Press release
Cystinosis Clinical Trials 2024: EMA, PDMA, FDA Approvals, Medication, Therapies, Treatment Market, Mechanism of Action, Route of Administration, and Companies by DelveInsight | BioCryst Pharmaceuticals, Bavarian Nordic, Flow Pharma, Public Health Vaccine
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 genetic metabolic disorder characterized by the abnormal accumulation of cystine, an amino acid, within cells. This buildup occurs due to mutations in the CTNS gene, which encodes the cystinosin protein responsible for transporting cystine out of lysosomes. The excessive cystine forms crystals that damage tissues and organs, leading to a range of complications.
Cystinosis is typically categorized into three forms based on the age of onset and severity: nephropathic (infantile), intermediate (juvenile), and ocular non-nephropathic. The infantile form is the most severe, often presenting within the first year of life with symptoms like poor growth, excessive thirst, frequent urination, and kidney dysfunction (Fanconi syndrome). Over time, other complications may arise, including hypothyroidism, muscle wasting, diabetes, and vision problems due to crystal deposition in the cornea.
Cystinosis Diagnosis involves clinical assessment, genetic testing, and measurement of cystine levels in white blood cells. Early treatment with cystine-depleting agents such as cysteamine is crucial to reduce cystine accumulation and slow disease progression. Supportive care, including renal management and symptom control, is essential. Advances in research and treatment options continue to improve outcomes and quality of life for individuals with cystinosis.
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
Find out more about the Cystinosis treatment options in development @ https://www.delveinsight.com/sample-request/cystinosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr
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
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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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