Bardet-Biedl Syndrome – Pipeline Insight, 2020
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DelveInsight’s, “Bardet-Biedl Syndrome – Pipeline Insight, 2020,” report provides comprehensive insights about 1+ companies and 1+ pipeline drugs in Bardet-Biedl 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
Bardet-Biedl Syndrome: Overview
Bardet–Biedl syndrome (BBS) is a rare autosomal recessive ciliopathy. The disease mapped to at least twenty different genes (BBS1-BBS20), follow oligogenic inheritance pattern. BBS proteins localizes to the centerosome and regulates the biogenesis and functions of the cilia. In BBS, the functioning of various systemic organs (with ciliated cells) gets deranged and results in systemic manifestations. The BBS phenotype evolves slowly throughout the first decade of life. As a result, most patients are diagnosed in late childhood or early adulthood.
Symptoms
The symptoms of Bardet-Biedl Syndrome include:
A diagnosis of Bardet-Biedl Syndrome is based upon characteristic findings and clinical findings. Genetic testing may help in confirming the diagnosis for some patients. Multigene panels offer the most effective approach in achieving molecular confirmation of BBS. Unless the diagnosis is suspected based on antenatal imaging revealing polydactyly and structural renal abnormalities, BBS is usually not diagnosed before the patient starts to develop the visual problems characteristic of rod-cone dystrophy.
Treatment
The treatment for BBS patients is focused on the specific signs and symptoms of each individual. Some of the physical abnormalities associated with BBS can be corrected with surgery. Management of obesity may include education, diet, and exercise. The eye problems can be managed with early evaluation by a specialist to provide vision aids and mobility training. Intellectual disability includes early intervention, special education along with speech therapy.
Bardet-Biedl Syndrome Emerging Drugs Chapters
This segment of the Bardet-Biedl 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.
Bardet-Biedl Syndrome Emerging Drugs
Further product details are provided in the report
Bardet-Biedl Syndrome: Therapeutic Assessment
This segment of the report provides insights about the different Bardet-Biedl Syndrome drugs segregated based on following parameters that define the scope of the report, such as:
Bardet-Biedl Syndrome: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Bardet-Biedl 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 Bardet-Biedl Syndrome drugs.
Report Highlights
Current Treatment Scenario and Emerging Therapies:
DelveInsight’s, “Bardet-Biedl Syndrome – Pipeline Insight, 2020,” report provides comprehensive insights about 1+ companies and 1+ pipeline drugs in Bardet-Biedl 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
Bardet-Biedl Syndrome: Overview
Bardet–Biedl syndrome (BBS) is a rare autosomal recessive ciliopathy. The disease mapped to at least twenty different genes (BBS1-BBS20), follow oligogenic inheritance pattern. BBS proteins localizes to the centerosome and regulates the biogenesis and functions of the cilia. In BBS, the functioning of various systemic organs (with ciliated cells) gets deranged and results in systemic manifestations. The BBS phenotype evolves slowly throughout the first decade of life. As a result, most patients are diagnosed in late childhood or early adulthood.
Symptoms
The symptoms of Bardet-Biedl Syndrome include:
- Truncal obesity
- Intellectual impairment
- Renal anomalies
- Polydactyly
- Retinal degeneration
- Hypogenitalism
A diagnosis of Bardet-Biedl Syndrome is based upon characteristic findings and clinical findings. Genetic testing may help in confirming the diagnosis for some patients. Multigene panels offer the most effective approach in achieving molecular confirmation of BBS. Unless the diagnosis is suspected based on antenatal imaging revealing polydactyly and structural renal abnormalities, BBS is usually not diagnosed before the patient starts to develop the visual problems characteristic of rod-cone dystrophy.
Treatment
The treatment for BBS patients is focused on the specific signs and symptoms of each individual. Some of the physical abnormalities associated with BBS can be corrected with surgery. Management of obesity may include education, diet, and exercise. The eye problems can be managed with early evaluation by a specialist to provide vision aids and mobility training. Intellectual disability includes early intervention, special education along with speech therapy.
Bardet-Biedl Syndrome Emerging Drugs Chapters
This segment of the Bardet-Biedl 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.
Bardet-Biedl Syndrome Emerging Drugs
- Setmelanotide: Rhythm Pharmaceuticals
Further product details are provided in the report
Bardet-Biedl Syndrome: Therapeutic Assessment
This segment of the report provides insights about the different Bardet-Biedl Syndrome drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Bardet-Biedl Syndrome
- Phases
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
- Oral
- Parenteral
- intravitreal
- Subretinal
- Topical.
- Molecule Type
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
Bardet-Biedl Syndrome: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Bardet-Biedl 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 Bardet-Biedl Syndrome drugs.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Bardet-Biedl Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Bardet-Biedl Syndrome.
- Bardet-Biedl 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 Bardet-Biedl Syndrome drugs?
- How many Bardet-Biedl Syndrome drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Bardet-Biedl Syndrome?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Bardet-Biedl 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 Bardet-Biedl Syndrome and their status?
- What are the key designations that have been granted to the emerging drugs?
- Rhythm Pharmaceuticals
- Setmelanotide
Introduction
Executive Summary
Bardet-Biedl 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
Bardet-Biedl Syndrome – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
Bardet-Biedl Syndrome companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Bardet-Biedl Syndrome Collaboration Deals
Company-Company Collaborations (Licensing / Partnering) Analysis
Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
Comparative Analysis
Setmelanotide: Rhythm Pharmaceuticals
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
Inactive Products
Comparative Analysis
Bardet-Biedl Syndrome Key Companies
Bardet-Biedl Syndrome Key Products
Bardet-Biedl Syndrome- Unmet Needs
Bardet-Biedl Syndrome- Market Drivers and Barriers
Bardet-Biedl Syndrome- Future Perspectives and Conclusion
Bardet-Biedl Syndrome Analyst Views
Bardet-Biedl Syndrome Key Companies
Appendix
Executive Summary
Bardet-Biedl 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
Bardet-Biedl Syndrome – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
Bardet-Biedl Syndrome companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Bardet-Biedl Syndrome Collaboration Deals
Company-Company Collaborations (Licensing / Partnering) Analysis
Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
Comparative Analysis
Setmelanotide: Rhythm Pharmaceuticals
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
Inactive Products
Comparative Analysis
Bardet-Biedl Syndrome Key Companies
Bardet-Biedl Syndrome Key Products
Bardet-Biedl Syndrome- Unmet Needs
Bardet-Biedl Syndrome- Market Drivers and Barriers
Bardet-Biedl Syndrome- Future Perspectives and Conclusion
Bardet-Biedl Syndrome Analyst Views
Bardet-Biedl Syndrome Key Companies
Appendix
LIST OF TABLES
Table 1 Total Products for Bardet-Biedl 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 Bardet-Biedl 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 Bardet-Biedl 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 Bardet-Biedl 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
