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Glucosylceramidase - Pipeline Review, H2 2019

December 2019 | 78 pages | ID: G694EA330F5EEN
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Glucosylceramidase - Pipeline Review, H2 2019

SUMMARY

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.

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 20 molecules. Out of which approximately 17 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Phase III, Phase II, Preclinical and Discovery stages are 1, 3, 11 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 Parkinson's Disease, Gaucher Disease, Gaucher Disease Type I, Gaucher Disease Type II, Gaucher Disease Type III and Amyotrophic Lateral Sclerosis.

The latest report Glucosylceramidase - Pipeline Review, H2 2019, 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. 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.

The report is built using data and information sourced from proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources.

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
REASONS TO BUY
  • 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
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
Recombinant Beta Glucosylceramidase Replacement for Parkinson’s Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant 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 Designation 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 Canada 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 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

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

COMPANIES MENTIONED

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


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