Press release
Cystinosis Clinical Trials and Pipeline Assessment: EMA, PDMA, FDA Approvals, Medication, IND, NDA Approvals, Therapies, Treatment Market, Mechanism of Action, Route of Administration, and Companies by DelveInsight
Cystinosis Clinical trials
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Key Takeaways from the Cystinosis Pipeline Report
• Over 5+ Cystinosis pipeline therapies are in various stages of development, and their anticipated acceptance in the Cystinosis market would significantly increase market revenue.
• Leading Cystinosis companies developing novel drug candidates to improve the Cystinosis treatment landscape include Avrobio, Inc., Gilead, Mylan N.V., Novartis AG, Orizon Pharma Plc, Nacuity Pharmaceuticals, and others.
• Promising Cystinosis pipeline therapies in various stages of development include AVR RD 04, NP-3, and others.
• AVROBIO has reported positive interim findings from a Phase I/II clinical trial of investigational gene therapy, AVR-RD-04, for cystinosis.
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Cystinosis Overview
Cystinosis is a rare, inherited metabolic disorder caused by mutations in the CTNS gene, leading to defective lysosomal cystine transport. This results in the accumulation of cystine within cells, causing progressive damage to multiple organs, particularly the kidneys, eyes, muscles, and endocrine system. Cystinosis is classified into three forms: nephropathic (infantile) cystinosis, the most severe and common type; intermediate cystinosis, which presents later in childhood; and non-nephropathic (ocular) cystinosis, which primarily affects the eyes.
The most severe form, nephropathic cystinosis, typically manifests in infancy with symptoms such as excessive thirst, frequent urination, failure to thrive, and rickets due to renal Fanconi syndrome. Without treatment, it progresses to kidney failure by late childhood or adolescence. Other complications include photophobia due to cystine crystal deposits in the cornea, hypothyroidism, muscle wasting, and neurological impairments.
Cystinosis Diagnosis is confirmed through cystine level measurement in white blood cells, genetic testing, and slit-lamp examination for corneal crystals. Treatment primarily involves cysteamine therapy, which helps reduce cystine accumulation and slows disease progression. Lifelong management, including kidney transplantation for end-stage renal disease and supportive therapies, is essential to improving patient outcomes and quality of life.
Cystinosis Pipeline Analysis: Drug Profile
AVR RD 04: AVROBIO
AVROBIO's investigational gene therapy for cystinosis is being studied in a Phase 1/2 investigational trial sponsored by the University of California, San Diego. The first patient was dosed in October 2019. The single-arm trial is expected to enroll four adults and a potential follow-on cohort of two adults or adolescents at least 14 years of age who are currently being treated with cysteamine, the standard of care for cystinosis.
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Cystinosis Pipeline Therapeutics Assessment
• By development stage
• By product type
• By route of administration
• By molecule type
• By MOA type
Scope of the Cystinosis Pipeline Report
• Coverage: Global
• Key Cystinosis Companies: Avrobio, Inc., Gilead, Mylan N.V., Novartis AG, Orizon Pharma Plc, Nacuity Pharmaceuticals, and others
• Key Cystinosis Pipeline Therapies: AVR RD 04, NP-3, and others
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Table of Contents
1. Introduction
2. Executive Summary
3. Cystinosis Overview
4. Cystinosis Pipeline Therapeutics
5. Late-Stage Products (Phase III)
6. Mid-Stage Products (Phase II)
7. Early Stage Products (Phase I/II)
8. Cystinosis Preclinical Stage Products
9. Discovery Stage Products
10. Cystinosis Therapeutic Assessment
11. Inactive Cystinosis Products
12. Collaborations Assessment- Licensing / Partnering / Funding
13. Cystinosis Unmet Needs
14. Cystinosis Market Drivers and Barriers
15. Appendix
16. About DelveInsight
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Contact Us:
Ankit Nigam
Manager Marketing
info@delveinsight.com
+14699457679
https://www.delveinsight.com/consulting/primary-research-services
Case study: https://www.delveinsight.com/case-study/cutaneous-squamous-cell-carcinoma-epidemiology-assessment
About DelveInsight
DelveInsight is a leading Life Science market research and business consulting company recognized for its off-the-shelf syndicated market research reports and customized solutions to firms in the healthcare sector.
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Introduction
Cystinosis is a rare, inherited lysosomal storage disorder caused by the accumulation of cystine within cells, leading to progressive damage to multiple organs, including the kidneys, eyes, muscles, pancreas, and thyroid. If left untreated, it results in renal Fanconi syndrome, growth retardation, photophobia, and eventually kidney failure. The disease is typically diagnosed in infancy or early childhood, and without intervention, life expectancy is severely reduced.
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