Press release
Cystinosis Pipeline and Clinical Trials 2023: Latest FDA Approvals, Therapeutic Advancements, Treatment, and Key Players by DelveInsight Featuring Avrobio, Inc., Gilead, Mylan N.V., Novartis AG, Orizon Pharma Plc, Nacuity Pharmaceuticals
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.
• AVROBIO Announces Positive Data from Phase 1/2 Clinical Trial of Investigational Gene Therapy for Cystinosis at the ASGCT 26th Annual Meeting.
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Cystinosis Overview
Cystinosis is a condition characterized by accumulation of the amino acid cystine (a building block of proteins) within cells. Excess cystine damages cells and often forms crystals that can build up and cause problems in many organs and tissues.
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
info@delveinsight.com
+91-9650213330
https://www.delveinsight.com/consulting
About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences.
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