Overview of the gene therapy pipeline for mucopolysaccharidosis
Mucopolysaccharidosis is a rare and autosomal recessive disease caused by an inherited deficiency of an enzyme that is involved in the degradation of acid glycosaminoglycans. Some of the symptoms of this disease include abnormalities of the skeleton, liver and spleen enlargement, heart abnormalities, and neurological complications. It has been observed that in the recent years, mucopolysaccharidoses I had the highest incidence and prevalence rate among the various types of mucopolysaccharidosis. Therapies already available for mucopolysaccharidosis are focused on slowing down its progression and currently, there is no permanent cure for this disease. The market research analysts have predicted that with the replacement gene therapy will emerge as one of the most promising approach to treat mucopolysaccharidoses and the global mucopolysaccharidoses market will witness significant growth in the coming years.
According to this pipeline analysis report, majority of molecules in the pipeline are targeted at mucopolysaccharidosis III A (sanfilippo type A syndrome) and five major companies are involved in the development of gene therapy molecules for this disease. The only company focusing on the development of treatment for mucopolysaccharidosis III B (sanfilippo type B syndrome) is Abeona Therapeutics.
This pipeline analysis report provides a detailed analysis of the companies that are actively involved in the development of gene therapy molecules for the treatment of mucopolysaccharidosis. In addition to providing detailed information on the various stages of molecules developed for different indications, this pipeline analysis report also provides information about the gene therapy molecules discontinued by companies.
Some of the companies covered in this pipeline analysis report are
- Sangamo Therapeutics
- Swedish Orphan Biovitrum
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.
PART 02: SCOPE OF THE REPORT
PART 03: RESEARCH METHODOLOGY
PART 04: GENE THERAPY FOR MUCOPOLYSACCHARIDOSES: AN INSIGHT
- MAJOR REGULATORY AUTHORITIES
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 08: THERAPEUTIC ASSESSMENT BY ROA
PART 09: KEY COMPANIES
- Active companies: Category and parameters
- 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