Cystinosis Market: Transforming Disease Management with Delayed Release Cysteamine Formulations and Gene Therapy Trials

The global cystinosis market is expected to grow due to the development of delayed release cysteamine formulations, ongoing gene therapy trials, and advancements in renal transplantation techniques. North America dominates the market, followed by Europe and Asia-Pacific. Key players include Recordati Rare Diseases, Amgen Inc, Viatris Inc, and Leadiant Biosciences, Inc. The market is expected to see further growth due to increasing newborn screening and early diagnosis rates, regulatory incentives for orphan drugs, and the emergence of gene therapy as a potentially curative treatment.

The global cystinosis market is poised for significant growth, driven by innovative advancements in treatment options and increasing awareness of the rare disease. According to a recent report by ResearchAndMarkets.com [1], the market is expected to expand due to the development of delayed release cysteamine formulations, ongoing gene therapy trials, and improvements in renal transplantation techniques. These developments are reshaping disease management and providing more effective treatment solutions.

North America is expected to dominate the market, with advanced healthcare systems, robust rare disease registries, and the presence of key market players such as Recordati Rare Diseases, Amgen Inc, Viatris Inc, and Leadiant Biosciences, Inc. Europe follows closely, driven by strong orphan drug policies and centralized healthcare systems. Asia-Pacific shows emerging growth potential due to increasing rare disease awareness and investment in genetic diagnostics [1].

Key drivers for market growth include increasing newborn screening and early diagnosis rates, continued innovation in cysteamine formulations and delivery systems, and the emergence of gene therapy as a potentially curative treatment. Additionally, regulatory incentives and support for orphan drugs are expected to sustain market momentum [1]. However, challenges persist, such as treatment adherence, side effects of long-term cysteamine use, and limited availability of specialized care in low-income regions.

ProBio, a leading contract development and manufacturing organization (CDMO), has recently launched its GMP Adeno-Associated Virus (AAV) manufacturing services at its 128,000 sq. ft. state-of-the-art facility in Hopewell, New Jersey. This strategic expansion aims to meet growing industry demand for high-quality viral vector production and reflects ProBio's commitment to supporting the advancement of life-changing gene therapies [2]. The Hopewell facility offers end-to-end AAV manufacturing solutions, including GMP plasmid DNA production, AAV vector manufacturing, and final drug product formulation and aseptic fill/finish, all under one roof.

In conclusion, the global cystinosis market is expected to grow significantly due to advancements in treatment options and increasing awareness of the rare disease. Key players and strategic partnerships, such as those formed by ProBio, are driving innovation and improving patient outcomes. The market is poised for further growth, driven by increasing newborn screening rates, regulatory incentives, and the emergence of gene therapy as a potentially curative treatment.

References:

[1] https://www.businesswire.com/news/home/20250813782971/en/Cystinosis-Global-Market-Research-Report-2025-2035-Innovative-Delayed-Release-Cysteamine-Formulations-Transforming-Disease-Management---ResearchAndMarkets.com
[2] https://www.morningstar.com/news/pr-newswire/20250811cl48246/probio-launches-gmp-aav-manufacturing-services-at-hopewell-facility-to-advance-gene-therapy-development-and-manufacturing