Fragile X Syndrome – Pipeline Insight, 2020
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DelveInsight’s, “Fragile X Syndrome – Pipeline Insight, 2020,” report provides comprehensive insights about 35+ companies and 35+ pipeline drugs in Fragile X Syndrome pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
Fragile X Syndrome: Overview
Fragile X syndrome (FXS) is a genetic disorder. FXS is caused by changes in a gene that scientists called the fragile X mental retardation 1 (FMR1) gene when it was first discovered. The FMR1 gene usually makes a protein called fragile X mental retardation protein (FMRP). FMRP is needed for normal brain development. People who have FXS do not make this protein. People who have other fragile X-associated disorders have changes in their FMR1 gene but usually make some of the protein.
FXS affects both males and females. However, females often have milder symptoms than males. The exact number of people who have FXS is unknown, but a review of research studies estimated that about 1 in 7,000 males about 1 in 11,000 females have been diagnosed with FXS.
Symptoms
Signs that a child might have FXS include:
FXS can be diagnosed by testing a person’s DNA from a blood test. A doctor or genetic counselor can order the test. Testing also can be done to find changes in the FMR1 gene that can lead to fragile X-associated disorders.
A diagnosis of FXS can be helpful to the family because it can provide a reason for a child’s intellectual disabilities and behavior problems. This allows the family and other caregivers to learn more about the disorder and manage care so that the child can reach his or her full potential. However, the results of DNA tests can affect other family members and raise many issues.
Treatment
There is no cure for FXS. However, treatment services can help people learn important skills. Services can include therapy to learn to talk, walk, and interact with others. In addition, medicine can be used to help control some issues, such as behavior problems.
Fragile X Syndrome Emerging Drugs Chapters
This segment of the Fragile X Syndrome report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Fragile X Syndrome Emerging Drugs
Further product details are provided in the report
Fragile X Syndrome: Therapeutic Assessment
This segment of the report provides insights about the different Fragile X Syndrome drugs segregated based on following parameters that define the scope of the report, such as:
Fragile X Syndrome: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Fragile X Syndrome therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Fragile X Syndrome drugs.
Report Highlights
Current Treatment Scenario and Emerging Therapies:
DelveInsight’s, “Fragile X Syndrome – Pipeline Insight, 2020,” report provides comprehensive insights about 35+ companies and 35+ pipeline drugs in Fragile X Syndrome pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
- Global coverage
Fragile X Syndrome: Overview
Fragile X syndrome (FXS) is a genetic disorder. FXS is caused by changes in a gene that scientists called the fragile X mental retardation 1 (FMR1) gene when it was first discovered. The FMR1 gene usually makes a protein called fragile X mental retardation protein (FMRP). FMRP is needed for normal brain development. People who have FXS do not make this protein. People who have other fragile X-associated disorders have changes in their FMR1 gene but usually make some of the protein.
FXS affects both males and females. However, females often have milder symptoms than males. The exact number of people who have FXS is unknown, but a review of research studies estimated that about 1 in 7,000 males about 1 in 11,000 females have been diagnosed with FXS.
Symptoms
Signs that a child might have FXS include:
- Developmental delays (not sitting, walking, or talking at the same time as other children the same age);
- Learning disabilities (trouble learning new skills); and
- Social and behavior problems (such as not making eye contact, anxiety, trouble paying attention, hand flapping, acting and speaking without thinking, and being very active).
FXS can be diagnosed by testing a person’s DNA from a blood test. A doctor or genetic counselor can order the test. Testing also can be done to find changes in the FMR1 gene that can lead to fragile X-associated disorders.
A diagnosis of FXS can be helpful to the family because it can provide a reason for a child’s intellectual disabilities and behavior problems. This allows the family and other caregivers to learn more about the disorder and manage care so that the child can reach his or her full potential. However, the results of DNA tests can affect other family members and raise many issues.
Treatment
There is no cure for FXS. However, treatment services can help people learn important skills. Services can include therapy to learn to talk, walk, and interact with others. In addition, medicine can be used to help control some issues, such as behavior problems.
Fragile X Syndrome Emerging Drugs Chapters
This segment of the Fragile X Syndrome report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Fragile X Syndrome Emerging Drugs
- Zygel: Zynerba Pharmaceuticals
- BPN14770: Tetra Therapeutics
- AUT00206: Autifony Therapeutics
- Pridopidine: Prilenia Therapeutics
Further product details are provided in the report
Fragile X Syndrome: Therapeutic Assessment
This segment of the report provides insights about the different Fragile X Syndrome drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Fragile X Syndrome
- Phases
- Late-stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage products (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
- Infusion
- Intradermal
- Intramuscular
- Intranasal
- Intravenous
- Oral
- Parenteral
- Subcutaneous
- Topical.
- Molecule Type
- Gene therapies
- Small molecule
- Vaccines
- Polymers
- Peptides
- Monoclonal antibodies
- Product Type
Fragile X Syndrome: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Fragile X Syndrome therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Fragile X Syndrome drugs.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Fragile X Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Fragile X Syndrome.
- Zynerba Pharmaceuticals announced that the US Food and Drug Association has granted orphan-drug designation to ZYN002 cannabidiol (CBD) gel, for the treatment of Fragile X syndrome (FXS).
- In July 2017, Autifony Therapeutics Limited announced that the US Food and Drug Administration (FDA) has granted AUT00206 an Orphan Drug Designation for the treatment of Fragile X Syndrome.
- Tetra Discovery Partners announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for BPN14770 for the treatment of Fragile X Syndrome.
- Fragile X Syndrome Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Fragile X Syndrome drugs?
- How many Fragile X Syndrome drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Fragile X Syndrome?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Fragile X Syndrome therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Fragile X Syndrome and their status?
- What are the key designations that have been granted to the emerging drugs?
- Zynerba Pharmaceuticals
- Tetra Therapeutics
- Autifony Therapeutics
- Prilenia Therapeutics
- Alcobra Ltd.
- Seaside Therapeutics, Inc.
- Marinus Pharmaceuticals
- Novartis Pharmaceuticals
- Neuren Pharmaceuticals
- Hoffmann-La Roche
- Zygel
- BPN14770
- AUT00206
- Pridopidine
- MG01CI
- Arbaclofen
- Ganaxolone
- AFQ056
- NNZ-2566
- RO4917523
Introduction
Executive Summary
Fragile X Syndrome: Overview
Causes
Mechanism of Action
Signs and Symptoms
Diagnosis
Disease Management
Pipeline Therapeutics
Comparative Analysis
Therapeutic Assessment
Assessment by Product Type
Assessment by Stage and Product Type
Assessment by Route of Administration
Assessment by Stage and Route of Administration
Assessment by Molecule Type
Assessment by Stage and Molecule Type
Fragile X Syndrome – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
Fragile X Syndrome companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Fragile X Syndrome Collaboration Deals
Company-Company Collaborations (Licensing / Partnering) Analysis
Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III and Phase II/III)
Comparative Analysis
Zygel: Zynerba Pharmaceuticals
Product Description
Research and Development
Product Development Activities
Mid Stage Products (Phase II)
Comparative Analysis
BPN14770: Tetra Therapeutics
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report…..
Early Stage Products (Phase I)
Comparative Analysis
AUT00206: Autifony Therapeutics
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report…..
Pre-clinical and Discovery Stage Products
Comparative Analysis
Pridopidine: Prilenia Therapeutics
Product Description
Research and Development
Product Development Activities
Inactive Products
Comparative Analysis
Fragile X Syndrome Key Companies
Fragile X Syndrome Key Products
Fragile X Syndrome- Unmet Needs
Fragile X Syndrome- Market Drivers and Barriers
Fragile X Syndrome- Future Perspectives and Conclusion
Fragile X Syndrome Analyst Views
Fragile X Syndrome Key Companies
Appendix
Executive Summary
Fragile X Syndrome: Overview
Causes
Mechanism of Action
Signs and Symptoms
Diagnosis
Disease Management
Pipeline Therapeutics
Comparative Analysis
Therapeutic Assessment
Assessment by Product Type
Assessment by Stage and Product Type
Assessment by Route of Administration
Assessment by Stage and Route of Administration
Assessment by Molecule Type
Assessment by Stage and Molecule Type
Fragile X Syndrome – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
Fragile X Syndrome companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Fragile X Syndrome Collaboration Deals
Company-Company Collaborations (Licensing / Partnering) Analysis
Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III and Phase II/III)
Comparative Analysis
Zygel: Zynerba Pharmaceuticals
Product Description
Research and Development
Product Development Activities
Mid Stage Products (Phase II)
Comparative Analysis
BPN14770: Tetra Therapeutics
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report…..
Early Stage Products (Phase I)
Comparative Analysis
AUT00206: Autifony Therapeutics
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report…..
Pre-clinical and Discovery Stage Products
Comparative Analysis
Pridopidine: Prilenia Therapeutics
Product Description
Research and Development
Product Development Activities
Inactive Products
Comparative Analysis
Fragile X Syndrome Key Companies
Fragile X Syndrome Key Products
Fragile X Syndrome- Unmet Needs
Fragile X Syndrome- Market Drivers and Barriers
Fragile X Syndrome- Future Perspectives and Conclusion
Fragile X Syndrome Analyst Views
Fragile X Syndrome Key Companies
Appendix
LIST OF TABLES
Table 1 Total Products for Fragile X Syndrome
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
Table 1 Total Products for Fragile X Syndrome
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
LIST OF FIGURES
Figure 1 Total Products for Fragile X Syndrome
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
Figure 1 Total Products for Fragile X Syndrome
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products