Alpha L-Iduronidase (IDUA or EC 3.2.1.76) – Drugs In Development, 2021

Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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 L-Iduronidase – Drugs In Development, 2021, 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, Preclinical and Discovery stages are 4, 6 and 2 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.

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 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 it’s 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

Companies mentioned

ArmaGen Inc

BioStrategies LC

Denali Therapeutics Inc

EdiGene Inc

Gain Therapeutics Inc

Immusoft Corp

JCR Pharmaceuticals Co Ltd

Orchard Therapeutics Plc

Ossianix Inc

Rain Bio Inc

RegenxBio Inc

Tega Therapeutics Inc

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

Denali Therapeutics Inc

EdiGene Inc

Gain Therapeutics Inc

Immusoft Corp

JCR Pharmaceuticals Co Ltd

Orchard Therapeutics Plc

Ossianix Inc

Rain Bio Inc

RegenxBio 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

Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Fusion Protein to Replace IDUA for Mucopolysaccharidosis I - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate Alpha-L-Iduronidase for Mucopolysaccharidosis 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

Oligonucleotide to Activate IDUA for Hurler Syndrome - 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

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) - Discontinued Products

Alpha L-Iduronidase (IDUA or EC

3.2.1.76) - Product Development Milestones

Featured News & Press Releases

Mar 30, 2021: EMA grants Orphan Drug Designation to JR-171 for the treatment of Mucopolysaccaridosis Type I (MPS I)

Feb 12, 2021: US FDA grants orphan drug designation to JR-171 for the treatment of Mucopolysaccaridosis Type I (MPS I)

Feb 09, 2021: Orchard Therapeutics announces interim data for OTL-203 showing positive clinical results in multiple disease manifestations of mucopolysaccharidosis type I Hurler syndrome (MPS-IH)

Jan 28, 2021: Orchard Therapeutics to present abstact on OTL-203 at 2021 WORLDSymposium

Dec 01, 2020: RegenxBio announces dosing of first patient in phase I/II trial of RGX-111 for the treatment of Mucopolysaccharidosis type I

Sep 28, 2020: Orchard Therapeutics receives EMA PRIME designation for OTL-203 for the treatment of MPS-I

Sep 01, 2020: Orchard Therapeutics announces additional interim results from proof-of-concept study of OTL-203 for MPS-I

Aug 24, 2020: Orchard Therapeutics announces clinical data presentations at the 46th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT)

Jul 20, 2020: Orchard Therapeutics announces orphan drug and rare pediatric disease designations for OTL-203 for the treatment of MPS-I

Jul 08, 2020: REGENXBIO provides update on progress of Clinical Programs including RGX-111 for Rare Genetic Neurodegenerative Diseases

May 15, 2020: Orchard Therapeutics presents new interim data from OTL-203 proof-of-concept study for MPS-I

Feb 10, 2020: Orchard Therapeutics announces presentation on OTL-203 at 16th Annual WORLD Symposium

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)

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, 2021

Number of Products under Development by Therapy Areas, 2021

Number of Products under Development by Indication, 2021

Number of Products under Development by Companies, 2021

Products under Development by Companies, 2021

Number of Products under Investigation by Universities/Institutes, 2021

Products under Investigation by Universities/Institutes, 2021

Number of Products by Stage and Mechanism of Actions, 2021

Number of Products by Stage and Route of Administration, 2021

Number of Products by Stage and Molecule Type, 2021

Pipeline by ArmaGen Inc, 2021

Pipeline by BioStrategies LC, 2021

Pipeline by Denali Therapeutics Inc, 2021

Pipeline by EdiGene Inc, 2021

Pipeline by Gain Therapeutics Inc, 2021

Pipeline by Immusoft Corp, 2021

Pipeline by JCR Pharmaceuticals Co Ltd, 2021

Pipeline by Orchard Therapeutics Plc, 2021

Pipeline by Ossianix Inc, 2021

Pipeline by Rain Bio Inc, 2021

Pipeline by RegenxBio Inc, 2021

Pipeline by Tega Therapeutics Inc, 2021

Dormant Projects, 2021

Discontinued Products, 2021

List of Figures

List of Figures

Number of Products under Development by Stage of Development, 2021

Number of Products by Mechanism of Actions, 2021

Number of Products by Stage and Mechanism of Actions, 2021

Number of Products by Routes of Administration, 2021

Number of Products by Stage and Routes of Administration, 2021

Number of Products by Molecule Types, 2021

Number of Products by Stage and Molecule Types, 2021

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