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Cystinosis Pipeline Analysis

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

Download Sample Pages to Get an in-depth Assessment of the Emerging Cystinosis Therapies and Key Companies: Cystinosis Clinical Trials and advancements

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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