Epidermolysis Bullosa – Pipeline Review, 2019

A group of genetic conditions that cause the skin to blister easily is known as epidermolysis bullosa. Epidermolysis bullosa simplex, junctional epidermolysis bullosa, and dystrophic epidermolysis bullosa are three types of epidermolysis bullosa. The blisters appear in response to rubbing, heat, scratching, or minor injuries. The condition generally shows up in early childhood or infancy. Majority of the types of epidermolysis bullosa are inherited. The symptoms of epidermolysis bullosa differ depending on the types, and symptoms include scarring alopecia, atrophic scarring, dysphagia, blisters inside mouth and throat, fragile skin that blisters easily, itchy and painful skin, etc. 

The current treatment of epidermolysis bullosa include surgery, medications, and rehabilitation therapy. The medications are given to prevent the complications and infection. The potential treatment of epidermolysis bullosa include gene therapy, bone marrow transplantation, protein replacement therapies, and cell based therapies. 

Academic research institutes and research laboratories have focused on studying and developing new treatment options for the epidermolysis bullosa. For instance; topical Bercolagene Telserpavec (KB103), which is being studied by Krystal Biotech, Inc., is currently in phase-2 clinical trials as gene therapy to restore functional collagen VII for the treatment of dystrophic epidermolysis bullosa.

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At present more than 80% of the pipeline candidates for epidermolysis bullosa are in the phase-1 and phase-2 stage. Majority of the studies have been sponsored by pharmaceutical companies.  

Report Description

The report on ‘Epidermolysis Bullosa – Pipeline Review, 2019’ provides a comprehensive overview of the drugs that are in the R&D pipeline by indication or molecule for Epidermolysis Bullosa. The report provides a thorough analysis of the distribution of the pipeline products by clinical trial stage, indication, company, therapy area and details such as clinical trial stage, sponsor, description on every product in the pipeline. Products in the preclinical and clinical stage along with dormant & discontinued pipeline candidates are included in the report. The report also covers additional insights such as epidemiology overview and current market scenario for Epidermolysis Bullosa.

The report on ‘Epidermolysis Bullosa – Pipeline Review, 2019’, which is built by following a robust research methodology involving primary interviews and desk research, provides a complete overview of the R&D activity and pipeline products to assist companies in developing growth strategies and identifying emerging players.  

Report Scope 

• Thorough assessment of the pipeline products by areas such as development stage; route of administration; drug class; indication; sponsor; molecule type and drug target
• Comprehensive profiles of the pipeline products with details such as company overview; product description; R&D status; development activities; mechanism of action; molecule type; development stage; indications; funding and route of administration
• Overview of dormant and discontinued pipeline products
• Key insights in relation to the epidemiology of conditions being treated by the pipeline products and overview of the addressable or current market for the pipeline products
• Overview of the latest developments; news articles, press releases and relevant conferences 

Report Methodology 

• All pipeline reports are built through the analysis of data primarily collected through credible desk research sources. Secondary research is supplemented by interviews conducted with key opinion leaders.
• Desk research sources includes global and regional clinical trials databases; annual reports, websites, press releases & investor presentations of companies; white papers; news articles; reports published by industry associations; articles/reports published on databases such as NCBI, ResearchGate; internal databases 

Reasons to Buy this Report 

• Develop effective growth strategies based on comprehensive overview of the R&D activity and pipeline products for Epidermolysis Bullosa
• Identify emerging players or competition in the market based on pipeline products and develop strategies to counter the emergence of these players
• Identify the focus of leading players in relation to R&D for Epidermolysis Bullosa
• Identify potential companies from a partnership or acquisition point of view based on current synergy in R&D activities or strategies to diversify R&D focus to drive growth in business
• Analyse the reasons behind dormant and discontinued products to make changes in the R&D focus if necessary