Recessive Dystrophic Epidermolysis - Pipeline Insight, 2024

Recessive Dystrophic Epidermolysis - Pipeline Insight, 2024

DelveInsight’s, “Recessive Dystrophic Epidermolysis - Pipeline Insight, 2024,” report provides comprehensive insights about 7+ companies and 7+ pipeline drugs in Recessive Dystrophic Epidermolysis 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

Recessive Dystrophic Epidermolysis Understanding

Recessive Dystrophic Epidermolysis: Overview

Recessive dystrophic epidermolysis bullosa severe generalized (RDEB-sev gen) is the classic form of the condition and is the most severe. Affected infants are typically born with widespread blistering and areas of missing skin, often caused by trauma that occurs during birth. Most often, blisters are present over the whole body and affect mucous membranes such as the moist lining of the mouth and digestive tract. As the blisters heal, they result in severe scarring. Scarring in the mouth and esophagus can make it difficult to chew and swallow food, leading to chronic malnutrition and slow growth. Additional complications of ongoing scarring can include fusion of the skin between the fingers and toes, loss of fingernails and toenails, joint deformities (contractures) that restrict movement, and eye inflammation leading to vision loss. Additionally, people with RDEB-sev gen have a very high risk of developing a form of skin cancer called squamous cell carcinoma in young adulthood. In these individuals, the cancer tends to be unusually aggressive and is often life-threatening.

""Recessive Dystrophic Epidermolysis - Pipeline Insight, 2024"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Recessive Dystrophic Epidermolysis pipeline landscape is provided which includes the disease overview and Recessive Dystrophic Epidermolysis treatment guidelines. The assessment part of the report embraces, in depth Recessive Dystrophic Epidermolysis 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, Recessive Dystrophic Epidermolysis 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 Recessive Dystrophic Epidermolysis.
• In the coming years, the Recessive Dystrophic Epidermolysis 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 Recessive Dystrophic Epidermolysis 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 Recessive Dystrophic Epidermolysis treatment market. Several potential therapies for Recessive Dystrophic Epidermolysis are under investigation. With the expected launch of these emerging therapies, it is expected that there will be a significant impact on the Recessive Dystrophic Epidermolysis market size in the coming years.
• Our in-depth analysis of the pipeline assets (in early-stage, mid-stage and late stage of development for the treatment of Recessive Dystrophic Epidermolysis) 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.

Recessive Dystrophic Epidermolysis Emerging Drugs Chapters

This segment of the Recessive Dystrophic Epidermolysis 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.

Recessive Dystrophic Epidermolysis Emerging Drugs

• FCX-007: Fibrocell Science

FCX-007 is an autologous dermal fibroblast genetically modified to express functional COL7 that is missing or deficient in these patients. Transduced with a lentiviral vector containing the COL7 producing gene, known as COL7A1, FCX-007 is injected directly into the papillary dermis of blisters and wounds where the protein enables formation of anchoring fibrils to hold the layers of skin together. FCX-007 offers the potential to address the underlying cause of RDEB by providing high levels of COL7 directly to the affected areas, thereby avoiding systemic treatment. The U. S. Food and Drug Administration has granted Orphan Drug designation to FCX-007 for the treatment of Dystrophic Epidermolysis Bullosa, which includes RDEB. In addition, FCX-007 has been granted Rare Pediatric Disease designation, Fast Track designation and Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA for treatment of RDEB.

• EB-101: Abeona Therapeutics

EB-101 is an autologous, gene-corrected cell therapy for RDEB, a rare connective tissue disorder without an approved treatment in which patients suffer with severe epidermal wounds that impact the length and quality of their lives. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce Type VII collagen that helps anchor the dermal and epidermal layers of the skin. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into an RDEB patient’s own skin cells (keratinocytes) and transplanting them back to the patient to enable normal Type VII collagen expression and skin function. EB-101 has the potential to be the first approved therapy for RDEB and the only durable treatment to address large chronic wounds, which are the most painful and debilitating. In a Phase 1/2 clinical trial, EB-101 was well-tolerated and resulted in significant and durable wound healing, with up to 5 years of follow-up.

Further product details are provided in the report……..

Recessive Dystrophic Epidermolysis: Therapeutic Assessment

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

• Major Players in Recessive Dystrophic Epidermolysis

There are approx. 7+ key companies which are developing the therapies for Recessive Dystrophic Epidermolysis. The companies which have their Recessive Dystrophic Epidermolysis drug candidates in the most advanced stage, i.e. phase III include, Abeona Therapeutics.

• Phases

DelveInsight’s report covers around 7+ 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

Recessive Dystrophic Epidermolysis 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.

Recessive Dystrophic Epidermolysis: Pipeline Development Activities

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

Recessive Dystrophic Epidermolysis Report Insights

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

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

Key Players

• Fibrocell Science
• Abeona Therapeutics
• Phoenix Tissue Repair, Inc.
• RHEACELL
• Onconova Therapeutics, Inc.
• Krystal Biotech
• Amryt Pharma
• Eloxx Pharmaceuticals

Key Products

• FCX-007
• EB-101
• PTR-01
• allo-APZ2-EB
• Rigosertib Sodium
• Beremagene Geperpavec
• AP103
• ZKN-013