Alpha L-Iduronidase (IDUA or EC 3.2.1.76) Development by Therapy Areas and Indications, Stages, MoA, RoA, Molecule Type and Key Players, 2022 Update

March 2022 | 62 pages | ID: A0BD469C74FEEN
Global Markets Direct

Abstracts

Alpha L-Iduronidase (IDUA or EC 3.2.1.76) Development by Therapy Areas and Indications, Stages, MoA, RoA, Molecule Type and Key Players, 2022 Update

SUMMARY

According to the recently published report 'Alpha L-Iduronidase - Drugs In Development, 2022'; Alpha L-Iduronidase (IDUA or EC 3.2.1.76) pipeline Target constitutes close to 14 molecules. Out of which approximately 13 molecules are developed by companies and remaining by the universities/institutes.

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 report 'Alpha L-Iduronidase - Drugs In Development, 2022' 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; that are being developed by Companies / Universities.

It 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. Currently, The molecules developed by companies in Phase II, IND/CTA Filed, Preclinical and Discovery stages are 4, 1, 6 and 2 respectively. Similarly, the universities portfolio in Discovery stages comprises 1 molecules, respectively. Report covers products from therapy areas Genetic Disorders, Central Nervous System, Musculoskeletal Disorders, Oncology and Undisclosed which include indications Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), Unspecified, Unspecified Cancer, Unspecified Musculoskeletal Disorders and Unspecified Neurologic Disorders.

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

Contents

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
RainBio Inc
RegenxBio Inc
Sigilon Therapeutics 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
History of Events
Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
History of Events
Fusion Protein to Replace IDUA for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
ISP-001 - Drug Profile
Product Description
Mechanism Of Action
History of Events
laronidase - Drug Profile
Product Description
Mechanism Of Action
History of Events
Oligonucleotide to Activate IDUA for Hurler Syndrome, Unspecified Cancer, Unspecified Muscular Diseases, Unspecified Neurologic Disorders, and Unspecified Non-Genetic Diseases - Drug Profile
Product Description
Mechanism Of Action
History of Events
OTL-203 - Drug Profile
Product Description
Mechanism Of Action
History of Events
RBIO-1 - Drug Profile
Product Description
Mechanism Of Action
History of Events
Recombinant Alpha L-Iduronidase Replacement for Mucopolysaccharidosis I (MPS I) - Drug Profile
Product Description
Mechanism Of Action
History of Events
Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
History of Events
RGX-111 - Drug Profile
Product Description
Mechanism Of Action
History of Events
SIG-005 - Drug Profile
Product Description
Mechanism Of Action
History of Events
TXB-4LS1 - Drug Profile
Product Description
Mechanism Of Action
X-372 - Drug Profile
Product Description
Mechanism Of Action
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
Feb 09, 2022: REGENXBIO presents positive initial data from Phase I/II of RGX-111 for the treatment of severe MPS I at 18th annual WORLDSymposium 2022
Feb 07, 2022: Immusoft to present at WORLDSymposium 2022 Conference
Feb 04, 2022: JCR pharmaceuticals to present posters on JR-171 at the 18th annual WORLDSymposium 2022
Jan 31, 2022: Sigilon Therapeutics to present preclinical data at the 18th Annual WORLDSymposium on Lysosomal Diseases, MPS-1 and MPS-6
Nov 18, 2021: Orchard Therapeutics announces new New England Journal Medicine publication of interim proof-of-concept study results of OTL-203 for Hurler Syndrome
Oct 05, 2021: JCR Pharmaceuticals: US FDA grants Fast Track Designation for JR-171 for the treatment of Mucopolysaccaridosis Type I (MPSI)
Sep 09, 2021: Sigilon Therapeutics announces acceptance of clinical trial application in the UK for SIG-005 for the treatment of mucopolysaccharidosis type I
Jul 23, 2021: Sigilon Therapeutics presents preclinical data at the 16th International Symposium on MPS and Related Diseases
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 17, 2020: Sigilon Therapeutics receives Orphan Drug Designation for SIG-005 for the treatment of mucopolysaccharidosis type I
Dec 01, 2020: RegenxBio announces dosing of first patient in phase I/II trial of RGX-111 for the treatment of Mucopolysaccharidosis type I
Oct 30, 2020: JCR announces first patient dosed in phase 1/2 global clinical trial of JR-171 for 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, 2022
Number of Products under Development by Therapy Areas, 2022
Number of Products under Development by Indication, 2022
Number of Products under Development by Companies, 2022
Products under Development by Companies, 2022
Products under Development by Companies, 2022 (Contd..1)
Number of Products under Investigation by Universities/Institutes, 2022
Products under Investigation by Universities/Institutes, 2022
Number of Products by Stage and Mechanism of Actions, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Pipeline by ArmaGen Inc, 2022
Pipeline by BioStrategies LC, 2022
Pipeline by Denali Therapeutics Inc, 2022
Pipeline by EdiGene Inc, 2022
Pipeline by Gain Therapeutics Inc, 2022
Pipeline by Immusoft Corp, 2022
Pipeline by JCR Pharmaceuticals Co Ltd, 2022
Pipeline by Orchard Therapeutics Plc, 2022
Pipeline by Ossianix Inc, 2022
Pipeline by RainBio Inc, 2022
Pipeline by RegenxBio Inc, 2022
Pipeline by Sigilon Therapeutics Inc, 2022
Pipeline by Tega Therapeutics Inc, 2022
Dormant Projects, 2022

List Of Figures

LIST OF FIGURES

Number of Products under Development by Stage of Development, 2022
Number of Products under Development by Therapy Areas, 2022
Number of Products under Development by Top 10 Indications, 2022
Number of Products by Mechanism of Actions, 2022
Number of Products by Stage and Mechanism of Actions, 2022
Number of Products by Routes of Administration, 2022
Number of Products by Stage and Routes of Administration, 2022
Number of Products by Molecule Types, 2022
Number of Products by Stage and Molecule Types, 2022