Cystinuria - Pipeline Insight, 2024

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

Cystinuria: Understanding

Cystinuria: Overview

Cystinuria is an inherited metabolic disorder characterized by excessive amounts of undissolved cystine in the urine, as well as three chemically similar amino acids: arginine, lysine, and ornithine. Excess cystine in the urine can lead to the formation of crystals and stones (calculi) in the kidney, bladder, and/or urinary tract (ureters). Some people with Cystinuria do not form stones, while others frequently form stones. People with cystinuria excrete abnormally high levels of cystine in the urine. The level of cystine is so high that it remains undissolved in the urine. The amino acids lysine, arginine, and ornithine are also excreted in massive amounts by people with this disorder, but they dissolve more readily in the urine and are not associated with any particular symptoms. Cystinuria is caused by changes (mutations) in the SLC3A1 and SLC7A9 genes. These mutations result in the abnormal transport of cystine in the kidney and this leads to the symptoms of cystinuria. Cystinuria is inherited in an autosomal recessive pattern. Recessive genetic disorders occur when an individual inherits two copies of an altered gene for the same trait, one from each parent. If an individual inherits one normal gene and one gene for the disease, the person will be a carrier for the disease but usually will not show symptoms. People who are carriers of these genes typically have slightly elevated levels of cystine and lysine in the urine.

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

This segment of the Cystinuria 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.

Cystinuria Emerging Drugs

Bucillamine: Revive Therapeutics Bucillamine (N-(mercapto-2-methylpropionyl)-l-cysteine), which has a well-known safety profile and is prescribed in the treatment of rheumatoid arthritis in Japan and South Korea for over 30 years, is a cysteine derivative with 2 thiol groups that is 16-fold more potent than NAC as a thiol donor in vivo, giving it vastly superior function in restoring glutathione and therefore greater potential to prevent acute lung injury during influenza infection. The drug is in phase 2 of clinical trials for the treatment of cystinuria.

Cystinuria: Therapeutic Assessment

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

Major Players in Cystinuria

There are approx. 5+ key companies which are developing the therapies for Cystinuria. The companies which have their Cystinuria drug candidates in the most advanced stage, i.e. Phase II include, Revive 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

Cystinuria 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.

Cystinuria: Pipeline Development Activities

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

Cystinuria Report Insights

• Cystinuria Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Revive Therapeutics
• Otsuka Pharmaceutical
• Eloxx Pharmaceuticals
• Travere Therapeutics
• Advicenne

Key Products

• Bucillamine
• Tolvaptan

Research programme: ribosomal protein modulators- Research programme: therapeutics

• Potassium bicarbonate/potassium citrate

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