The Tay-Sachs Disease drugs in development market research report provides comprehensive information on the therapeutics under development for Tay-Sachs Disease, 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 Tay-Sachs Disease. 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 Tay-Sachs Disease and features dormant and discontinued products.
GlobalData tracks 21 drugs in development for Tay-Sachs Disease by 19 companies/universities/institutes. The top development phase for Tay-Sachs Disease is preclinical with 13 drugs in that stage. The Tay-Sachs Disease pipeline has 16 drugs in development by companies and five by universities/ institutes. Some of the companies in the Tay-Sachs Disease pipeline products market are: Orphi Therapeutics, Polaryx Therapeutics and Azafaros.
The key targets in the Tay-Sachs Disease pipeline products market include Beta-Hexosaminidase Subunit Alpha (Beta-N-Acetylhexosaminidase Subunit Alpha or N-Acetyl-Beta-Glucosaminidase Subunit Alpha or HEXA or EC 3.2.1.52), 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 Protein O GlcNAcase (Beta N Acetylglucosaminidase or Beta N Acetylhexosaminidase or Beta Hexosaminidase or OGA or EC 3.2.1.169).
The key mechanisms of action in the Tay-Sachs Disease 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 Tay-Sachs Disease pipeline products include four routes of administration with the top ROA being Oral and six key molecule types in the Tay-Sachs Disease pipeline products market including Small Molecule, and Gene Therapy.
Tay-Sachs Disease overview
Tay-Sachs disease is a rare, inherited metabolic disease that mostly affects young children and involves progressive damage to and death of cells, particularly in the brain. It is caused by a mutation in the enzyme hexosaminidase A, which allows the harmful buildup of lipids (fatty materials such as oils and acids) in cells. Both parents must carry the mutated gene in order to have a child with Tay-Sachs disease. Tay-Sachs disease is part of a group of genetic disorders called the GM2 gangliosidoses
For a complete picture of Tay-Sachs Disease’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.
