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Alpha N-Acetylglucosaminidase - Pipeline Review, H2 2019

December 2019 | 52 pages | ID: A84A4019A3F0EN
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Alpha N-Acetylglucosaminidase - Pipeline Review, H2 2019

SUMMARY

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.

Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) pipeline Target constitutes close to 7 molecules. The molecules developed by companies in Phase II, Preclinical and Discovery stages are 2, 4 and 1 respectively. Report covers products from therapy areas Genetic Disorders which include indications Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ).

The latest report Alpha N-Acetylglucosaminidase - Pipeline Review, H2 2019, 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. It 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.

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 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
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 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
Allievex Corp
Amicus Therapeutics Inc
ArmaGen Inc
Esteve Pharmaceuticals SA
Orchard Therapeutics Plc
Phoenix Nest Inc
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
AGT-187 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
EGT-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate NAGLU for Mucopolysaccharidosis Type IIIB - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate NAGLU for Mucopolysaccharidosis Type IIIB - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
OTL-202 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
tralesinidase alfa - 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) - Discontinued Products
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) - Product Development Milestones
Featured News & Press Releases
May 14, 2019: Abeona Therapeutics treats first patient in second cohort of Phase 1/2 clinical trial for ABO-101 in Sanfilippo Syndrome Type B (MPS IIIB)
Apr 04, 2019: Abeona Therapeutics receives FDA Fast Track Designation for ABO-101 for treatment of Sanfilippo Syndrome Type B (MPS IIIB)
Feb 04, 2019: BioMarin announces presentation of data on drug candidate BMN 250 at 15th Annual WORLDSymposium 2019
Sep 12, 2018: Abeona Therapeutics announces authorization to commence phase 1/2 gene therapy clinical Study for patients with MPS IIIB in Spain
Feb 07, 2018: Abeona Therapeutics Reports on Initial Safety and Biopotency Signals in MPS IIIB Gene Therapy Clinical Trial
Feb 07, 2018: BioMarin Presents Interim Data of Phase 1/2 Study of BMN 250 for Treatment of Sanfilippo B Syndrome (MPS IIIB) at WORLDSymposium 2018
Dec 20, 2017: Abeona Therapeutics Enrolls First Patient in ABO-101 Phase 1/2 Clinical Trial for MPS IIIB
Sep 06, 2017: BioMarin Presents Interim Data of Phase 1/2 Study of BMN 250 for Treatment of Sanfilippo B Syndrome at 13th International Congress of Inborn Errors of Metabolism 2017
Sep 05, 2017: BioMarin To Present Data On ICV BMN 250 At 13th International Congress of Inborn Errors of Metabolism 2017
Jan 19, 2017: Abeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-101 Gene Therapy in Sanfilippo Syndrome Type B
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)
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
May 20, 2015: PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations For ABX-101 From FDA
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 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 Abeona Therapeutics Inc, H2 2019
Pipeline by Allievex Corp, H2 2019
Pipeline by Amicus Therapeutics Inc, H2 2019
Pipeline by ArmaGen Inc, H2 2019
Pipeline by Esteve Pharmaceuticals SA, H2 2019
Pipeline by Orchard Therapeutics Plc, H2 2019
Pipeline by Phoenix Nest Inc, 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

Abeona Therapeutics Inc
Allievex Corp
Amicus Therapeutics Inc
ArmaGen Inc
Esteve Pharmaceuticals SA
Orchard Therapeutics Plc
Phoenix Nest Inc


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