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Alpha L-Iduronidase - Pipeline Review, H2 2019

DESCRIPTION

TABLE OF CONTENTS

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DESCRIPTION

Alpha L-Iduronidase - Pipeline Review, H2 2019

Alpha L-Iduronidase (IDUA or EC 3.2.1.76) pipeline Target constitutes close to 12 molecules. Out of which approximately 11 molecules are developed by companies and remaining by the universities/institutes. The latest report Alpha L-Iduronidase - Pipeline Review, H2 2019, outlays comprehensive information on the Alpha L-Iduronidase (IDUA or EC 3.2.1.76) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type.

Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Alpha L-iduronidase is an enzyme encoded by IDUA gene. It hydrolyzes the terminal alpha-L-iduronic acid residues of two glycosaminoglycans, dermatan sulfate and heparan sulfate. Mutations in this gene that result in enzymatic deficiency lead to the autosomal recessive disease mucopolysaccharidosis type I (MPS I). The molecules developed by companies in Phase II, Phase I, Preclinical and Discovery stages are 3, 1, 6 and 1 respectively. Similarly, the universities portfolio in Discovery stages comprises 1 molecules, respectively. Report covers products from therapy areas Genetic Disorders which include indications Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ).

Furthermore, this report also reviews key players involved in Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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 L-Iduronidase (IDUA or EC 3.2.1.76)
- The report reviews Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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 L-Iduronidase (IDUA or EC 3.2.1.76) targeted therapeutics and enlists all their major and minor projects
- The report assesses Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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 L-Iduronidase (IDUA or EC 3.2.1.76) 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 L-Iduronidase (IDUA or EC 3.2.1.76)
- 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 its most promising pipeline therapeutics
- Devise corrective measures for pipeline projects by understanding Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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

TABLE OF CONTENTS

Table of Contents
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Overview
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - 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 L-Iduronidase (IDUA or EC 3.2.1.76) - Therapeutics Assessment
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Companies Involved in Therapeutics Development
ArmaGen Inc
BioStrategies LC
bluebird bio Inc
Immusoft Corp
JCR Pharmaceuticals Co Ltd
Orchard Therapeutics Plc
Ossianix Inc
RegenxBio Inc
Sangamo Therapeutics Inc
Tamid Bio Inc
Tega Therapeutics Inc
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Drug Profiles
AGT-181 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Alpha L-Iduronidase for Mucopolysaccharidosis I (MPS I) - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ISP-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
laronidase - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
OTL-203 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Alpha L-Iduronidase Replacement for Mucopolysaccharidosis I (MPS I) - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RGX-111 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SB-318 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Tamid-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
TXB-4LS1 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
X-372 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Dormant Products
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Product Development Milestones
Featured News & Press Releases
Dec 08, 2019: Orchard Therapeutics presents clinical data on OTL-203, at the 61st American Society of Hematology Annual Meeting
Nov 06, 2019: Orchard Therapeutics to present on its Hurler Syndrome drug candidate OTL-203 at the 61st American Society of Hematology Annual Meeting
Sep 04, 2019: Orchard Therapeutics announces encouraging update from proof-of-concept study of OTL-203 for the treatment of Mucopolysaccharidosis Type I (MPS-I)
Aug 27, 2019: Orchard Therapeutics announces presentation on OTL-203 at Upcoming Society for the Study of Inborn Errors of Metabolism (SSIEM) 2019 Annual Symposium
Jul 17, 2019: Immusoft announces the appointment of Robert Hayes, Ph.D. as Chief Scientific Officer
Apr 02, 2019: Sangamo provided an update on its in vivo genome editing program: SB-318
Feb 12, 2019: JCR announces presentation on JR-171 for hurler syndrome at the 15th Annual ORLDSymposium 2019
Feb 07, 2019: Sangamo Announces Interim Results Of Phase 1/2 EMPOWERS Study Evaluating SB-318 Zinc Finger Nuclease (ZFN) In Vivo Genome Editing Demonstrating Increased Leukocyte IDUA Activity In Patients With MPS I
Feb 01, 2019: Sangamo Therapeutics to host conference call to review interim results from phase 1/2 EMPOWERS study for Mucopolysaccharidosis type I
Jan 04, 2019: Sangamo announces upcoming clinical data Presentation on SB-318 At WORLDSymposium 2019
Oct 17, 2018: Immusoft receives rare pediatric disease designation for treatment of MPS I
Jul 23, 2018: Sangamo treats first patient in EMPOWERS Study of SB-318
Jul 10, 2018: ArmaGens AGT-181 52-Week Phase 1/2 Proof-of-Concept Study Results Published in Orphanet Journal of Rare Diseases
Jun 12, 2018: REGENXBIO Receives FDA Fast Track Designation for RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I
Jun 04, 2018: Sangamo Announces U.K. Authorization Of Clinical Trials Evaluating Zinc Finger Nuclease In Vivo Genome Editing Treatment SB-318 For MPS I
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 ArmaGen Inc, H2 2019
Pipeline by BioStrategies LC, H2 2019
Pipeline by bluebird bio Inc, H2 2019
Pipeline by Immusoft Corp, H2 2019
Pipeline by JCR Pharmaceuticals Co Ltd, H2 2019
Pipeline by Orchard Therapeutics Plc, H2 2019
Pipeline by Ossianix Inc, H2 2019
Pipeline by RegenxBio Inc, H2 2019
Pipeline by Sangamo Therapeutics Inc, H2 2019
Pipeline by Tamid Bio Inc, H2 2019
Pipeline by Tega Therapeutics Inc, H2 2019
Dormant Projects, 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

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