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

Pages: 110 Published: July 29, 2022 Report Code: GDGMDHC22241IDB

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.

* Global Markets Direct, is a research brand owned and operated by GlobalData.

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.

Key Players

  • 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

Table of Contents

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

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 Us

Disclaimer

List of Tables

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

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

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