Dystrophic Epidermolysis Bullosa - Pipeline Insight, 2024

This “Dystrophic Epidermolysis Bullosa - Pipeline Insight, 2024,” report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in Dystrophic Epidermolysis Bullosa 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

Dystrophic Epidermolysis Bullosa Understanding

Dystrophic Epidermolysis Bullosa: Overview

Dystrophic epidermolysis bullosa is one of the major forms of a group of conditions called epidermolysis bullosa. Epidermolysis bullosa cause the skin to be very fragile and to blister easily. Blisters and skin erosions form in response to minor injury or friction, such as rubbing or scratching. The signs and symptoms of dystrophic epidermolysis bullosa vary widely among affected individuals. In mild cases, blistering may primarily affect the hands, feet, knees, and elbows. Severe cases of this condition involve widespread blistering that can lead to vision loss, scarring, and other serious medical problems. The signs and symptoms of dystrophic epidermolysis bullosa vary widely among affected individuals. In mild cases, blistering may primarily affect the hands, feet, knees, and elbows. Severe cases of this condition involve widespread blistering that can lead to vision loss, scarring, and other serious medical problems.

Dystrophic Epidermolysis Bullosa - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Dystrophic Epidermolysis Bullosa pipeline landscape is provided which includes the disease overview and Dystrophic Epidermolysis Bullosa treatment guidelines. The assessment part of the report embraces, in depth Dystrophic Epidermolysis Bullosa commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Dystrophic Epidermolysis Bullosa collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• A better understanding of disease pathogenesis contributing to the development of novel therapeutics for Dystrophic Epidermolysis Bullosa.
• In the coming years, the Dystrophic Epidermolysis Bullosa market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.

The companies and academics that are working to assess challenges and seek opportunities that could influence Dystrophic Epidermolysis Bullosa R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.

• A detailed portfolio of major pharma players who are involved in fueling the Dystrophic Epidermolysis Bullosa treatment market. Several potential therapies for Dystrophic Epidermolysis Bullosa are under investigation. With the expected launch of these emerging therapies, it is expected that there will be a significant impact on the Dystrophic Epidermolysis Bullosa market size in the coming years.
• This in-depth analysis of the pipeline assets (in early-stage, mid-stage and late stage of development for the treatment of Dystrophic Epidermolysis Bullosa) includes therapeutic assessment and comparative analysis. This will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

Dystrophic Epidermolysis Bullosa Emerging Drugs Chapters

This segment of the Dystrophic Epidermolysis Bullosa report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.

Dystrophic Epidermolysis Bullosa Emerging Drugs

PTR-01: Phoenix Tissue Repair Phoenix Tissue Repair is advancing an investigational therapy known as PTR-01, a systemic recombinant collagen type VII (rC7) for the treatment of Recessive Dystrophic Epidermolysis Bullosa. rC7 is a potentially disease-modifying drug that is delivered intravenously to patients, replacing defective collagen type VII with healthy collagen at the sites where it is needed both internally and externally. Phoenix Tissue Repair acquired worldwide rights to PTR-01 from Shire Plc in 2017 and has initiated its first clinical trial. Preclinical studies of PTR-01 have shown that it selectively anchors in the skin and other tissues affected by an absence of collagen type VII. In four animal models of the disease, intravenous injections of PTR-01 promoted healing of DEB wounds. These experiments have shown improvements in tissue structure, disease presentation and survival, indicating a restoration of natural skinarchitecture.

FCX-007: Castle Creek BiosciencesD-Fi, also known as FCX-007, (dabocemagene autoficel), is being developed as an ex vivo, autologous cell-based gene therapy to address the deficiency of functional COL7 in patients with dystrophic epidermolysis bullosa (DEB). D-Fi has been clinically studied in a Phase 1/2 clinical study (NCT02810951), which assessed 6 patients with RDEB. In this study, 80% (8/10) of treated chronic wounds demonstrated complete wound healing 12 weeks after the first injection of D-Fi, while none of the untreated wounds were healed. D-Fi was well tolerated post-administration with few reports of temporary redness or discoloration at the injection site. D-Fi is currently in Phase 3 clinical development for the localized treatment of chronic wounds in individuals with RDEB.

Dystrophic Epidermolysis Bullosa: Therapeutic Assessment

This segment of the report provides insights about the different Dystrophic Epidermolysis Bullosa drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Dystrophic Epidermolysis Bullosa

There are approx. 15+ key companies which are developing the therapies for Dystrophic Epidermolysis Bullosa. The companies which have their Dystrophic Epidermolysis Bullosa drug candidates in the most advanced stage, i.e. phase III include, Castle Creek Biosciences.

Phases

This report covers around 15+ products under different phases of clinical development like

• 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

Dystrophic Epidermolysis Bullosa pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Inhalation
• Inhalation/Intravenous/Oral
• Intranasal
• Intravenous
• Intravenous/ Subcutaneous
• NA
• Oral
• Oral/intranasal/subcutaneous
• Parenteral
• Subcutaneous

Molecule Type

Products have been categorized under various Molecule types such as

• Antibody
• Antisense oligonucleotides
• Immunotherapy
• Monoclonal antibody
• Peptides
• Protein
• Recombinant protein
• Small molecule
• Stem Cell
• Vaccine

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Dystrophic Epidermolysis Bullosa: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Dystrophic Epidermolysis Bullosa 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 Dystrophic Epidermolysis Bullosa drugs.

Dystrophic Epidermolysis Bullosa Report Insights

• Dystrophic Epidermolysis Bullosa Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Dystrophic Epidermolysis Bullosa Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Dystrophic Epidermolysis Bullosa drugs?
• How many Dystrophic Epidermolysis Bullosa drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Dystrophic Epidermolysis Bullosa?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Dystrophic Epidermolysis Bullosa 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 Dystrophic Epidermolysis Bullosa and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Phoenix Tissue Repair
• Castle Creek Biosciences
• Phoenicis Therapeutics
• Aegle Therapeutics
• RHEACELL GmbH & Co. KG
• Anterogen Co., Ltd.
• Abeona Therapeutics, Inc
• InMed Pharmaceuticals
• BridgeBio Inc.
• Eloxx Pharmaceuticals
• Relief Therapeutics
• Quoin Pharmaceuticals

Key Products

• PTR-01
• FCX-007
• PTW-002
• AGLE 102
• allo-APZ2-EB
• ALLO-ASC-SHEET
• EB-101
• INM-755
• BBP-589
• ZKN-013
• Nexodyn
• QRX004

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