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) - Pipeline Review, H1 2018
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) - Pipeline Review, H1 2018
SUMMARY
According to the recently published report 'Glucosylceramidase - Pipeline Review, H1 2018'; 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) pipeline Target constitutes close to 14 molecules. Out of which approximately 11 molecules are developed by companies and remaining by the universities/institutes.
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) - Glucosylceramidase belong to the family of hydrolases that catalyzes the hydrolysis of the glycolipid, glucocerebroside, to glucose and ceramide as part of the normal degradation pathway for membrane lipids.
Deficiency in Beta-glucocerebrosidase enzymatic activity, results in accumulation of lipid glucocerebroside in tissue macrophages (Gaucher cells). Enzyme replacement therapy leads to elevated serum levels of the enzyme and reduction in the accumulation of glucocerebroside.
The report 'Glucosylceramidase - Pipeline Review, H1 2018' outlays comprehensive information on the 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) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type; that are being developed by Companies/Universities.
It also reviews key players involved in 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) targeted therapeutics development with respective active and dormant or discontinued projects.
Currently, The molecules developed by companies in Phase II, Phase I, Preclinical and Discovery stages are 1, 3, 5 and 2 respectively. Similarly, the universities portfolio in Phase I and Discovery stages comprises 1 and 2 molecules, respectively. Report covers products from therapy areas Genetic Disorders and Central Nervous System which include indications Gaucher Disease, Parkinson's Disease, Gaucher Disease Type I, Gaucher Disease Type II and Gaucher Disease Type III.
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
SCOPE
SUMMARY
According to the recently published report 'Glucosylceramidase - Pipeline Review, H1 2018'; 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) pipeline Target constitutes close to 14 molecules. Out of which approximately 11 molecules are developed by companies and remaining by the universities/institutes.
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) - Glucosylceramidase belong to the family of hydrolases that catalyzes the hydrolysis of the glycolipid, glucocerebroside, to glucose and ceramide as part of the normal degradation pathway for membrane lipids.
Deficiency in Beta-glucocerebrosidase enzymatic activity, results in accumulation of lipid glucocerebroside in tissue macrophages (Gaucher cells). Enzyme replacement therapy leads to elevated serum levels of the enzyme and reduction in the accumulation of glucocerebroside.
The report 'Glucosylceramidase - Pipeline Review, H1 2018' outlays comprehensive information on the 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) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type; that are being developed by Companies/Universities.
It also reviews key players involved in 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) targeted therapeutics development with respective active and dormant or discontinued projects.
Currently, The molecules developed by companies in Phase II, Phase I, Preclinical and Discovery stages are 1, 3, 5 and 2 respectively. Similarly, the universities portfolio in Phase I and Discovery stages comprises 1 and 2 molecules, respectively. Report covers products from therapy areas Genetic Disorders and Central Nervous System which include indications Gaucher Disease, Parkinson's Disease, Gaucher Disease Type I, Gaucher Disease Type II and Gaucher Disease Type III.
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
SCOPE
- The report provides a snapshot of the global therapeutic landscape for 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 report reviews 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) targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
- The report reviews key players involved in 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) targeted therapeutics and enlists all their major and minor projects
- The report assesses 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) targeted therapeutics based on mechanism of action (MoA), route of administration (RoA) and molecule type
- The report summarizes all the dormant and discontinued pipeline projects
- The report reviews latest news and deals related to 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) targeted therapeutics
- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Identify and understand the targeted therapy areas and indications for 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)
- Identify the use of drugs for target identification and drug repurposing
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies
- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics
- Devise corrective measures for pipeline projects by understanding 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) development landscape
- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope
Introduction
Global Markets Direct Report Coverage
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) - Overview
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) - Therapeutics Development
Products under Development by Stage of Development
Products under Development by Therapy Area
Products under Development by Indication
Products under Development by Companies
Products under Development by Universities/Institutes
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) - Therapeutics Assessment
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
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) - Companies Involved in Therapeutics Development
Bioasis Technologies Inc
JCR Pharmaceuticals Co Ltd
Protalix BioTherapeutics Inc
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) - Drug Profiles
ADN-LYS - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ambroxol - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AVRRD-02 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Biologic to Activate Glucocerebrosidase for Genetic Disorders - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
imiglucerase biosimilar - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LTI-291 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NCGC-607 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Pcgin - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PRX-112 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Activate GBA for Gaucher Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Activate GCase for Parkinson's Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Inhibit Glucocerebrosidase for Gaucher Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
taliglucerase alfa - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
taliglucerase alfa - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
velaglucerase alfa - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
xB-3003 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
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) - Dormant Products
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) - Discontinued Products
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) - Product Development Milestones
Featured News & Press Releases
Mar 22, 2017: AVROBIO Expands Rare Disease Pipeline with Gene Therapy to Treat Gaucher Disease
Dec 27, 2016: Protalix BioTherapeutics Receives Confirmation of Order for over $24 Million of alfataliglicerase to treat Gaucher Patients in Brazil
Dec 14, 2016: Protalix BioTherapeutics Receives Letter Detailing Intended Purchases of Approximately $24 Million of alfataliglicerase to treat Gaucher Patients in Brazil
Nov 22, 2016: Alfataliglicerase Approved for Pediatric Indications in Brazil for the Treatment of Gaucher Disease in Children Four years and Older
Oct 25, 2016: Asthma research unexpectedly yields new treatment approach for inherited enzyme disease
Jul 12, 2016: Researchers make advance in possible treatments for Gaucher, Parkinson's diseases
Feb 12, 2015: Safety and Efficacy of ELELYSO (taliglucerase alfa) for Injection in Pediatric Patients with Type 1 Gaucher Disease in Long-term Outcome Study Presented at the WORLDSymposium 2015
Sep 02, 2014: Shire’s VPRIV400 units/vial launched in Japan for the improvement of symptoms of Gaucher disease
Aug 28, 2014: Pfizer and Protalix BioTherapeutics Announce FDA Approval of Pediatric Indication for ELELYSO (taliglucerase alfa) for Injection, for Intravenous Use for the Treatment of Type 1 Gaucher Disease
Jul 04, 2014: Shire’s VPRIV approved in Japan for the improvement of symptoms of Gaucher disease
Jul 01, 2014: Pfizer Receives Kosher Certification for Elelyso (taliglucerase alfa) for Injection, for the Treatment of Type 1 Gaucher Disease
Jun 27, 2014: Protalix Announces New Data on ELELYSO (taliglucerase alfa) Presented at the European Working Group on Gaucher Disease 2014 11th Meeting
Jun 23, 2014: Protalix BioTherapeutics Announces New Data on ELELYSO (taliglucerase alfa) and Oral GCD to be Presented at the European Working Group on Gaucher Disease 2014 11th Meeting
Jun 18, 2014: Protalix BioTherapeutics Initiates Phase II Study with PRX-112, an Orally-Administered Enzyme Replacement Therapy for the Treatment of Gaucher Disease
May 30, 2014: Protalix Announces ELELYSO (taliglucerase alfa) Approved in Canada for the Treatment of Gaucher Disease in Both Adult and Pediatric Patients
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
Global Markets Direct Report Coverage
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) - Overview
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) - Therapeutics Development
Products under Development by Stage of Development
Products under Development by Therapy Area
Products under Development by Indication
Products under Development by Companies
Products under Development by Universities/Institutes
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) - Therapeutics Assessment
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
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) - Companies Involved in Therapeutics Development
Bioasis Technologies Inc
JCR Pharmaceuticals Co Ltd
Protalix BioTherapeutics Inc
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) - Drug Profiles
ADN-LYS - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ambroxol - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AVRRD-02 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Biologic to Activate Glucocerebrosidase for Genetic Disorders - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
imiglucerase biosimilar - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LTI-291 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NCGC-607 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Pcgin - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PRX-112 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Activate GBA for Gaucher Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Activate GCase for Parkinson's Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Inhibit Glucocerebrosidase for Gaucher Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
taliglucerase alfa - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
taliglucerase alfa - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
velaglucerase alfa - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
xB-3003 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
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) - Dormant Products
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) - Discontinued Products
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) - Product Development Milestones
Featured News & Press Releases
Mar 22, 2017: AVROBIO Expands Rare Disease Pipeline with Gene Therapy to Treat Gaucher Disease
Dec 27, 2016: Protalix BioTherapeutics Receives Confirmation of Order for over $24 Million of alfataliglicerase to treat Gaucher Patients in Brazil
Dec 14, 2016: Protalix BioTherapeutics Receives Letter Detailing Intended Purchases of Approximately $24 Million of alfataliglicerase to treat Gaucher Patients in Brazil
Nov 22, 2016: Alfataliglicerase Approved for Pediatric Indications in Brazil for the Treatment of Gaucher Disease in Children Four years and Older
Oct 25, 2016: Asthma research unexpectedly yields new treatment approach for inherited enzyme disease
Jul 12, 2016: Researchers make advance in possible treatments for Gaucher, Parkinson's diseases
Feb 12, 2015: Safety and Efficacy of ELELYSO (taliglucerase alfa) for Injection in Pediatric Patients with Type 1 Gaucher Disease in Long-term Outcome Study Presented at the WORLDSymposium 2015
Sep 02, 2014: Shire’s VPRIV400 units/vial launched in Japan for the improvement of symptoms of Gaucher disease
Aug 28, 2014: Pfizer and Protalix BioTherapeutics Announce FDA Approval of Pediatric Indication for ELELYSO (taliglucerase alfa) for Injection, for Intravenous Use for the Treatment of Type 1 Gaucher Disease
Jul 04, 2014: Shire’s VPRIV approved in Japan for the improvement of symptoms of Gaucher disease
Jul 01, 2014: Pfizer Receives Kosher Certification for Elelyso (taliglucerase alfa) for Injection, for the Treatment of Type 1 Gaucher Disease
Jun 27, 2014: Protalix Announces New Data on ELELYSO (taliglucerase alfa) Presented at the European Working Group on Gaucher Disease 2014 11th Meeting
Jun 23, 2014: Protalix BioTherapeutics Announces New Data on ELELYSO (taliglucerase alfa) and Oral GCD to be Presented at the European Working Group on Gaucher Disease 2014 11th Meeting
Jun 18, 2014: Protalix BioTherapeutics Initiates Phase II Study with PRX-112, an Orally-Administered Enzyme Replacement Therapy for the Treatment of Gaucher Disease
May 30, 2014: Protalix Announces ELELYSO (taliglucerase alfa) Approved in Canada for the Treatment of Gaucher Disease in Both Adult and Pediatric Patients
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
LIST OF TABLES
Number of Products under Development by Stage of Development, H1 2018
Number of Products under Development by Therapy Areas, H1 2018
Number of Products under Development by Indication, H1 2018
Number of Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018
Number of Products under Investigation by Universities/Institutes, H1 2018
Products under Investigation by Universities/Institutes, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Pipeline by Bioasis Technologies Inc, H1 2018
Pipeline by JCR Pharmaceuticals Co Ltd, H1 2018
Pipeline by Protalix BioTherapeutics Inc, H1 2018
Dormant Products, H1 2018
Dormant Products, H1 2018 (Contd..1), H1 2018
Discontinued Products, H1 2018
Number of Products under Development by Stage of Development, H1 2018
Number of Products under Development by Therapy Areas, H1 2018
Number of Products under Development by Indication, H1 2018
Number of Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018
Number of Products under Investigation by Universities/Institutes, H1 2018
Products under Investigation by Universities/Institutes, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Pipeline by Bioasis Technologies Inc, H1 2018
Pipeline by JCR Pharmaceuticals Co Ltd, H1 2018
Pipeline by Protalix BioTherapeutics Inc, H1 2018
Dormant Products, H1 2018
Dormant Products, H1 2018 (Contd..1), H1 2018
Discontinued Products, H1 2018
LIST OF FIGURES
Number of Products under Development by Stage of Development, H1 2018
Number of Products under Development by Therapy Areas, H1 2018
Number of Products under Development by Indications, H1 2018
Number of Products by Mechanism of Actions, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Molecule Types, H1 2018
COMPANIES MENTIONED
Bioasis Technologies Inc
JCR Pharmaceuticals Co Ltd
Protalix BioTherapeutics Inc
Number of Products under Development by Stage of Development, H1 2018
Number of Products under Development by Therapy Areas, H1 2018
Number of Products under Development by Indications, H1 2018
Number of Products by Mechanism of Actions, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Molecule Types, H1 2018
COMPANIES MENTIONED
Bioasis Technologies Inc
JCR Pharmaceuticals Co Ltd
Protalix BioTherapeutics Inc
