Alport Syndrome - Pipeline Insight, 2024

This “Alport Syndrome - Pipeline Insight, 2024,” report provides comprehensive insights about 8+ companies and 10+ pipeline drugs in Alport 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

Alport Syndrome: Understanding

Alport Syndrome: Overview

Alport syndrome, also known as hereditary nephritis is a genetic disorder arising from the mutations in the genes encoding alpha-3, alpha-4, and alpha-5 of type 4 collagen (COL4A3, COL4A4, COL4A5) or collagen 4 a345 network. The type 4 collagen alpha chains are primarily located in the kidneys, eyes, and cochlea. Alport syndrome is X-linked (XLAS) and can be transmitted in an autosomal recessive (ARAS) or autosomal dominant fashion (ADAS). In 1927, the syndrome of hereditary nephritis and deafness was described by a British physician, A. Cecil Alport. It was observed that hematuria was the most common symptom and males were affected more than females. In 1961, it was named Alport syndrome after having described in multiple family members. It is characterized by renal failure, bilateral sensorineural hearing loss, and eye abnormalities. Eventually, the patients present with proteinuria, hypertension, progressive loss of kidney function (gradual decline in GFR), and end-stage renal disease.

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

This segment of the Alport Syndrome 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.

Alport Syndrome Emerging Drugs

Bardoxolone: Reata Pharmaceuticals, Inc.Bardoxolone is an investigational, once-daily, orally administered activator of Nrf2, a transcription factor that induces molecular pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling. The FDA has granted Orphan Drug designation to bardoxolone for the treatment of Alport syndrome and autosomal dominant polycystic kidney disease (“ADPKD”).

Alport Syndrome: Therapeutic Assessment

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

Major Players in Alport Syndrome

There are approx. 8+ key companies which are developing the therapies for Alport Syndrome. The companies which have their Alport Syndrome drug candidates in the most advanced stage, i.e. Preregistration include, Reata Pharmaceuticals.

Phases

This report covers around 10+ 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

Alport 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

• Intravenous

Molecule Type

Products have been categorized under various Molecule types such as

• Peptide
• Protein
• Propylene glycols
• Cell Therapy

Product Type

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

Alport Syndrome: Pipeline Development Activities

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

Alport Syndrome Report Insights

• Alport Syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Eloxx Pharmaceuticals, Inc.
• Chinook Therapeutic
• River 3 Renal Corp.
• Travere Therapeutics, Inc.
• Reata Pharmaceuticals, Inc.

Key Products

• ELX-02
• R3R01
• Sparsentan
• Atrasentan
• R3R01
• Bardoxolone methyl

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