Gene Therapy for Mucopolysaccharidosis – A Pipeline Analysis Report

◆상품코드 : IRTNTR20377
◆발행일 : 2018년 2월
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[조사보고서 개요]

This pipeline analysis report provides detailed insights into the clinical trials landscape of the gene therapy for mucopolysaccharidosis including molecules at pre-clinical and various other development stages. The report also offers comprehensive information about the therapeutic assessment of the pipeline molecules based on various segmentations including route of administration
Key questions answered in the report include
• What are the gene therapy molecules in the various development stages for mucopolysaccharidosis?
• What are the companies that are currently involved in the development of gene therapy molecules for mucopolysaccharidosis?
• Insight into discontinued/inactive molecules with appropriate reasoning?
• What are the major regulatory authorities approving drugs in various regions?
• Detailed profiling of each active molecule.

[조사보고서 목차]

Table of Contents
PART 01: EXECUTIVE SUMMARY

PART 02: SCOPE OF THE REPORT

PART 03: RESEARCH METHODOLOGY

PART 04: GENE THERAPY FOR MUCOPOLYSACCHARIDOSES: AN INSIGHT
• Introduction
• MAJOR REGULATORY AUTHORITIES
• US
• Europe
• Japan
• Australia
• China

PART 05: PIPELINE LANDSCAPE

PART 06: COMPARATIVE ANALYSIS
• Mid-stage molecules (Phase I/II), 2017
• Early-stage molecules (pre-IND and IND), 2017
• Pre-clinical stage molecules, 2017
• Suspended molecule, 2017

PART 07: INDICATION ANALYSIS

PART 08: THERAPEUTIC ASSESSMENT BY ROA

PART 09: KEY COMPANIES
• Active companies: Category and parameters
• APPENDIX
• List of abbreviations


Exhibit 01: Drug approval process by US FDA
Exhibit 02: Pipeline molecules (active) by vendors
Exhibit 03: Pipeline landscape
Exhibit 04: Pipeline molecules in different developmental stages 2017
Exhibit 05: Percentage of pipeline molecules in different developmental stages 2017
Exhibit 06: Overview: ABO-101
Exhibit 07: Clinical trials description of ABO-101
Exhibit 08: Overview: ABO-102
Exhibit 09: Clinical trials description of AB0-102
Exhibit 10: Overview: SAF-301
Exhibit 11: Clinical trials description of SAF-301
Exhibit 12: Overview: SAF302
Exhibit 13: Overview: SB-913
Exhibit 14: Clinical trials description of SB-913
Exhibit 15: Overview: SB-318
Exhibit 16: Clinical trials description of SB-318
Exhibit 17: Overview: EGT-101
Exhibit 18: Clinical trials description of EGT-101
Exhibit 19: Overview: Autologous ex-vivo lentiviral gene therapy
Exhibit 20: Overview: RGX-111
Exhibit 21: Overview: RGX-121
Exhibit 22: Overview: EGT-201
Exhibit 23: Overview: EGT-301
Exhibit 24: Overview: Gene therapy for mucopolysaccharidosis I (Hurler’s syndrome)
Exhibit 25: Overview: SOBI003
Exhibit 26: Suspended molecule, 2017
Exhibit 27: Analysis by type of indication
Exhibit 28: Assessment by RoA
Exhibit 29: Assessment of gene therapy for mucopolysaccharidosis by stage and RoA
Exhibit 30: Active companies: Category and parameters 2017
Exhibit 31: Segmentation of companies 2017
Exhibit 32: Overview: Abeona Therapeutics
Exhibit 33: Overview: ESTEVE
Exhibit 34: Overview: Immusoft
Exhibit 35: Overview: Lysogene
Exhibit 36: Overview: Orchard Therapeutics
Exhibit 37: Overview: REGENXBIO
Exhibit 38: Overview: Sangamo Therapeutics
Exhibit 39: Overview: Swedish Orphan Biovitrum
Exhibit 40: Overview: uniQure



[보고서에 포함된 내용]
세계시장규모, 세그멘트별 시장규모, 시장동향, 시장예측, 관련 기업정보 등

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