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

Fragile X 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 Fragile X Syndrome – Drugs In Development, 2022, provides an overview of the Fragile X Syndrome (Genetic Disorders) pipeline landscape.

Fragile X syndrome is a genetic condition that causes a range of developmental problems including learning disabilities and cognitive impairment. Fragile X syndrome is caused by a change in a gene called FMR1. Fragile X syndrome occurs both in male and female. Symptoms include delay in crawling, walking, or twisting, hyperactive or impulsive behavior, speech and language delay and intellectual disability.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Fragile X Syndrome – Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Fragile X Syndrome (Genetic Disorders), 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 Fragile X Syndrome (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Fragile X 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, IND/CTA Filed, Preclinical and Discovery stages are 3, 2, 6, 1, 27 and 7 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 3 and 1 molecules, respectively.

Fragile X Syndrome (Genetic Disorders) 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 Fragile X Syndrome (Genetic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Fragile X Syndrome (Genetic Disorders) 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 Fragile X Syndrome (Genetic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Fragile X Syndrome (Genetic Disorders) 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 Fragile X Syndrome (Genetic Disorders)
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 Fragile X Syndrome (Genetic Disorders).
  • 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 Fragile X Syndrome (Genetic Disorders) 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
Fragile X Syndrome – Overview
Fragile X Syndrome – Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Fragile X Syndrome – Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Fragile X Syndrome – Companies Involved in Therapeutics Development
Actinogen Medical Ltd
Allos Pharma Inc
Anavex Life Sciences Corp
Anima Biotech Inc
Astellas Pharma Inc
Autifony Therapeutics Ltd
Avekshan LLC
Cognosetta Inc
Confluence Pharmaceuticals LLC
Connecta Therapeutics SL
DRI Biosciences Corp
Epigen Biosciences Inc
GEXVal Inc
GSK plc
Healx Ltd
Hua Medicine Shanghai Ltd
Jazz Pharmaceuticals Plc
Lysogene SAS
Neuren Pharmaceuticals Ltd
Neurochlore
NeuroNascent Inc
Novartis AG
Paxmedica Inc
Pilz Bioscience Corp
Prilenia Therapeutics Development Ltd
Prous Institute for Biomedical Research SA
Puretech Health Plc
Quris Technologies Ltd
Recursion Pharmaceuticals Inc
RespireRx Pharmaceuticals Inc
Sentinel Oncology Ltd
Synaptogenix Inc
Takeda Pharmaceutical Co Ltd
Taysha Gene Therapies Inc
Tetra Therapeutics
Triplet Therapeutics Inc
Zynerba Pharmaceuticals Inc
Fragile X Syndrome – Drug Profiles
acamprosate calcium SR – Drug Profile
ACT-01 – Drug Profile
arbaclofen – Drug Profile
AUT-00206 – Drug Profile
balipodect – Drug Profile
blarcamesine hydrochloride – Drug Profile
brexanolone – Drug Profile
Bryostatin-1 – Drug Profile
bumetanide – Drug Profile
cannabidiol – Drug Profile
CS-0022 – Drug Profile
CTH-120 – Drug Profile
CTH-121 – Drug Profile
CTH-122 – Drug Profile
CTH-221 – Drug Profile
CX-1739 – Drug Profile
CX-717 – Drug Profile
Drug for Fragile X Syndrome – Drug Profile
EPGN-1370 – Drug Profile
EPGN-2036 – Drug Profile
flindokalner – Drug Profile
Gene Therapy to Inhibit FMR1 for Fragile X Syndrome – Drug Profile
GSK-2702926A – Drug Profile
GTFX for Fragile X Syndrome – Drug Profile
GWP-42006 – Drug Profile
GXV-001 – Drug Profile
ibudilast – Drug Profile
JRP-655 – Drug Profile
LYSFXS-01 – Drug Profile
mavoglurant – Drug Profile
mGLUR5 NAM – Drug Profile
NNI-351 – Drug Profile
NNZ-2591 – Drug Profile
PAX-101 – Drug Profile
PAX-102 – Drug Profile
PLZ-1013 – Drug Profile
pridopidine hydrochloride – Drug Profile
Recombinant Peptide to Activate FMRP for Fragile X Syndrome – Drug Profile
Small Molecules to Activate FMRP for Fragile X Syndrome – Drug Profile
Small Molecules to Activate Kv3.1 for Fragile X Syndrome – Drug Profile
Small Molecules to Activate Slack Channels for Fragile X Syndrome – Drug Profile
Small Molecules to Antagonize KOR1 for Fragile X Syndrome and Pervasive Developmental Disorder (PDD) – Drug Profile
Small Molecules to Inhibit FMR1 for Fragile X Syndrome – Drug Profile
SOL-784 – Drug Profile
sulindac – Drug Profile
trofinetide – Drug Profile
TSHA-114 – Drug Profile
TTX-3360 – Drug Profile
UE-2343 – Drug Profile
zatolmilast – Drug Profile
Fragile X Syndrome – Dormant Projects
Fragile X Syndrome – Discontinued Products
Fragile X Syndrome – Product Development Milestones
Featured News & Press Releases
Jun 03, 2022: Zynerba Pharmaceuticals announces podium and poster presentations at the American Society of Clinical Psychopharmacology Annual Meeting
Jun 01, 2022: Neuronascent receives FDA orphan drug designation for NNI-351 treatment of Fragile X syndrome
Jun 01, 2022: Zynerba Pharmaceuticals presents data on Zygel at the American Society of Clinical Psychopharmacology Annual Meeting
May 24, 2022: Neuronascent receives FDA rare pediatric drug designation for NNI-351 treatment for Fragile X syndrome
May 11, 2022: Zynerba Pharmaceuticals presents long-term safety and efficacy data of Zygel in children and adolescents with Fragile X Syndrome (FXS) at the International Society for Autism Research Annual Meeting
May 09, 2022: Zynerba Pharmaceuticals announces podium presentation at the International Society for Autism Research Annual Meeting
May 04, 2022: Actinogen prioritizes phase 2 trials in Alzheimer’s disease and depression
May 02, 2022: Zynerba Pharmaceuticals presents long-term safety and efficacy data of Zygel in children and adolescents with Fragile X Syndrome (FXS) at the 2022 Society of Biological Psychiatry Annual Meeting
Apr 21, 2022: Lysogene publishes positive preliminary preclinical results in the treatment of Fragile X Syndrome
Apr 21, 2022: Zynerba Pharmaceuticals announces presentation of poster at the 2022 Society of Biological Psychiatry Annual Meeting
Mar 23, 2022: Nova Mentis begins enrollment in North American Autism Study
Mar 22, 2022: Synaptogenix to present at Sachs 5th Annual Neuroscience Innovation Forum
Mar 01, 2022: ACW CEO presents at Sachs 15th Annual European Life Sciences Conference
Feb 28, 2022: Zynerba Pharmaceuticals announces European commission has granted Orphan Drug Designation for Zygel in Fragile X Syndrome
Feb 16, 2022: Nova Mentis study confirms oral microdose psilocybin treatment of Autism
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Table 1: Number of Products under Development for Fragile X 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 Target, 2022 (Contd..1)
Table 11: Number of Products by Stage and Mechanism of Action, 2022
Table 12: Number of Products by Stage and Mechanism of Action, 2022 (Contd..1)
Table 13: Number of Products by Stage and Route of Administration, 2022
Table 14: Number of Products by Stage and Molecule Type, 2022
Table 15: Fragile X Syndrome – Pipeline by Actinogen Medical Ltd, 2022
Table 16: Fragile X Syndrome – Pipeline by Allos Pharma Inc, 2022
Table 17: Fragile X Syndrome – Pipeline by Anavex Life Sciences Corp, 2022
Table 18: Fragile X Syndrome – Pipeline by Anima Biotech Inc, 2022
Table 19: Fragile X Syndrome – Pipeline by Astellas Pharma Inc, 2022
Table 20: Fragile X Syndrome – Pipeline by Autifony Therapeutics Ltd, 2022
Table 21: Fragile X Syndrome – Pipeline by Avekshan LLC, 2022
Table 22: Fragile X Syndrome – Pipeline by Cognosetta Inc, 2022
Table 23: Fragile X Syndrome – Pipeline by Confluence Pharmaceuticals LLC, 2022
Table 24: Fragile X Syndrome – Pipeline by Connecta Therapeutics SL, 2022
Table 25: Fragile X Syndrome – Pipeline by DRI Biosciences Corp, 2022
Table 26: Fragile X Syndrome – Pipeline by Epigen Biosciences Inc, 2022
Table 27: Fragile X Syndrome – Pipeline by GEXVal Inc, 2022
Table 28: Fragile X Syndrome – Pipeline by GSK plc, 2022
Table 29: Fragile X Syndrome – Pipeline by Healx Ltd, 2022
Table 30: Fragile X Syndrome – Pipeline by Hua Medicine Shanghai Ltd, 2022
Table 31: Fragile X Syndrome – Pipeline by Jazz Pharmaceuticals Plc, 2022
Table 32: Fragile X Syndrome – Pipeline by Lysogene SAS, 2022
Table 33: Fragile X Syndrome – Pipeline by Neuren Pharmaceuticals Ltd, 2022
Table 34: Fragile X Syndrome – Pipeline by Neurochlore, 2022
Table 35: Fragile X Syndrome – Pipeline by NeuroNascent Inc, 2022
Table 36: Fragile X Syndrome – Pipeline by Novartis AG, 2022
Table 37: Fragile X Syndrome – Pipeline by Paxmedica Inc, 2022
Table 38: Fragile X Syndrome – Pipeline by Pilz Bioscience Corp, 2022
Table 39: Fragile X Syndrome – Pipeline by Prilenia Therapeutics Development Ltd, 2022
Table 40: Fragile X Syndrome – Pipeline by Prous Institute for Biomedical Research SA, 2022
Table 41: Fragile X Syndrome – Pipeline by Puretech Health Plc, 2022
Table 42: Fragile X Syndrome – Pipeline by Quris Technologies Ltd, 2022
Table 43: Fragile X Syndrome – Pipeline by Recursion Pharmaceuticals Inc, 2022
Table 44: Fragile X Syndrome – Pipeline by RespireRx Pharmaceuticals Inc, 2022
Table 45: Fragile X Syndrome – Pipeline by Sentinel Oncology Ltd, 2022
Table 46: Fragile X Syndrome – Pipeline by Synaptogenix Inc, 2022
Table 47: Fragile X Syndrome – Pipeline by Takeda Pharmaceutical Co Ltd, 2022
Table 48: Fragile X Syndrome – Pipeline by Taysha Gene Therapies Inc, 2022
Table 49: Fragile X Syndrome – Pipeline by Tetra Therapeutics, 2022
Table 50: Fragile X Syndrome – Pipeline by Triplet Therapeutics Inc, 2022
Table 51: Fragile X Syndrome – Pipeline by Zynerba Pharmaceuticals Inc, 2022
Table 52: Fragile X Syndrome – Dormant Projects, 2022
Table 53: Fragile X Syndrome – Dormant Projects, 2022 (Contd..1)
Table 54: Fragile X Syndrome – Discontinued Products, 2022
List of Figures
Figure 1: Number of Products under Development for Fragile X Syndrome, 2022
Figure 2: Number of Products under Development by Companies, 2022
Figure 3: Number of Products under Development by Universities/Institutes, 2022
Figure 4: Number of Products by Top 10 Targets, 2022
Figure 5: Number of Products by Stage and Top 10 Targets, 2022
Figure 6: Number of Products by Top 10 Mechanism of Actions, 2022
Figure 7: Number of Products by Stage and Top 10 Mechanism of Actions, 2022
Figure 8: Number of Products Routes of Administration, 2022
Figure 9: Number of Products by Stage and Routes of Administration, 2022
Figure 10: Number of Products by Molecule Types, 2022
Figure 11: Number of Products by Stage and Molecule Types, 2022

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