Press release
Cystinosis Pipeline Insight Report 2023: Clinical Trials 2023 (Updates), FDA Approvals, Therapies, Treatment Market and Companies working by DelveInsight | BioCryst Pharmaceuticals, Bavarian Nordic, Flow Pharma, Public Health Vaccines and Others
Cystinosis pipeline
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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.
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Cystinosis Overview
Cystinosis is a rare, inherited metabolic disorder characterized by the accumulation of the amino acid cystine within cells due to a defective transport protein. This leads to the formation of crystals that can damage various organs, especially the kidneys and eyes. There are three main forms of cystinosis: infantile, juvenile, and adult-onset. Infantile cystinosis is the most severe and typically manifests within the first year of life, causing kidney dysfunction, growth retardation, and potentially fatal complications. Juvenile and adult-onset forms have milder symptoms that may include kidney dysfunction, muscle weakness, and vision problems. Treatment primarily involves managing symptoms and reducing cystine levels with medications like cysteamine, as well as kidney transplantation in severe cases. Ongoing research aims to develop better therapies to alleviate the impact of this debilitating genetic disorder.
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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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences.
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