Dystrophic Epidermolysis Bullosa Pipeline Insight

DelveInsight’s, “Dystrophic Epidermolysis Bullosa - Pipeline Insight, 2022,” 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

Epidermolysis bullosa (EB) is the umbrella term for a group of rare inherited skin fragility disorders caused by mutations in at least 20 different genes. It is characterized by moderate to excessive fragility of epithelial tissues with prototypic blistering or erosions following minimal trauma (mechanobullous dermatoses). There is no cure for any of the subtypes of EB resulting from different mutations, and current therapy only focuses on the management of wounds and pain. The major consequences of recurrent blistering of the skin in patients with EB are the development of erosions and ulcers with compromised wound healing, while patients with certain subtypes of EB, especially the dystrophic variants, show extensive scarring that can lead to mutilation, particularly of the hands and feet.

 

The diagnosis of DEB is established in a proband with characteristic clinical findings and the identification of biallelic pathogenic variants (RDEB). The only gene in which pathogenic variants are known to cause DEB is COL7A1. If molecular genetic testing is not diagnostic, examination of a skin biopsy with direct immunofluorescence (IF) for specific cutaneous markers and/or electron microscopy (EM) may be necessary for diagnosis.

"Dystrophic Epidermolysis Bullosa - Pipeline Insight, 2022" report by DelveInsight 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, collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

  • The companies and academics are working to assess challenges and seek opportunities that could influence in Dystrophic Epidermolysis Bullosa R&D. The therapies under development are focused on novel approaches to treat/improve in Dystrophic Epidermolysis Bullosa.
  • In March 2022, Krystal Biotech, presented results from the GEM-3 Phase 3 study of beremagene geperpavec (B-VEC), an investigational, topical gene therapy, for the treatment of dystrophic epidermolysis bullosa (DEB), at the 2022 American Academy of Dermatology Annual Meeting in Boston, Mass. B-VEC met the primary and secondary efficacy endpoints in complete wound healing relative to placebo. The proportion of primary wounds with complete wound healing was significantly greater with B-VEC than placebo at both 3- and 6-month timepoints (p <0.005). The long-term use of B-VEC is being further investigated in an ongoing open-label extension study (NCT04917874), regardless of prior enrollment in GEM-3.
  • In Octomber 2021, Krystal Biotech and GeneDx, a wholly-owned subsidiary of BioReference Laboratories, Inc., an OPKO Health company (NASDAQ:OPK), today announced a collaboration offering no-charge genetic testing for all types of Epidermolysis Bullosa (EB). The goal of the program, called Krystal Decode DEB, is to help patients with the dystrophic form of this genetic condition, also known as DEB, get a definitive diagnosis sooner, with highly accurate results obtained with a blood or cheek swab sample.
  • In May 2022, Eloxx Pharmaceuticals, announces that ZKN-013 continues to demonstrate dose dependent inducement of functional Collagen VIIA protein (truncated in patients with nonsense mutations patients), in RDEB patient cells. Eloxx continues to expect to file an IND application to start a First in Human (FIH) Phase 1 study in 2022 with ZKN-013 after recently completing 28-day non Good Laboratory Practice (GLP) animal studies.

Dystrophic Epidermolysis Bullosa Emerging Drugs Chapter

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

 

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. Currently, it is in Phase III stage of clinical trial evaluation to treat Dystrophic Epidermolysis Bullosa. EB-101 is currently being investigated in Abeona’s pivotal Phase 3 VIITAL study for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. The EB-101 VIITAL™ study is a randomized clinical trial enrolling 10 to 15 RDEB patients with approximately 35 large, chronic wound sites treated in total.

 

FCX-007: Castle Creek Biosciences

FCX-007 is being developed to address the deficiency of functional type VII collagen protein (COL7) in patients with dystrophic epidermolysis bullosa (DEB). FCX-007 is comprised of autologously-derived dermal fibroblasts genetically modified with a lentiviral vector containing the COL7A1 gene to express COL7. FCX-007 is locally administered by injection directly into the papillary dermis of persistent and non-healing recurrent wounds of DEB where the COL7 protein can support the formation of anchoring fibrils in the skin.

 

PTR-01: Phoenix Tissue Repair

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. 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.

 

Redasemtide: Shionogi

Redasemtide is a development-stage regeneration-inducing medicine that regenerates tissue damaged by injury or disease by administration of a drug without using living cells. Shionogi is advancing development of this compound in dystrophic epidermolysis bullosa, chronic liver disease, knee osteoarthritis and cardiomyopathy, in addition to acute ischemic stroke. Based on the results of this trial, Shionogi will prepare for the transition to global Phase 3 clinical trials for acute ischemic stroke.

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

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, Abeona Therapeutics.

 

Phases

DelveInsight’s 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 

  • Subcutaneous
  • Intradermal
  • Intravenous
  • Oral
  • Topical

 

Molecule Type

Products have been categorized under various Molecule types such as

  • Cell therapy
  • Gene therapies
  • Small molecules
  • Peptide
  • Protein
  • Small interfering RNA Antidepressants

 

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?

Introduction

Executive Summary

Dystrophic Epidermolysis Bullosa : 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

Dystrophic Epidermolysis Bullosa  – DelveInsight’s Analytical Perspective

Late Stage Products (Phase III)

  • Comparative Analysis

EB-101: Abeona Therapeutics

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Mid Stage Products (Phase II)

  • Comparative Analysis

PTR 01: Phoenix Tissue Repair

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I/II)

  • Comparative Analysis

QR 313: ProQR Therapeutics

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I)

  • Comparative Analysis

Drug name: Company name

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Preclinical and Discovery Stage Products

  • Comparative Analysis

TXA127: Constant Therapeutics

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Inactive Products

  • Comparative Analysis

Dystrophic Epidermolysis Bullosa  Key Companies

Dystrophic Epidermolysis Bullosa  Key Products

Dystrophic Epidermolysis Bullosa - Unmet Needs

Dystrophic Epidermolysis Bullosa - Market Drivers and Barriers

Dystrophic Epidermolysis Bullosa - Future Perspectives and Conclusion

Dystrophic Epidermolysis Bullosa  Analyst Views

Dystrophic Epidermolysis Bullosa  Key Companies

Appendix

List of Table

Table 1: Total Products for Dystrophic Epidermolysis Bullosa

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 Dystrophic Epidermolysis Bullosa

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   

• Abeona Therapeutics
• Castle Creek Biosciences
• Krystal Biotech
• Amryt Pharma
• RegeneRx
• Rheacell
• TWi Biotechnology
• Shionogi & Co.
• Quoin Pharmaceuticals
• ProQR Therapeutics
• Phoenix Tissue Repair
• Aegle Therapeutics
• Constant Therapeutics

 

 

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