DelveInsight’s, “Bardet-Biedl Syndrome – Pipeline Insights, 2021,” 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.
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.
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.
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.
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.
Setmelanotide is an investigational, melanocortin-4 receptor (MC4R) agonist in late-stage clinical development. It restores the impaired MC4R pathway function, caused by genetic variants that occur upstream of the receptor. Setmelanotide has the potential to reduce weight and hunger. The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to Setmelanotide for the treatment of Bardet-Biedl syndrome (BBS). Also, the FDA and European Commission has granted Orphan Drug Designation for Setmelanotide for the treatment of BBS.
Further product details are provided in the report……..
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:
There are approx. 1+ key companies which are developing the therapies for Bardet-Biedl Syndrome. The companies which have their Bardet-Biedl Syndrome drug candidates in the most advanced stage, i.e. phase III include, Rhythm Pharmaceuticals.
DelveInsight’s report covers around 1+ products under different phases of clinical development like
Bardet-Biedl Syndrome pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
Products have been categorized under various Molecule types such as
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
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.
Bardet-Biedl Syndrome Report Insights
Bardet-Biedl Syndrome Report Assessment
Current Treatment Scenario and Emerging Therapies:
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
List of Table
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
• Rhythm Pharmaceuticals
