Alpha Galactosidase A – Pipeline Review, H2 2019

“Global Markets Direct’s, ‘Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC 3.2.1.22)- Pipeline Review, H2 2019’, provides in depth analysis on Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC 3.2.1.22) 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 Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC 3.2.1.22) targeted therapeutics development and features dormant and discontinued projects. The report analyses the pipeline products from therapy areas Genetic Disorders under development targeting Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC 3.2.1.22).

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 Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC 3.2.1.22)

– The report reviews Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC 3.2.1.22) 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 Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC 3.2.1.22) targeted therapeutics and enlists all their major and minor projects

– The report assesses Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC 3.2.1.22) 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 Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC 3.2.1.22) 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 Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC 3.2.1.22)

- 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 Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC 3.2.1.22) 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

“4D Molecular Therapeutics Inc

Amicus Therapeutics Inc

AVROBIO Inc

Biosidus SA

Chiesi Farmaceutici SpA

Freeline Therapeutics Ltd

Greenovation Biotech GmbH

Moderna Therapeutics Inc

Pharming Group NV

Sangamo Therapeutics Inc

UniQure NV”

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC

3.2.1.22) - Overview

Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC

3.2.1.22) - 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

Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC

3.2.1.22) - Therapeutics Assessment

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC

3.2.1.22) - Companies Involved in Therapeutics Development

Amicus Therapeutics Inc

Avrobio Inc

Biosidus SA

Chiesi Farmaceutici SpA

greenovation Biotech GmbH

iBio Inc

Moderna Therapeutics Inc

Pharming Group NV

Sangamo Therapeutics Inc

Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC

3.2.1.22) - Drug Profiles

(ATB-101 + migalastat hydrochloride) - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

agalsidase alfa - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

agalsidase alfa - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

agalsidase beta biosimilar - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AVRRD-01 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate Alpha Galactosidase A for Fabry Disease - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate GLA for Fabry Disease - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

migalastat hydrochloride - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

MOSS-AGAL - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

mRNA-3630 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

pegunigalsidase alfa - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

PGN-005 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ST-920 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC

3.2.1.22) - Dormant Products

Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC

3.2.1.22) - Discontinued Products

Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC

3.2.1.22) - Product Development Milestones

Featured News & Press Releases

Oct 01, 2018: AVROBIO announces updated clinical data for AVR-RD-01 gene therapy in fabry disease

Sep 21, 2018: Protalix BioTherapeutics reports positive preliminary data from the BRIDGE Study of pegunigalsidase alfa for the treatment of Fabry Disease

Sep 17, 2018: Protalix BioTherapeutics to present preliminary data from the BRIDGE Study of pegunigalsidase alfa for the treatment of Fabry Disease at the 1st Canadian Symposium on Lysosomal Diseases 2018

Aug 10, 2018: FDA approves Galafold (migalastat) for the treatment of certain adult patients with Fabry Disease

May 30, 2018: Amicus Therapeutics Launches Galafold (Migalastat) for Fabry Disease in Japan

May 25, 2018: Protalix BioTherapeutics Announces Poster Presentation on Baseline Characteristics for Fabry Disease Patients Screened in the Phase III BALANCE Study of Pegunigalsidase Alfa at the 55th ERA-EDTA Congress

Mar 22, 2018: Amicus Therapeutics Announces Approval of Galafold (Migalastat) for Fabry Disease in Japan

Feb 12, 2018: U.S. FDA Files New Drug Application Under Priority Review for Migalastat for Treatment of Fabry Disease

Jan 31, 2018: Protalix BioTherapeutics' pegunigalsidase alfa Receives Fast Track Designation from the U.S. Food and Drug Administration

Jan 29, 2018: Canadian Drug Expert Committee Grants Positive Recommendation for Reimbursement of Galafold (migalastat) for Fabry Disease in Canada

Jan 29, 2018: Protalix BioTherapeutics to Participate in the 14th Annual WORLDSymposium 2018

Jan 17, 2018: Amicus Therapeutics Launches Galafold (Migalastat) for Treatment of Fabry Disease in Spain

Dec 27, 2017: Protalix BioTherapeutics’ PRX-102 Granted Orphan Drug Designation by the European Commission

Dec 20, 2017: Greenovation Biotech Reports Positive Clinical Data from the Phase 1 Safety Study for moss-aGal

Dec 14, 2017: Amicus Therapeutics Submits New Drug Application to U.S. FDA for Migalastat for Treatment of Fabry Disease

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

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 4D Molecular Therapeutics Inc, H2 2019

Pipeline by Amicus Therapeutics Inc, H2 2019

Pipeline by AVROBIO Inc, H2 2019

Pipeline by Biosidus SA, H2 2019

Pipeline by Chiesi Farmaceutici SpA, H2 2019

Pipeline by Freeline Therapeutics Ltd, H2 2019

Pipeline by Greenovation Biotech GmbH, H2 2019

Pipeline by Moderna Therapeutics Inc, H2 2019

Pipeline by Pharming Group NV, H2 2019

Pipeline by Sangamo Therapeutics Inc, H2 2019

Pipeline by UniQure NV, H2 2019

Dormant Projects, 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 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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