Tuberous Sclerosis - Pipeline Insight, 2024

This “Tuberous Sclerosis- Pipeline Insight, 2024” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Tuberous Sclerosis 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

Tuberous Sclerosis: Understanding

Tuberous Sclerosis: Overview

Tuberous sclerosis, also known as tuberous sclerosis complex, is a rare genetic condition that causes mainly non-cancerous (benign) tumors to develop in different parts of the body. The tumors most often affect the brain, skin, kidneys, heart, eyes and lungs. Tuberous sclerosis is present from birth, although it may not cause obvious problems immediately. Tuberous sclerosis is caused by changes (mutations) in either the TSC1 or TSC2 gene. These genes are involved in regulating cell growth, and the mutations lead to uncontrolled growth and multiple tumors throughout the body.

The common symptoms of the disease are caused by noncancerous growths in parts of the body, most commonly in the skin, brain, eyes, kidneys, heart and lungs. But any part of the body can be affected. Symptoms can range from mild to severe, depending on the size or location of the growths. Although symptoms are different for each person with tuberous sclerosis, they can include skin changes, seizures, problems in thinking, reasoning and learning, behavior problems, kidney problems, heart issues, lung problems eye problems and dental changes.

Depending on symptoms, the child may see several different specialists who are experts in tuberous sclerosis. These may include specialists in problems of the brain (neurologist), heart (cardiologist), eyes (ophthalmologist), skin (dermatologist) and kidneys (nephrologist). The health care provider usually does a physical exam and talks with about symptoms and family history. The provider looks for growths, also called noncancerous tumors that are commonly caused by tuberous sclerosis. The provider also will likely order several tests - including blood tests and genetic tests - to diagnose tuberous sclerosis and identify related problems. Seizure evaluation diagnostic testing will likely include an electroencephalogram sometimes called an EEG. Brain, lungs, kidneys and liver evaluation to detect growths in the body, tests may include MRI, CT scan, and Ultrasound and heart evaluation to determine whether the heart is affected, tests usually include echocardiogram and electrocardiogram. Eye exam and dental exam developmental or mental health evaluation genetic testing.

Although there is no cure for tuberous sclerosis, treatment can help manage specific symptoms. For example medicine anti-seizure medicines may be prescribed to control seizures, surgery, therapy, educational and vocational services, mental health and behavior issues. Tuberous sclerosis is a lifelong condition that requires careful ongoing monitoring and follow-up because many symptoms may take years to develop.

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

Tuberous Sclerosis Emerging Drugs Chapters

This segment of the Tuberous Sclerosis 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.

Tuberous Sclerosis Emerging Drugs

Ganaxolone: Marinus Pharmaceuticals Ganaxolone is a first-in-class small molecule that is GABAA receptor modulator that acts by regulating brain activity. Ganaxolone binds to allosteric sites of the GABAA receptor to modulate and open the chloride ion channel, resulting in a hyperpolarization of the neuron. It is developed in oral suspension, to maximize its therapeutic potential in both acute care and outpatient settings for adult and pediatric patients. Currently the drug is in Phase III stage of clinical trial for the treatment of tuberous sclerosis.

Basimglurant: Noema Pharma AGBasimglurant (NOE-101) is a highly selective, potent, small molecule and cell-penetrant negative allosteric modulator of mGlu5 receptors for the treatment of seizures associated with Tuberous Sclerosis Complex (TSC). mGluR5 blockade has been shown to be effective in controlling seizures in a transgenic animal model of TSC. Currently the drug is in Phase II stage of clinical trial for the treatment of tuberous sclerosis complex.

Tuberous Sclerosis: Therapeutic Assessment

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

Major Players in Tuberous Sclerosis

• There are approx. 8+ key companies which are developing the therapies for Tuberous Sclerosis. The companies which have their Tuberous Sclerosis drug candidates in the most advanced stage, i.e. phase III include, Marinus Pharmaceuticals.

Phases

This report covers around 8+ 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

Tuberous Sclerosis 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
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

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

Tuberous Sclerosis: Pipeline Development Activities

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

Tuberous Sclerosis Report Insights

• Tuberous Sclerosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Marinus Pharmaceuticals
• Virpax Pharmaceuticals
• Noema Pharma
• BridgeBio Pharma

Key Products

• Ganaxolone
• Cannabidiol
• Basimglurant
• BBP 821

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