Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - Pipeline Review, H2 2017
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - Pipeline Review, H2 2017
Summary
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) pipeline Target constitutes close to 5 molecules. Out of which approximately 5 molecules are developed by Companies. The latest report Alpha N-Acetylglucosaminidase - Pipeline Review, H2 2017, outlays comprehensive information on the Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type.
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - Alpha-N-acetylglucosaminidase is a protein associated with Sanfilippo syndrome. This enzyme is located in lysosomes compartments within cells that digest and recycle different types of molecules. Alpha-N-acetylglucosaminidase is involved in the step wise breakdown of large molecules called glycosaminoglycans (GAGs). Alpha-N-acetylglucosaminidase removes a sugar called N-acetylglucosamine when it is at the end of the GAG chain. The molecules developed by companies in Phase II, IND/CTA Filed, Preclinical and Discovery stages are 2, 1, 1 and 1 respectively. Report covers products from therapy areas Genetic Disorders which include indications Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ).
Furthermore, this report also reviews key players involved in Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) 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.
Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
Scope
Summary
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) pipeline Target constitutes close to 5 molecules. Out of which approximately 5 molecules are developed by Companies. The latest report Alpha N-Acetylglucosaminidase - Pipeline Review, H2 2017, outlays comprehensive information on the Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type.
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - Alpha-N-acetylglucosaminidase is a protein associated with Sanfilippo syndrome. This enzyme is located in lysosomes compartments within cells that digest and recycle different types of molecules. Alpha-N-acetylglucosaminidase is involved in the step wise breakdown of large molecules called glycosaminoglycans (GAGs). Alpha-N-acetylglucosaminidase removes a sugar called N-acetylglucosamine when it is at the end of the GAG chain. The molecules developed by companies in Phase II, IND/CTA Filed, Preclinical and Discovery stages are 2, 1, 1 and 1 respectively. Report covers products from therapy areas Genetic Disorders which include indications Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ).
Furthermore, this report also reviews key players involved in Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) 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.
Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
Scope
- The report provides a snapshot of the global therapeutic landscape for Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50)
- The report reviews Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) 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 N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) targeted therapeutics and enlists all their major and minor projects
- The report assesses Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) 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 N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) 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 N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50)
- 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 N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) 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 N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - Overview
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - 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
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - Therapeutics Assessment
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - Companies Involved in Therapeutics Development
Abeona Therapeutics Inc
Alexion Pharmaceuticals Inc
BioMarin Pharmaceutical Inc
Laboratorios Del Dr Esteve SA
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - Drug Profiles
ABO-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BMN-250 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
EGT-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
lesinidase alfa - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy to Activate NAGLU for Mucopolysaccharidosis IIIB - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - Dormant Products
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - Product Development Milestones
Featured News & Press Releases
Jan 19, 2017: Abeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-101 Gene Therapy in Sanfilippo Syndrome Type B
Jul 14, 2016: Alexion Presents New SBC-103 (rhNAGLU enzyme) Phase 1/2 Data on Brain MRI and Neurocognitive Assessments in Patients with Mucopolysaccharidosis IIIB (MPS IIIB)
May 24, 2016: Abeona Therapeutics Announces FDA Allowance of Investigational New Drug for Phase 1/2 Clinical Study With ABO-101 Gene Therapy for Patients With Sanfilippo Syndrome Type B (MPS IIIB)
Apr 21, 2016: BioMarin Enrolls First Patient in Phase 1/2 Trial of NAGLU Fusion Protein BMN 250 for Treatment of MPS IIIB (Sanfilippo B Syndrome)
Mar 01, 2016: SBC-103 (rhNAGLU enzyme) Shows a 26.2 Percent Mean Reduction in Heparan Sulfate in Cerebrospinal Fluid at the Highest Dose Studied in Patients with Mucopolysaccharidosis IIIB in Phase 1/2 Study at Six Months
Feb 25, 2016: ESTEVE Provides Update On EGT-201 For Sanfilippo B Syndrome
Jan 11, 2016: Abeona Therapeutics Announces Initial European Regulatory Approval for Phase 1/2 Gene Therapy Clinical Study for Patients With Sanfilippo Syndrome
Jun 04, 2015: Synageva BioPharma Completes Targeted Enrollment in Phase 1/2 Trial with SBC-103 for Mucopolysaccharidosis IIIB
May 20, 2015: PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations For ABX-101 From FDA
Jan 26, 2015: Synageva BioPharma Announces Dosing Of Patients Commenced With SBC-103 In Phase 1/2 Study For Mucopolysaccharidosis IIIB And FDA Fast Track Designation Granted
Dec 15, 2014: Synageva BioPharma Announces Active Investigational New Drug Application For SBC-103 For The Treatment Of Mucopolysaccharidosis IIIB
Dec 10, 2014: FDA Grants BioMarin Orphan Drug Designation for NAGLU Fusion Protein, BMN 250, for the Treatment of MPS IIIB (Sanfilippo Syndrome Type B)
Feb 12, 2014: Synageva BioPharma Highlights SBC-103 Data at the Lysosomal Disease Network WORLD Symposium
Feb 11, 2014: BioMarin Announces Selection of NAGLU Fusion Protein Drug Development Candidate BMN 250 for the Treatment of Sanfilippo B
Jan 29, 2014: Synageva BioPharma Announces Data Presentations On SBC-103 at the 10th Annual Lysosomal Disease Network World Symposium
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
Global Markets Direct Report Coverage
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - Overview
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - 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
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - Therapeutics Assessment
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - Companies Involved in Therapeutics Development
Abeona Therapeutics Inc
Alexion Pharmaceuticals Inc
BioMarin Pharmaceutical Inc
Laboratorios Del Dr Esteve SA
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - Drug Profiles
ABO-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BMN-250 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
EGT-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
lesinidase alfa - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy to Activate NAGLU for Mucopolysaccharidosis IIIB - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - Dormant Products
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - Product Development Milestones
Featured News & Press Releases
Jan 19, 2017: Abeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-101 Gene Therapy in Sanfilippo Syndrome Type B
Jul 14, 2016: Alexion Presents New SBC-103 (rhNAGLU enzyme) Phase 1/2 Data on Brain MRI and Neurocognitive Assessments in Patients with Mucopolysaccharidosis IIIB (MPS IIIB)
May 24, 2016: Abeona Therapeutics Announces FDA Allowance of Investigational New Drug for Phase 1/2 Clinical Study With ABO-101 Gene Therapy for Patients With Sanfilippo Syndrome Type B (MPS IIIB)
Apr 21, 2016: BioMarin Enrolls First Patient in Phase 1/2 Trial of NAGLU Fusion Protein BMN 250 for Treatment of MPS IIIB (Sanfilippo B Syndrome)
Mar 01, 2016: SBC-103 (rhNAGLU enzyme) Shows a 26.2 Percent Mean Reduction in Heparan Sulfate in Cerebrospinal Fluid at the Highest Dose Studied in Patients with Mucopolysaccharidosis IIIB in Phase 1/2 Study at Six Months
Feb 25, 2016: ESTEVE Provides Update On EGT-201 For Sanfilippo B Syndrome
Jan 11, 2016: Abeona Therapeutics Announces Initial European Regulatory Approval for Phase 1/2 Gene Therapy Clinical Study for Patients With Sanfilippo Syndrome
Jun 04, 2015: Synageva BioPharma Completes Targeted Enrollment in Phase 1/2 Trial with SBC-103 for Mucopolysaccharidosis IIIB
May 20, 2015: PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations For ABX-101 From FDA
Jan 26, 2015: Synageva BioPharma Announces Dosing Of Patients Commenced With SBC-103 In Phase 1/2 Study For Mucopolysaccharidosis IIIB And FDA Fast Track Designation Granted
Dec 15, 2014: Synageva BioPharma Announces Active Investigational New Drug Application For SBC-103 For The Treatment Of Mucopolysaccharidosis IIIB
Dec 10, 2014: FDA Grants BioMarin Orphan Drug Designation for NAGLU Fusion Protein, BMN 250, for the Treatment of MPS IIIB (Sanfilippo Syndrome Type B)
Feb 12, 2014: Synageva BioPharma Highlights SBC-103 Data at the Lysosomal Disease Network WORLD Symposium
Feb 11, 2014: BioMarin Announces Selection of NAGLU Fusion Protein Drug Development Candidate BMN 250 for the Treatment of Sanfilippo B
Jan 29, 2014: Synageva BioPharma Announces Data Presentations On SBC-103 at the 10th Annual Lysosomal Disease Network World Symposium
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 2017
Number of Products under Development by Therapy Areas, H2 2017
Number of Products under Development by Indication, H2 2017
Number of Products under Development by Companies, H2 2017
Products under Development by Companies, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Stage and Route of Administration, H2 2017
Number of Products by Stage and Molecule Type, H2 2017
Pipeline by Abeona Therapeutics Inc, H2 2017
Pipeline by Alexion Pharmaceuticals Inc, H2 2017
Pipeline by BioMarin Pharmaceutical Inc, H2 2017
Pipeline by Laboratorios Del Dr Esteve SA, H2 2017
Dormant Projects, H2 2017
Number of Products under Development by Stage of Development, H2 2017
Number of Products under Development by Therapy Areas, H2 2017
Number of Products under Development by Indication, H2 2017
Number of Products under Development by Companies, H2 2017
Products under Development by Companies, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Stage and Route of Administration, H2 2017
Number of Products by Stage and Molecule Type, H2 2017
Pipeline by Abeona Therapeutics Inc, H2 2017
Pipeline by Alexion Pharmaceuticals Inc, H2 2017
Pipeline by BioMarin Pharmaceutical Inc, H2 2017
Pipeline by Laboratorios Del Dr Esteve SA, H2 2017
Dormant Projects, H2 2017
LIST OF FIGURES
Number of Products under Development by Stage of Development, H2 2017
Number of Products by Mechanism of Actions, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Routes of Administration, H2 2017
Number of Products by Stage and Routes of Administration, H2 2017
Number of Products by Molecule Types, H2 2017
Number of Products by Stage and Molecule Types, H2 2017
Number of Products under Development by Stage of Development, H2 2017
Number of Products by Mechanism of Actions, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Routes of Administration, H2 2017
Number of Products by Stage and Routes of Administration, H2 2017
Number of Products by Molecule Types, H2 2017
Number of Products by Stage and Molecule Types, H2 2017
