Cystinuria Pipeline Insight 2025: Redefining Treatment through Precision Therapies and Rare Disease Innovation | DelveInsight

DelveInsight's "Cystinuria - Pipeline Insight, 2025" provides a detailed examination of the ongoing therapeutic R&D landscape targeting cystinuria, a rare inherited disorder characterized by defective renal reabsorption of cystine and other dibasic amino acids, leading to recurrent kidney stone formation. The disease is driven by mutations in the SLC3A1 or SLC7A9 genes, and remains a chronic urological challenge due to its lifelong recurrence risk and resistance to standard stone-prevention strategies.

Current treatment involves high fluid intake, urinary alkalinization, and use of thiol-based drugs like tiopronin or D-penicillamine to increase cystine solubility. However, these approaches often fail to fully prevent stone recurrence and are associated with poor tolerability, adherence challenges, and long-term complications. This unmet need has spurred a new wave of research focused on more targeted, well-tolerated, and potentially curative interventions.

The emerging pipeline is exploring several innovative avenues:
• Novel Cystine-Lowering Agents: Small molecules with better pharmacokinetics and safety profiles are in early-stage development, aiming to enhance cystine solubility without the side effects of traditional thiol drugs.
• Gene Therapy and RNA-Based Platforms: Preclinical research is investigating gene replacement and gene-editing strategies to restore transporter function at the molecular level, offering potential one-time interventions.
• Targeted Transporter Modulators: Programs aimed at correcting or compensating for defective SLC gene function through precision mechanisms are gaining momentum.
• Supportive Therapies and Diagnostics: Noninvasive monitoring tools and companion diagnostics are under evaluation to personalize treatment and improve disease tracking.

Although still in early stages, the pipeline benefits from regulatory incentives like orphan drug designations and rare pediatric disease support, which are accelerating clinical entry and investment in this niche indication. With growing advocacy and cross-specialty collaboration, the cystinuria therapeutic landscape is entering a transformative phase that promises to shift from reactive stone management to proactive and molecularly guided disease modification.

Interested in learning more about the current treatment landscape and the key drivers shaping the cystinuria pipeline? Click here: https://www.delveinsight.com/report-store/cystinuria-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Key Takeaways from the Cystinuria Pipeline Report
• DelveInsight's cystinuria pipeline analysis depicts a strong space with 5+ active players working to develop 5+ pipeline drugs for cystinuria treatment.
• The leading cystinuria companies include Revive Therapeutics, Otsuka Pharmaceutical, Eloxx Pharmaceuticals, Travere Therapeutics, Advicenne, and others are evaluating their lead assets to improve the cystinuria treatment landscape.
• Key cystinuria pipeline therapies in various stages of development include Bucillamine, Tolvaptan, Research programme: ribosomal protein modulators, Research programme: therapeutics, Potassium bicarbonate/potassium citrate, and others.
• In June 2025, Sarepta Therapeutics (NASDAQ: SRPT) announced that the FDA granted platform technology designation to its rAAVrh74 viral vector, used in the investigational gene therapy SRP-9003 (bidridistrogene xeboparvovec) for treating limb-girdle muscular dystrophy type 2E/R4.
• In June 2025, Cure Rare Disease (CRD) announced the successful completion of a pre-IND meeting with the FDA for its investigational gene therapy targeting Limb-Girdle Muscular Dystrophy type 2I/R9 (LGMDR9).
• In November 2024, Atamyo Therapeutics announced FDA clearance of its IND application for ATA-200, a one-time gene therapy for γ-sarcoglycan-related LGMD2C/R5, allowing it to proceed to a Phase 1b/2b clinical trial.

Request a sample and discover the recent breakthroughs happening in the cystinuria pipeline landscape at https://www.delveinsight.com/report-store/cystinuria-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Cystinuria Overview
Cystinuria is a rare genetic disorder characterized by the impaired reabsorption of cystine and other amino acids in the kidneys, leading to the formation of cystine kidney stones. This condition results from mutations affecting cystine transporters, causing high cystine levels in the urine that can crystallize and form stones, often causing pain, urinary tract infections, and kidney damage.

Currently, treatment focuses on preventing stone formation through increased fluid intake, dietary modifications, and medications such as thiol-containing drugs (e.g., bucillamine) and potassium citrate to alkalinize urine. Research is ongoing into novel therapies, including ribosomal protein modulators and other targeted treatments, to improve patient outcomes.

Find out more about cystinuria medication at https://www.delveinsight.com/report-store/cystinuria-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Cystinuria Treatment Analysis: Drug Profile
Bucillamine: Revive Therapeutics
Bucillamine (N-(mercapto-2-methylpropionyl)-l-cysteine) is a cysteine derivative with two thiol groups, known for its strong safety record after over 30 years of use in treating rheumatoid arthritis in Japan and South Korea. It is 16 times more potent than NAC as a thiol donor, enhancing glutathione restoration and showing greater potential to prevent acute lung injury during influenza. Currently, Bucillamine is in Phase 2 clinical trials for treating cystinuria.

Learn more about the novel and emerging cystinuria pipeline therapies at https://www.delveinsight.com/report-store/cystinuria-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Cystinuria Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.

By Stage
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

By Route of Administration
• Intravenous

By Molecule Type
• Peptide
• Protein
• Propylene glycols
• Cell Therapy

Scope of the Cystinuria Pipeline Report
• Coverage: Global
• Key Cystinuria Companies: Revive Therapeutics, Otsuka Pharmaceutical, Eloxx Pharmaceuticals, Travere Therapeutics, Advicenne, and others.
• Key Cystinuria Pipeline Therapies: Bucillamine, Tolvaptan, Research programme: ribosomal protein modulators, Research programme: therapeutics, Potassium bicarbonate/potassium citrate, and others.

To dive deep into rich insights for drugs used for cystinuria treatment, visit: https://www.delveinsight.com/report-store/cystinuria-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Table of Contents
1. Introduction
2. Executive Summary
3. Cystinuria Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Cystinuria Pipeline Therapeutics
6. Cystinuria Pipeline: Late-Stage Products (Phase III)
7. Cystinuria Pipeline: Mid-Stage Products (Phase II)
8. Cystinuria Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix

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Jatin Vimal
jvimal@delveinsight.com
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This release was published on openPR.