Table of Content


List of Tables
List of Figures
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
Adienne Pharma & Biotech
Alectos Therapeutics
Apollo Therapeutics LLC
AVROBIO Inc
Belrose Pharma Inc
Bioasis Technologies Inc
Biosidus SA
Blue Turtle Bio Technologies Inc
Erad Therapeutics Inc
Freeline Therapeutics Ltd
GT Gain Therapeutics SA
Lysosomal Therapeutics Inc
Oxyrane Belgium NV
Spedding Research Solutions SAS
Takeda Pharmaceutical Co Ltd
Trucode Gene Repair 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
ambroxol hydrochloride - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AVRRD-02 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
FLT-200 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Glucocerebrosidase for Genetic Disorders - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Glucosylceramidase for Gaucher Disease and Parkinson’s Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
imiglucerase biosimilar - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JT-408T - 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
Oligonucleotides to Activate GBA for Gaucher Disease Type I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides to Activate GBA for Parkinson’s Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Pcgin - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombint Beta Glucosylceramidase Replacement for Parkinson’s Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombint Glucosylceramidase Replacement for Type I and Type III Gaucher’s Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Activate GBA for Gaucher Disease and Parkinson’s 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
velaglucerase alfa - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
xB-3007 - 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
Nov 06, 2019: Freeline to present data on its Gaucher Disease drug candidate FLT-200 at the 61st ASH Annual Meeting
Oct 24, 2019: AVROBIO receives Orphan-Drug Desigtion from the U.S. FDA for AVR-RD-02 for the treatment of Gaucher Disease
Sep 23, 2019: Safety and Biomarker effects of novel therapeutic for Parkinson’s disease with GBA mutations
Jul 05, 2019: Freeline presents preclinical liver-directed AAV gene therapy data at the European Working Group on Gaucher Disease.
Apr 26, 2019: AVROBIO highlights the plato platform and Gaucher program at the 2019 Annual Meeting of the American Society of Gene and Cell Therapy
Feb 26, 2019: Gain Therapeutics announces award of grant support from leading Parkinson’s Research Foundations to advance its proprietary non competitive molecular chaperones for parkinson’s disease
Oct 01, 2018: AVROBIO receives no objection to clinical trial application from Health Cada for AVR-RD-02 gene therapy for gaucher disease
Jul 17, 2018: Fetal gene therapy prevents fatal neurodegenerative disease
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
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures


Number of Products under Development by Stage of Development, H2 2019
Number of Products under Development by Therapy Areas, H2 2019
Number of Products under Development by Top 10 Indications, H2 2019
Number of Products by Mechanism of Actions, H2 2019
Number of Products by Stage and Mechanism of Actions, H2 2019
Number of Products by Routes of Administration, H2 2019
Number of Products by Stage and Routes of Administration, H2 2019
Number of Products by Molecule Types, H2 2019
Number of Products by Stage and Molecule Types, H2 2019


List of Tables


Number of Products under Development by Stage of Development, H2 2019
Number of Products under Development by Therapy Areas, H2 2019
Number of Products under Development by Indication, H2 2019
Number of Products under Development by Companies, H2 2019
Products under Development by Companies, H2 2019
Products under Development by Companies, H2 2019 (Contd..1), H2 2019
Number of Products under Investigation by Universities/Institutes, H2 2019
Products under Investigation by Universities/Institutes, H2 2019
Number of Products by Stage and Mechanism of Actions, H2 2019
Number of Products by Stage and Route of Administration, H2 2019
Number of Products by Stage and Molecule Type, H2 2019
Pipeline by Adienne Pharma & Biotech, H2 2019
Pipeline by Alectos Therapeutics, H2 2019
Pipeline by Apollo Therapeutics LLC, H2 2019
Pipeline by AVROBIO Inc, H2 2019
Pipeline by Belrose Pharma Inc, H2 2019
Pipeline by Bioasis Technologies Inc, H2 2019
Pipeline by Biosidus SA, H2 2019
Pipeline by Blue Turtle Bio Technologies Inc, H2 2019
Pipeline by Erad Therapeutics Inc, H2 2019
Pipeline by Freeline Therapeutics Ltd, H2 2019
Pipeline by GT Gain Therapeutics SA, H2 2019
Pipeline by Lysosomal Therapeutics Inc, H2 2019
Pipeline by Oxyrane Belgium NV, H2 2019
Pipeline by Spedding Research Solutions SAS, H2 2019
Pipeline by Takeda Pharmaceutical Co Ltd, H2 2019
Pipeline by Trucode Gene Repair Inc, H2 2019
Dormant Products, H2 2019
Dormant Products, H2 2019 (Contd..1), H2 2019
Discontinued Products, H2 2019