Cystinosis Pipeline constitutes 5+ key companies continuously working towards developing 5+ Cystinosis treatment therapies, analyzes DelveInsight.
Cystinosis Overview:
Cystinosis is a rare genetic disorder where the amino acid cystine accumulates in various organs and tissues, including the kidneys, eyes, muscles, liver, pancreas, and brain. It is categorized into three types: nephropathic cystinosis, intermediate cystinosis, and non-nephropathic (ocular) cystinosis. The severity, symptoms, and age of onset vary widely between individuals. Nephropathic cystinosis, the most severe and common form, appears in infancy and often requires a kidney transplant. Non-nephropathic cystinosis only affects the eyes, specifically the corneas.
Cystinosis is caused by mutations in the CTNS gene and inherited in an autosomal recessive pattern, meaning both parents must carry a mutated gene for a child to develop the disorder. While the condition was once fatal at a young age, advancements in cysteamine medication (which reduces cystine levels) and kidney transplants have significantly improved life expectancy, allowing many affected individuals to live into adulthood, and even beyond 50 years. Early diagnosis and treatment can slow disease progression, which can eventually affect all body tissues. Cystinosis affects both males and females equally and is estimated to occur in 1 in 100,000 to 200,000 individuals worldwide. It is found in all ethnic groups.
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“Cystinosis Pipeline Insight 2025” report by DelveInsight provides a comprehensive analysis of the ongoing clinical development activities and growth prospects across the Cystinosis Therapeutics Market.
Key Takeaways from the Cystinosis Pipeline Report
DelveInsight’s Cystinosis pipeline report depicts a robust space with 5+ active players working to develop 5+ pipeline therapies for Cystinosis treatment.
In May 2023, AVROBIO Announced Deal to Sell Its Cystinosis Gene Therapy Program for $87.5 Million.
Key Cystinosis companies such as AVROBIO, Birch Therapeutics, Nacuity Pharmaceuticals, and others are evaluating new drugs for Cystinosis to improve the treatment landscape.
Promising Cystinosis pipeline therapies in various stages of development include AVR RD 04, NP-3, and others.
Cystinosis Pipeline Analysis
The report provides insights into:
The report provides detailed insights into the key companies that are developing therapies in the Cystinosis Market.
The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Cystinosis treatment.
It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
It navigates the emerging drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement, and financing details for future advancement of the Cystinosis market.
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Cystinosis Emerging Drugs
AVR RD 04: AVROBIO
NP-3: Nacuity Pharmaceuticals
Cystinosis Companies
There are over five major companies working on therapies for Cystinosis. Among them, AVROBIO has drug candidates for the condition in the most advanced stage, specifically Phase I/II.
DelveInsight’s report covers around 22+ 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
Cystinosis 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
Subcutaneous
Oral
Intramuscular
Cystinosis Products have been categorized under various Molecule types such as
Monoclonal antibody
Small molecule
Peptide
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Cystinosis Pipeline Therapeutic Assessment
• Cystinosis Assessment by Product Type
• Cystinosis By Stage
• Cystinosis Assessment by Route of Administration
• Cystinosis Assessment by Molecule Type
Download Cystinosis Sample report to know in detail about the Cystinosis treatment market @ Cystinosis Therapeutic Assessment
Table of Content
1. Report Introduction
2. Executive Summary
3. Cystinosis Current Treatment Patterns
4. Cystinosis – DelveInsight’s Analytical Perspective
5. Therapeutic Assessment
6. Cystinosis Late-Stage Products (Phase-III)
7. Cystinosis Mid-Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Cystinosis Discontinued Products
13. Cystinosis Product Profiles
14. Cystinosis Key Companies
15. Cystinosis Key Products
16. Dormant and Discontinued Products
17. Cystinosis Unmet Needs
18. Cystinosis Future Perspectives
19. Cystinosis Analyst Review
20. Appendix
21. Report Methodology
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