DelveInsight’s ‘Cystinosis Pipeline Insight 2023‘ report provides comprehensive global coverage of available, marketed, and pipeline Cystinosis therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the Cystinosis pipeline domain.
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, Nacuity Pharmaceuticals, and others.
- Promising Cystinosis pipeline therapies in various stages of development include AVR RD 04, NP-3,and others.
Cystinosis is characterized by the 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.
Discover more about the emerging Cystinosis drugs @ Cystinosis Treatment Drugs
Cystinosis Key Companies
- Nacuity Pharmaceuticals
Cystinosis Pipeline Therapies
- AVR RD 04
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, 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 @ Cystinosis Clinical Trials
Table of Contents
2. Executive Summary
4. Pipeline Therapeutics
5. Late-Stage Products (Phase III)
6. Mid-Stage Products (Phase II)
7. Early Stage Products (Phase I/II)
8. Preclinical Stage Products
9. Discovery Stage Products
10. Therapeutic Assessment
11. Inactive Products
12. Collaborations Assessment- Licensing / Partnering / Funding
13. Unmet Needs
14. Market Drivers and Barriers
16. About DelveInsight
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