The Gaucher Disease Type I drugs in development market research report provides comprehensive information on the therapeutics under development for Gaucher Disease Type I, 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 I. 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 I and features dormant and discontinued products.
GlobalData tracks 14 drugs in development for Gaucher Disease Type I by 14 companies/universities/institutes. The top development phase for Gaucher Disease Type I is preclinical with four drugs in that stage. The Gaucher Disease Type I pipeline has 14 drugs in development by companies and 0 by universities/ institutes. Some of the companies in the Gaucher Disease Type I pipeline products market are: Sanofi, CANbridge Life Sciences and Takeda Pharmaceutical.
The key targets in the Gaucher Disease Type I pipeline products market include 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), 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 Mast/Stem Cell Growth Factor Receptor Kit (Proto Oncogene c Kit or Tyrosine Protein Kinase Kit or v Kit Hardy Zuckerman 4 Feline Sarcoma Viral Oncogene Homolog or Piebald Trait Protein or CD117 or KIT or EC 2.7.10.1).
The key mechanisms of action in the Gaucher Disease Type I pipeline product include 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) Activator with five drugs in Phase II. The Gaucher Disease Type I pipeline products include four routes of administration with the top ROA being Intravenous and six key molecule types in the Gaucher Disease Type I pipeline products market including Gene Therapy, and Small Molecule.
Gaucher Disease Type I overview
Type 1 Gaucher disease is a genetic condition that occurs when a fatty substance called glucocerebroside is not able to break down into glucose. It affects both children and adults. It is caused when not enough glucocerebrosidase (GBA) is made. GBA is an important enzyme that breaks down a fatty chemical called glucocerebroside. Because the body cannot break down this chemical, fat-filled Gaucher cells build up in areas like the spleen, liver and bone marrow. Major signs and symptoms include enlargement of the liver and spleen (hepatosplenomegaly), a low number of red blood cells (anemia), easy bruising caused by a decrease in blood platelets (thrombocytopenia), bone abnormalities such as bone pain and fractures, and joint conditions such as arthritis
For a complete picture of Gaucher Disease Type I’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.
