrome – Drug Profile
STRX-230 – Drug Profile
tianeptine – Drug Profile
triheptanoin – Drug Profile
trofinetide – Drug Profile
TSHA-102 – Drug Profile
TTI-0102 – Drug Profile
VYNT-0126 – Drug Profile
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 Us
Disclaimer
List of Tables
Table 1: Number of Products under Development for Rett Syndrome, 2022
Table 2: Number of Products under Development by Companies, 2022
Table 3: Number of Products under Development by Companies, 2022 (Contd..1)
Table 4: Number of Products under Development by Universities/Institutes, 2022
Table 5: Products under Development by Companies, 2022
Table 6: Products under Development by Companies, 2022 (Contd..1)
Table 7: Products under Development by Companies, 2022 (Contd..2)
Table 8: Products under Development by Universities/Institutes, 2022
Table 9: Number of Products by Stage and Target, 2022
Table 10: Number of Products by Stage and Mechanism of Action, 2022
Table 11: Number of Products by Stage and Route of Administration, 2022
Table 12: Number of Products by Stage and Molecule Type, 2022
Table 13: Rett Syndrome – Pipeline by 1st Bio Therapeutics Inc, 2022
Table 14: Rett Syndrome – Pipeline by Acadia Pharmaceuticals Inc, 2022
Table 15: Rett Syndrome – Pipeline by Adge Pharmaceuticals Inc, 2022
Table 16: Rett Syndrome – Pipeline by Alcyone Therapeutics Inc, 2022
Table 17: Rett Syndrome – Pipeline by Amicus Therapeutics Inc, 2022
Table 18: Rett Syndrome – Pipeline by AMO Pharma Ltd, 2022
Table 19: Rett Syndrome – Pipeline by Ananda Scientific Inc, 2022
Table 20: Rett Syndrome – Pipeline by Anavex Life Sciences Corp, 2022
Table 21: Rett Syndrome – Pipeline by ArmaGen Inc, 2022
Table 22: Rett Syndrome – Pipeline by Biohaven Pharmaceutical Holding Company Ltd, 2022
Table 23: Rett Syndrome – Pipeline by BioXcel Corp, 2022
Table 24: Rett Syndrome – Pipeline by Connecta Therapeutics SL, 2022
Table 25: Rett Syndrome – Pipeline by CuroNZ Ltd, 2022
Table 26: Rett Syndrome – Pipeline by DepYmed Inc, 2022
Table 27: Rett Syndrome – Pipeline by Echo Pharmaceuticals BV, 2022
Table 28: Rett Syndrome – Pipeline by Epeius Pharma Co, 2022
Table 29: Rett Syndrome – Pipeline by Epigen Biosciences Inc, 2022
Table 30: Rett Syndrome – Pipeline by EuMentis Therapeutics Inc, 2022
Table 31: Rett Syndrome – Pipeline by GEXVal Inc, 2022
Table 32: Rett Syndrome – Pipeline by Herophilus, 2022
Table 33: Rett Syndrome – Pipeline by Jazz Pharmaceuticals Plc, 2022
Table 34: Rett Syndrome – Pipeline by LizarBio Therapeutics Inc, 2022
Table 35: Rett Syndrome – Pipeline by Lucy Therapeutics Inc, 2022
Table 36: Rett Syndrome – Pipeline by Neuren Pharmaceuticals Ltd, 2022
Table 37: Rett Syndrome – Pipeline by Neurogene Inc, 2022
Table 38: Rett Syndrome – Pipeline by Neurolixis Inc, 2022
Table 39: Rett Syndrome – Pipeline by Novartis Gene Therapies, 2022
Table 40: Rett Syndrome – Pipeline by OrganAI AB, 2022
Table 41: Rett Syndrome – Pipeline by PharmatrophiX Inc, 2022
Table 42: Rett Syndrome – Pipeline by Prilenia Therapeutics Development Ltd, 2022
Table 43: Rett Syndrome – Pipeline by Q-State Biosciences Inc, 2022
Table 44: Rett Syndrome – Pipeline by Sarepta Therapeutics Inc, 2022
Table 45: Rett Syndrome – Pipeline by Shape Therapeutics Inc, 2022
Table 46: Rett Syndrome – Pipeline by Starwise Therapeutics LLC, 2022
Table 47: Rett Syndrome – Pipeline by StrideBio Inc, 2022
Table 48: Rett Syndrome – Pipeline by Synaptogenix Inc, 2022
Table 49: Rett Syndrome – Pipeline by Taysha Gene Therapies Inc, 2022
Table 50: Rett Syndrome – Pipeline by Thiogenesis Therapeutics SARL, 2022
Table 51: Rett Syndrome – Pipeline by Ultragenyx Pharmaceutical Inc, 2022
Table 52: Rett Syndrome – Pipeline by Unravel Biosciences, 2022
Table 53: Rett Syndrome – Pipeline by Vaccinex Inc, 2022
Table 54: Rett Syndrome – Pipeline by Vico Therapeutics BV, 2022
Table 55: Rett Syndrome – Pipeline by Vyant Bio Inc, 2022
Table 56: Rett Syndrome – Dormant Projects, 2022
Table 57: Rett Syndrome – Dormant Projects, 2022 (Contd..1)
Table 58: Rett Syndrome – Discontinued Products, 2022
List of Figures
Figure 1: Number of Products under Development for Rett Syndrome, 2022
Figure 2: Number of Products under Development by Companies, 2022
Figure 3: Number of Products by Top 10 Targets, 2022
Figure 4: Number of Products by Stage and Top 10 Targets, 2022
Figure 5: Number of Products by Top 10 Mechanism of Actions, 2022
Figure 6: Number of Products by Stage and Top 10 Mechanism of Actions, 2022
Figure 7: Number of Products by Routes of Administration, 2022
Figure 8: Number of Products by Stage and Routes of Administration, 2022
Figure 9: Number of Products by Molecule Types, 2022
Figure 10: Number of Products by Stage and Molecule Types, 2022

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