Cystinosis FDA Approvals, Clinical Trials and Pipeline Insights 2024 | AVROBIO, Nacuity Pharmaceuticals

DelveInsight's 'Cystinosis Pipeline Insight 2024' 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.
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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, Nacuity Pharmaceuticals, and others.
Promising Cystinosis pipeline therapies in various stages of development include AVR RD 04, NP-3,and others.
Cystinosis Overview
Cystinosis is a rare genetic disorder characterized by the accumulation of cystine, a type of amino acid, within cells throughout the body. This buildup leads to the formation of crystals that can damage various organs and tissues, particularly the kidneys and eyes. Here's a comprehensive overview of cystinosis, including its causes, signs and symptoms, diagnosis, and treatment options:

Cystinosis Causes:
Cystinosis is caused by mutations in the CTNS gene, which encodes a protein responsible for transporting cystine out of cells. When this protein is defective or absent, cystine accumulates inside cells instead of being transported out, leading to the formation of crystals.

Cystinosis Signs and Symptoms:
The symptoms of cystinosis can vary depending on the age of onset and the severity of the disease. The most common form, nephropathic cystinosis, typically manifests in infants within the first year of life and includes:

Renal Symptoms: Excessive thirst and urination (polyuria/polydipsia), growth retardation, electrolyte imbalances, and eventually, kidney failure.
Ophthalmic Symptoms: Photophobia (sensitivity to light), eye pain, and corneal crystals that can lead to vision impairment or blindness if untreated.
Systemic Symptoms: In later stages, cystine crystals can accumulate in other organs such as muscles, pancreas, thyroid, and brain, causing additional complications.
Non-nephropathic cystinosis, a milder form, typically presents later in childhood or adulthood and primarily affects the eyes.

Cystinosis Diagnosis:
Diagnosing cystinosis involves a combination of clinical evaluation, genetic testing to identify mutations in the CTNS gene, and measuring cystine levels in cells or tissues. Early diagnosis is crucial to initiate treatment promptly and prevent irreversible organ damage.

Cystinosis Treatment Options:
Currently, the mainstay of treatment for cystinosis is cysteamine therapy, which helps reduce cystine accumulation by converting cystine into a less harmful form that can be excreted through urine. Treatment aims to delay the progression of kidney disease and preserve renal function. Other supportive therapies include:

Renal Replacement Therapy: In advanced cases, dialysis or kidney transplantation may be necessary.
Ophthalmic Care: Regular eye examinations and treatment for corneal cystine crystals, such as eye drops and surgical intervention if required.
Nutritional Support: Adequate hydration, electrolyte balance, and nutritional supplementation to support growth and development.
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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 @ https://www.delveinsight.com/sample-request/cystinosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Cystinosis Key Companies
AVROBIO
Nacuity Pharmaceuticals
And others
Cystinosis Pipeline Therapies
AVR RD 04
NP-3
And others
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 @ https://www.delveinsight.com/sample-request/cystinosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Table of Contents
1. Introduction
2. Executive Summary
3. Overview
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
15. Appendix
16. About DelveInsight
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This release was published on openPR.