Congenital Ichthyosis - Pipeline Insight, 2024

This “Congenital Ichthyosis - Pipeline Insight, 2024,” report provides comprehensive insights about companies and pipeline drugs in the Congenital Ichthyosis 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

Congenital Ichthyosis Understanding

Congenital Ichthyosis: Overview

Congenital Ichthyosis is a general term for a family of rare genetic skin diseases characterized by dry, thickened, scaling skin by birth. There may be an abnormal quality or quantity of scale produced, abnormal thickness of stratum corneum or abnormal keratinocyte kinetics, often associated with skin inflammation. Congenital Ichthyosis is caused by the genetic mutation. Most cases of ichthyosis are mild, but some are severe.

Symptoms
The symptoms are usually present at birth and cover the entire body. Infants are often born in a collodion membrane, a shiny, wax outer layer on the skinI It is characterized by generalized, abnormally red, dry, and rough skin with large coarse and fine white scales. Itchiness (pruritus) usually also develops. Skin on the palms of the hands and soles of the feet can be abnormally thick.

Diagnosis
A parchment like membrane at birth is associated with clinical forms of congenital ichthyosis, a hallmark for the diagnosis of congenital Ichthyosis.

Treatment
Congenital Ichthyosis doesn't have a known cure, so the goal of treatment is to manage the condition. The primary treatment consists of moisturizing the skin, reducing fluid loss, and preventing infections. Prescription creams and ointments containing alpha hydroxy acids, such as lactic acid and glycolic acid, help control scaling and increase skin moisture. Vitamin A-derived medications called retinoids to reduce the production of skin cells are also used.

Congenital Ichthyosis Emerging Drugs Chapters

This segment of the Congenital Ichthyosis 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.

Congenital Ichthyosis Emerging Drugs

TMB001: Timber Pharmaceuticals LLCTMB-001 (topical isotretinoin) is being developed for the treatment of moderate to severe subtypes of CI. In a Phase 2a study, treatment with TMB-001 was shown to be well tolerated with minimal evidence of systemic absorption of isotretinoin. The Company is currently enrolling for phase 2b studies of TMB001.

KB105: Krystal Biotech, Inc.

KB105, a replication-defective, non-integrating viral vector, utilizes the STAR-D (Skin TARgeted Delivery) platform to deliver functional human TGM-1 gene directly to the patients’ skin cells. The modified HSV-1 viral vector can penetrate a broad range of skin cells with its high payload capacity and low immunogenicity. B105 is designed to be an off-the-shelf treatment for TGM1-deficient ARCI that can be applied topically, directly to a patient’s skin. In June 2019, Krystal Biotech submitted an IND application with the US FDA to initiate a Phase I/II of KB105.

Congenital Ichthyosis: Therapeutic Assessment

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

Major Players in Congenital Ichthyosis

There are companies which are developing the therapies for Congenital Ichthyosis. The companies which have their Congenital Ichthyosis drug candidates in the mid to advanced stage, i.e. phase II and Phase I include, Timber Pharmaceuticals and others.

Phases

This report covers products under different phases of clinical development like

• Mid-stage products (Phase II)
• Early-stage products (Phase I/II) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

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

• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Gene Therapy
• Product Type

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

Congenital Ichthyosis: Pipeline Development Activities

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

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Congenital Ichthyosis R&D. The therapies under development are focused on novel approaches to treat/improve Congenital Ichthyosis.

Congenital Ichthyosis Report Insights

• Congenital Ichthyosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Timber Pharmaceuticals
• Krystal Biotech Inc.

Key Products

• TMB001
• KB105

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