Gorlin-syndrome - Pipeline Insight, 2024

This Gorlin-syndrome - Pipeline Insight, 2024,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Gorlin-syndrome 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

Gorlin-syndrome Understanding

Gorlin-syndrome: Overview

A rare, inherited disorder that affects many organs and tissues in the body. People with this disorder have a very high risk of developing basal cell skin cancer during adolescence or early adulthood. They are also at risk of developing medulloblastoma (a type of brain cancer) and other types of cancer. Gorlin syndrome may also cause benign (not cancer) tumors in the jaw, heart, or ovaries. Other signs and symptoms include a large head and unusual facial features; small pits in the skin on the hands and feet; abnormalities of the spine, ribs, or skull; eye problems; and developmental problems. Gorlin syndrome is caused by a mutation (change) in the PTCH1 gene. Also called basal cell nevus syndrome, BCNS, NBCCS, and nevoid basal cell carcinoma syndrome.

Gorlin-syndrome - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Gorlin-syndrome pipeline landscape is provided which includes the disease overview and Gorlin-syndrome treatment guidelines. The assessment part of the report embraces, in depth Gorlin-syndrome 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, Gorlin-syndrome 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 R&D Gorlin-syndrome. The therapies under development are focused on novel approaches to treat/improve Gorlin-syndrome.

Gorlin-syndrome Emerging Drugs

PTX-022: Palvella Therapeutics
PTX-022 is a novel formulation of rapamycin which leverages Palvella’s QTORIN technology. QTORIN is a proprietary and patent-pending technology that employs a specific composition of excipients that enable distribution of rapamycin into the basal keratinocytes which harbor the mutant keratin genes that are the primary defect in pachyonychia congenita. In addition to PC, QTORIN and its related technologies are being investigated in other serious, rare genodermatoses, including Gorlin Syndrome.

Gorlin-syndrome: Therapeutic Assessment

This segment of the report provides insights about the Gorlin-syndrome drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Gorlin-syndrome

There are approx. 5+ key companies which are developing the therapies Gorlin-syndrome. The companies which have their Gorlin-syndrome drug candidates in the most advanced stage, i.e Phase II include Palvella Therapeutics

Phases

This report covers around 5+ 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

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

• Oral
• Intravenous
• Subcutaneous

Molecule Type

Products have been categorized under various Molecule types such as

• Small molecule
• Cell Therapy
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type

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

Gorlin-syndrome: Pipeline Development Activities

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

Gorlin-syndrome Report Insights

• Gorlin-syndrome Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Palvella Therapeutics, Inc
• PellePharm
• Ascend Biopharmaceuticals

Key Products

• PTX-022
• Patidegib
• ASN-002

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