Table of Content


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
Anda Scientific Inc
Avex 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
OrgaI AB
PharmatrophiX Inc
Prilenia Therapeutics Development Ltd
Q-State Biosciences Inc
Sarepta Therapeutics Inc
Shape Therapeutics Inc
Starwise Therapeutics LLC
StrideBio Inc
Syptogenix 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
canbidiol - 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 Desigtions 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 Mosh Biomedicine Discovery Institute to study the role of a new generation of PTP1B inhibitors against a novel intracellular checkpoint in cancer
Feb 04, 2022: Avex life sciences provides supplemental information on AVEX 2-73 (blarcamesine) AVATAR Phase 3 trial for the treatment of adult patients with Rett syndrome
Feb 01, 2022: AVEX 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 Desigtion from the European Commission for TSHA-102 for the treatment of Rett Syndrome
Sep 08, 2021: Taysha Gene Therapies to host key opinion leader webir 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: Avex Life Sciences announces AVEX2-73 (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 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

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