Glucosylceramidase – Pipeline Review, H2 2019

“Global Markets Direct’s, ‘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, H2 2019’, provides in depth analysis on 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 pipeline therapeutics. The report provides comprehensive information complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of 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 and features dormant and discontinued projects. The report analyses the pipeline products from therapy areas Genetic Disorders and Central Nervous System under development targeting 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).

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct’s 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

*Updated report will be delivered in 48 hours of order confirmation.”

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

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”

Table of Contents

Table of Contents

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

Avrobio Inc

Biosidus SA

GT Gain Therapeutics SA

JCR Pharmaceuticals Co Ltd

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

Biologic to Activate Glucocerebrosidase for Genetic Disorders - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate Glucosylceramidase for Gaucher Disease Type II - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

imiglucerase biosimilar - 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

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 biosimilar - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

velaglucerase alfa - 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

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

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

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

“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

“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”

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