The Cystinosis drugs in development market research report provides comprehensive information on the therapeutics under development for Cystinosis, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. GlobalData’s report assesses key aspects of the companies and drugs in development for Cystinosis. Buy the report here.

Smarter leaders trust GlobalData


Data Insights Cystinosis - Drugs In Development, 2023

Buy the Report

Data Insights

The gold standard of business intelligence.

Find out more

The report also covers the descriptive pharmacological action of the therapeutics and the latest news and press releases. Additionally, the report provides an overview of the key players involved in therapeutic development for Cystinosis and features dormant and discontinued products.

GlobalData tracks eight drugs in development for Cystinosis by five companies/universities/institutes. The top development phase for Cystinosis is phase ii with three drugs in that stage. The Cystinosis pipeline has eight drugs in development by companies and 0 by universities/ institutes. Some of the companies in the Cystinosis pipeline products market are: Nacuity Pharmaceuticals, Novartis and InSilico Medicine.

The key targets in the Cystinosis pipeline products market include Cysteine, and Cystinosin (CTNS).

The key mechanisms of action in the Cystinosis pipeline product include Cystine Depletor with two drugs in Phase III. The Cystinosis pipeline products include three routes of administration with the top ROA being Ophthalmic and three key molecule types in the Cystinosis pipeline products market including Small Molecule, and Gene-Modified Cell Therapy.

Cystinosis overview

Cystinosis is a rare genetic disorder that causes the amino acid cystine to accumulate in cells. The excess cystine can form crystals that build up and cause problems in organs, commonly the kidneys and eyes. Symptoms includes poor feeding, vomiting, dehydration, kidney problems, vision problems, and light sensitivity. It can be diagnosed by blood tests, a genetic test, slit lamp examinations, urinalysis, the presence of conjunctival crystals, and funduscopic examination. The first line of treatment for cystinosis is a medication called cysteamine, a cystine-depleting agent.

For a complete picture of Cystinosis’s pipeline drug market, buy the report here.

Data Insights


The gold standard of business intelligence.

Blending expert knowledge with cutting-edge technology, GlobalData’s unrivalled proprietary data will enable you to decode what’s happening in your market. You can make better informed decisions and gain a future-proof advantage over your competitors.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s pipeline drugs offers detailed profiles of pharmaceutical drugs in all stages of pre-clinical and clinical development, from discovery through to pre-registration. Coverage is limited to novel human medicinal drugs and biosimilars seeking market approval proprietary and is one of two primary repositories of pharmaceutical drug information offered by GlobalData through its Pharmaceutical Intelligence Center.