The Urea Cycle Disorders drugs in development market research report provides comprehensive information on the therapeutics under development for Urea Cycle Disorders, 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 Urea Cycle Disorders. Buy the report here.
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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 Urea Cycle Disorders and features dormant and discontinued products.
GlobalData tracks 37 drugs in development for Urea Cycle Disorders by 34 companies/universities/institutes. The top development phase for Urea Cycle Disorders is preclinical with 16 drugs in that stage. The Urea Cycle Disorders pipeline has 34 drugs in development by companies and three by universities/ institutes. Some of the companies in the Urea Cycle Disorders pipeline products market are: iECURE, CAMP4 Therapeutics and Carbiotix.
The key targets in the Urea Cycle Disorders pipeline products market include Ornithine Carbamoyltransferase Mitochondrial, Argininosuccinate Lyase, and Argininosuccinate Synthase.
The key mechanisms of action in the Urea Cycle Disorders pipeline product include Ornithine Carbamoyltransferase Mitochondrial Activator with ten drugs in Phase III. The Urea Cycle Disorders pipeline products include six routes of administration with the top ROA being Intravenous and eight key molecule types in the Urea Cycle Disorders pipeline products market including Gene Therapy, and Small Molecule.
Urea Cycle Disorders overview
Urea cycle disorders (UCDs) are a group of inherited metabolic diseases that affect the ability to remove excess nitrogen from the body. Nitrogen is a by-product of protein breakdown and is normally converted into urea, a harmless substance that is excreted in urine. However, in UCDs, one of the enzymes or transporters involved in the urea cycle is defective, leading to the accumulation of ammonia, a toxic substance, in the blood and brain. This can cause serious neurological problems, such as seizures, coma, and brain damage. UCDs are rare, affecting about 1 in 35,000 newborns. There are eight types of UCDs, each named after the deficient enzyme or transporter. They are N-acetylglutamate synthase (NAGS) deficiency, carbamoyl phosphate synthetase I (CPS1) deficiency, ornithine transcarbamylase (OTC) deficiency, argininosuccinate synthase 1 (ASS1) deficiency or Citrullinemia type I, citrin deficiency or citrullinemia type II, argininosuccinic lyase (ASL) deficiency, arginase (ARG) deficiency, and ornithine translocase deficiency.
For a complete picture of Urea Cycle Disorders’s pipeline drug market, buy the report here.
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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.
