The Gaucher Disease Type III drugs in development market research report provides comprehensive information on the therapeutics under development for Gaucher Disease Type III, 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 Gaucher Disease Type III. 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 Gaucher Disease Type III and features dormant and discontinued products.
GlobalData tracks seven drugs in development for Gaucher Disease Type III by six companies/universities/institutes. The top development phase for Gaucher Disease Type III is phase ii with three drugs in that stage. The Gaucher Disease Type III pipeline has seven drugs in development by companies and 0 by universities/ institutes. Some of the companies in the Gaucher Disease Type III pipeline products market are: Sanofi, CANbridge Life Sciences and Blue Turtle Bio Technologies.
The key targets in the Gaucher Disease Type III pipeline products market include Ceramide Glucosyltransferase (Glucosylceramide Synthase or GLCT 1 or UDP Glucose:N Acylsphingosine D Glucosyltransferase or UDP Glucose Ceramide Glucosyltransferase or UGCG or EC 2.4.1.80), and Glucosylceramidase (Acid Beta Glucosidase or Alglucerase or D Glucosyl N Acylsphingosine Glucohydrolase or Beta Glucocerebrosidase or Imiglucerase or GBA or EC 3.2.1.45).
The key mechanisms of action in the Gaucher Disease Type III pipeline product include Ceramide Glucosyltransferase (Glucosylceramide Synthase or GLCT 1 or UDP Glucose:N Acylsphingosine D Glucosyltransferase or UDP Glucose Ceramide Glucosyltransferase or UGCG or EC 2.4.1.80) Inhibitor with three drugs in Phase III. The Gaucher Disease Type III pipeline products include two routes of administration with the top ROA being Oral and four key molecule types in the Gaucher Disease Type III pipeline products market including Small Molecule, and Recombinant Enzyme.
Gaucher Disease Type III overview
Gaucher disease type III is the subacute neurological form of Gaucher disease characterized by progressive encephalopathy and associated with the systemic manifestations (organomegaly, bone involvement, cytopenia) of Gaucher disease type 1. It is caused by a mutation in the GBA gene (1q21) that codes for the lysosomal enzyme, glucocerebrosidase. The deficiency in glucocerebrosidase leads to the accumulation of glucosylceramidase (or beta-glucocerebrosidase) deposits in the cells of the reticuloendothelial system of the liver, spleen and the bone marrow (Gaucher cells)
For a complete picture of Gaucher Disease Type III’s pipeline drug market, buy the report here.
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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.
