Gorlin-syndrome - Pipeline Insight, 2022
DelveInsight’s, Gorlin-syndrome - Pipeline Insight, 2022,” 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, 2022"" report by DelveInsight 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.
Further product details are provided in the report……..
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
DelveInsight’s 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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