Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Pipeline Review, H1 2018
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Pipeline Review, H1 2018
SUMMARY
According to the recently published report 'Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Pipeline Review, H1 2018'; Alpha L-Iduronidase (IDUA or EC 3.2.1.76) pipeline Target constitutes close to 11 molecules. Out of which approximately 9 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 (IDUA or EC 3.2.1.76) - Pipeline Review, H1 2018' 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 2, 1, 4 and 2 respectively. Similarly, the universities portfolio in Preclinical and Discovery stages comprises 1 and 1 molecules, respectively. Report covers products from therapy areas Genetic Disorders which include indications Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ).
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
SCOPE
SUMMARY
According to the recently published report 'Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Pipeline Review, H1 2018'; Alpha L-Iduronidase (IDUA or EC 3.2.1.76) pipeline Target constitutes close to 11 molecules. Out of which approximately 9 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 (IDUA or EC 3.2.1.76) - Pipeline Review, H1 2018' 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 2, 1, 4 and 2 respectively. Similarly, the universities portfolio in Preclinical and Discovery stages comprises 1 and 1 molecules, respectively. Report covers products from therapy areas Genetic Disorders which include indications Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ).
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
- 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
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
AngioChem Inc
ArmaGen Inc
CRISPR Therapeutics
Immusoft Corp
JCR Pharmaceuticals Co Ltd
RegenxBio Inc
Sangamo 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
Cell Therapy to Activate IDUA for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
laronidase - 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
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
Stem Cell Therapy to Activate Alpha L-Iduronidase for Type1 Mucopolysaccharidosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy to Target Alpha L-Iduronidase for Hurler Sydrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Tamid-001 - 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
May 02, 2018: Immusoft Granted Orphan Drug Designation for Treatment of Rare Childhood Disease
Feb 08, 2018: ArmaGen’s AGT-181 Demonstrates Neurocognitive Benefit in Children with Severe MPS I
Dec 08, 2017: Sangamo Announces EMA Recommendation of Orphan Medicinal Product Designation for Investigational Genome Editing Treatment for Mucopolysaccharidosis Type I
Dec 05, 2017: Fortress Biotech Forms Subsidiary Tamid Bio to Develop Novel AAV Gene Therapies in Orphan Diseases With Unmet Medical Needs
Nov 30, 2017: ArmaGen's AGT-181 Granted Fast Track Designation for the Treatment of Hurler Syndrome (MPS I)
Oct 26, 2017: JCR to Initiate Development of JR-171, a New Drug Candidate for Hurler Syndrome Using J-Brain Cargo
Aug 08, 2017: REGENXBIO Announces IND Active for Phase I Trial of RGX-111 to Treat Mucopolysaccharidosis Type I
Jul 13, 2017: Sangamo Receives Fast Track Designation From The FDA For SB-318 In Vivo Genome Editing Product Candidate For The Treatment Of MPS I
Feb 27, 2017: Sangamo Therapeutics Receives Rare Pediatric Disease Designation From FDA For SB-318 In Vivo Genome Editing Therapeutic For MPS I
Feb 16, 2017: ArmaGen Reports Preliminary Evidence of Cognitive Improvement in Children with Hurler Syndrome (MPS I) Treated with AGT-181
Feb 08, 2017: Sangamo Therapeutics Announces Data on SB-318 at The 13th Annual WORLDSymposium Meeting
Feb 07, 2017: ArmaGen Announces Oral Presentation of Preliminary Results from its Phase 2 Clinical Trial of AGT-181 in Patients with MPS 1 to be Presented at WORLDSymposium 2017
Jan 11, 2017: Sangamo Therapeutics Receives Orphan Drug Designation from the FDA for SB-318 Genome Editing Treatment for MPS I
Sep 13, 2016: REGENXBIO Publishes Data from Ongoing Preclinical Studies of NAV Gene Therapy RGX-111
Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-111
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
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
AngioChem Inc
ArmaGen Inc
CRISPR Therapeutics
Immusoft Corp
JCR Pharmaceuticals Co Ltd
RegenxBio Inc
Sangamo 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
Cell Therapy to Activate IDUA for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
laronidase - 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
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
Stem Cell Therapy to Activate Alpha L-Iduronidase for Type1 Mucopolysaccharidosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy to Target Alpha L-Iduronidase for Hurler Sydrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Tamid-001 - 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
May 02, 2018: Immusoft Granted Orphan Drug Designation for Treatment of Rare Childhood Disease
Feb 08, 2018: ArmaGen’s AGT-181 Demonstrates Neurocognitive Benefit in Children with Severe MPS I
Dec 08, 2017: Sangamo Announces EMA Recommendation of Orphan Medicinal Product Designation for Investigational Genome Editing Treatment for Mucopolysaccharidosis Type I
Dec 05, 2017: Fortress Biotech Forms Subsidiary Tamid Bio to Develop Novel AAV Gene Therapies in Orphan Diseases With Unmet Medical Needs
Nov 30, 2017: ArmaGen's AGT-181 Granted Fast Track Designation for the Treatment of Hurler Syndrome (MPS I)
Oct 26, 2017: JCR to Initiate Development of JR-171, a New Drug Candidate for Hurler Syndrome Using J-Brain Cargo
Aug 08, 2017: REGENXBIO Announces IND Active for Phase I Trial of RGX-111 to Treat Mucopolysaccharidosis Type I
Jul 13, 2017: Sangamo Receives Fast Track Designation From The FDA For SB-318 In Vivo Genome Editing Product Candidate For The Treatment Of MPS I
Feb 27, 2017: Sangamo Therapeutics Receives Rare Pediatric Disease Designation From FDA For SB-318 In Vivo Genome Editing Therapeutic For MPS I
Feb 16, 2017: ArmaGen Reports Preliminary Evidence of Cognitive Improvement in Children with Hurler Syndrome (MPS I) Treated with AGT-181
Feb 08, 2017: Sangamo Therapeutics Announces Data on SB-318 at The 13th Annual WORLDSymposium Meeting
Feb 07, 2017: ArmaGen Announces Oral Presentation of Preliminary Results from its Phase 2 Clinical Trial of AGT-181 in Patients with MPS 1 to be Presented at WORLDSymposium 2017
Jan 11, 2017: Sangamo Therapeutics Receives Orphan Drug Designation from the FDA for SB-318 Genome Editing Treatment for MPS I
Sep 13, 2016: REGENXBIO Publishes Data from Ongoing Preclinical Studies of NAV Gene Therapy RGX-111
Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-111
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, H1 2018
Number of Products under Development by Therapy Areas, H1 2018
Number of Products under Development by Indication, H1 2018
Number of Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018
Number of Products under Investigation by Universities/Institutes, H1 2018
Products under Investigation by Universities/Institutes, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Pipeline by AngioChem Inc, H1 2018
Pipeline by ArmaGen Inc, H1 2018
Pipeline by CRISPR Therapeutics, H1 2018
Pipeline by Immusoft Corp, H1 2018
Pipeline by JCR Pharmaceuticals Co Ltd, H1 2018
Pipeline by RegenxBio Inc, H1 2018
Pipeline by Sangamo Therapeutics Inc, H1 2018
Dormant Projects, H1 2018
Number of Products under Development by Stage of Development, H1 2018
Number of Products under Development by Therapy Areas, H1 2018
Number of Products under Development by Indication, H1 2018
Number of Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018
Number of Products under Investigation by Universities/Institutes, H1 2018
Products under Investigation by Universities/Institutes, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Pipeline by AngioChem Inc, H1 2018
Pipeline by ArmaGen Inc, H1 2018
Pipeline by CRISPR Therapeutics, H1 2018
Pipeline by Immusoft Corp, H1 2018
Pipeline by JCR Pharmaceuticals Co Ltd, H1 2018
Pipeline by RegenxBio Inc, H1 2018
Pipeline by Sangamo Therapeutics Inc, H1 2018
Dormant Projects, H1 2018
LIST OF FIGURES
Number of Products under Development by Stage of Development, H1 2018
Number of Products by Mechanism of Actions, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Molecule Types, H1 2018
COMPANIES MENTIONED
AngioChem Inc
ArmaGen Inc
CRISPR Therapeutics
Immusoft Corp
JCR Pharmaceuticals Co Ltd
RegenxBio Inc
Sangamo Therapeutics Inc
Number of Products under Development by Stage of Development, H1 2018
Number of Products by Mechanism of Actions, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Molecule Types, H1 2018
COMPANIES MENTIONED
AngioChem Inc
ArmaGen Inc
CRISPR Therapeutics
Immusoft Corp
JCR Pharmaceuticals Co Ltd
RegenxBio Inc
Sangamo Therapeutics Inc
