Alpha Galactosidase A - Pipeline Review, H2 2019
Alpha Galactosidase A - Pipeline Review, H2 2019
SUMMARY
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 Target constitutes close to 13 molecules. Out of which approximately 12 molecules are developed by companies and remaining by the universities/institutes. The latest report Alpha Galactosidase A - Pipeline Review, H2 2019, outlays comprehensive information on the 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, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and 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) - Alpha-galactosidase is a glycoside hydrolase enzyme encoded by the GLA gene. This enzyme is a homodimeric glycoprotein that hydrolyses the terminal alpha-galactosyl moieties from glycolipids and glycoproteins. It predominantly hydrolyzes ceramide trihexoside, and it can catalyze the hydrolysis of melibiose into galactose and glucose. Mutations in this gene affect the synthesis and stability of this enzyme which causes Fabry's disease. The molecules developed by companies in Pre-Registration, Phase III, Phase II and Preclinical stages are 1, 1, 4 and 6 respectively. Similarly, the universities portfolio in Unknown stages comprises 1 molecules, respectively. Report covers products from therapy areas Genetic Disorders which include indications Fabry Disease.
Furthermore, this report also 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 development with respective active and dormant or discontinued projects. Driven by 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
SUMMARY
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 Target constitutes close to 13 molecules. Out of which approximately 12 molecules are developed by companies and remaining by the universities/institutes. The latest report Alpha Galactosidase A - Pipeline Review, H2 2019, outlays comprehensive information on the 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, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and 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) - Alpha-galactosidase is a glycoside hydrolase enzyme encoded by the GLA gene. This enzyme is a homodimeric glycoprotein that hydrolyses the terminal alpha-galactosyl moieties from glycolipids and glycoproteins. It predominantly hydrolyzes ceramide trihexoside, and it can catalyze the hydrolysis of melibiose into galactose and glucose. Mutations in this gene affect the synthesis and stability of this enzyme which causes Fabry's disease. The molecules developed by companies in Pre-Registration, Phase III, Phase II and Preclinical stages are 1, 1, 4 and 6 respectively. Similarly, the universities portfolio in Unknown stages comprises 1 molecules, respectively. Report covers products from therapy areas Genetic Disorders which include indications Fabry Disease.
Furthermore, this report also 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 development with respective active and dormant or discontinued projects. Driven by 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 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
- 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
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
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
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
4D-310 - 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
AMT-190 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AVRRD-01 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
FLT-190 - 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
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
Nov 27, 2019: Protalix BioTherapeutics hosting key opinion leader meeting on PRX-102 drug candidate for the treatment of Fabry Disease
Nov 18, 2019: Protalix BioTherapeutics and Chiesi Farmaceutici announce successful pre-BLA meeting with FDA for accelerated approval of pegunigalsidase alfa for the treatment of Fabry Disease in the United States
Oct 17, 2019: Protalix BioTherapeutics announces positive 12-month interim data from the BRIDGE phase III open label switch-over study of Pegunigalsidase Alfa for the treatment of Fabry Disease
Sep 25, 2019: Protalix and Chiesi close enrolment in Phase III Fabry disease trial
Sep 12, 2019: Freeline doses patient in first Fabry Disease AAV gene therapy trial globally
Aug 02, 2019: Amicus Therapeutics receives marketing authorization for galafold (migalastat) for fabry disease in Argentina
Jul 15, 2019: AVROBIO announces 87% substrate reduction in first kidney biopsy and additional positive data from clinical trials of AVR-RD-01 investigational gene therapy in Fabry Disease
Jul 02, 2019: Handok releases Fabry disease treatment
Jun 17, 2019: Protalix Biotherapeutics completes enrollment in the phase III bright clinical trial of Pegunigalsidase Alfa (PRX 102) for the treatment of Fabry disease
Jun 06, 2019: Protalix BioTherapeutics and Chiesi Farmaceutici to apply for accelerated approval of pegunigalsidase alfa for the treatment of Fabry Disease in the United States
May 30, 2019: 4D Molecular Therapeutics announces presentation of preclinical data at the 6th International Update on Fabry Disease and provides clinical update
May 28, 2019: Freeline presents preclinical data and phase 1/2 study design of the FLT190 AAV gene therapy for Fabry Disease
May 02, 2019: uniQure announces new preclinical data on fabry disease in oral presentations at the 22nd ASGCT Annual Meeting
May 01, 2019: Avrobio to incorporate plato platform into FAB-201 Phase II trial
Apr 15, 2019: uniQure to present data on Gene Therapy for Fabry Disease at ASGCT 2019
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
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
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
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
4D-310 - 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
AMT-190 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AVRRD-01 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
FLT-190 - 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
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
Nov 27, 2019: Protalix BioTherapeutics hosting key opinion leader meeting on PRX-102 drug candidate for the treatment of Fabry Disease
Nov 18, 2019: Protalix BioTherapeutics and Chiesi Farmaceutici announce successful pre-BLA meeting with FDA for accelerated approval of pegunigalsidase alfa for the treatment of Fabry Disease in the United States
Oct 17, 2019: Protalix BioTherapeutics announces positive 12-month interim data from the BRIDGE phase III open label switch-over study of Pegunigalsidase Alfa for the treatment of Fabry Disease
Sep 25, 2019: Protalix and Chiesi close enrolment in Phase III Fabry disease trial
Sep 12, 2019: Freeline doses patient in first Fabry Disease AAV gene therapy trial globally
Aug 02, 2019: Amicus Therapeutics receives marketing authorization for galafold (migalastat) for fabry disease in Argentina
Jul 15, 2019: AVROBIO announces 87% substrate reduction in first kidney biopsy and additional positive data from clinical trials of AVR-RD-01 investigational gene therapy in Fabry Disease
Jul 02, 2019: Handok releases Fabry disease treatment
Jun 17, 2019: Protalix Biotherapeutics completes enrollment in the phase III bright clinical trial of Pegunigalsidase Alfa (PRX 102) for the treatment of Fabry disease
Jun 06, 2019: Protalix BioTherapeutics and Chiesi Farmaceutici to apply for accelerated approval of pegunigalsidase alfa for the treatment of Fabry Disease in the United States
May 30, 2019: 4D Molecular Therapeutics announces presentation of preclinical data at the 6th International Update on Fabry Disease and provides clinical update
May 28, 2019: Freeline presents preclinical data and phase 1/2 study design of the FLT190 AAV gene therapy for Fabry Disease
May 02, 2019: uniQure announces new preclinical data on fabry disease in oral presentations at the 22nd ASGCT Annual Meeting
May 01, 2019: Avrobio to incorporate plato platform into FAB-201 Phase II trial
Apr 15, 2019: uniQure to present data on Gene Therapy for Fabry Disease at ASGCT 2019
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
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
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
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
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
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
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
