Home / Therapeutic Area / Genetic Ophthalmology Disorders Drug Development Pipeline Review, 2017

Genetic Ophthalmology Disorders Drug Development Pipeline Review, 2017

Published: Aug 2017 | Published By: GBI Research

Description

This report provides an overview of the pipeline landscape for genetic ophthalmological disorders. The report provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for Stargardt disease, Leber congenital amaurosis, Leber’s hereditary optic neuropathy, Usher Syndrome and Retinitis pigmentosa, and features dormant and discontinued projects.

Juvenile macular degeneration is a series of inherited eye disorders that affects children and young adults, with the most common form being Stargardt disease, an inherited autosomal recessive syndrome. Leber congenital amaurosis primarily affects the retina, which is the specialized tissue at the back of the eye that detects light and color. It is the most common cause of inherited blindness in childhood. Leber’s hereditary optic neuropathy usually begins in a person's teens or twenties, rare cases may appear in early childhood or later in adulthood. Usher syndrome is characterized by hearing impairment and progressive vision loss. The major symptoms of Usher syndrome are hearing loss and secondary retinitis pigmentosa. Finally, retinitis pigmentosa refers to a group of diseases which cause a slow but progressive vision loss. Symptoms include night blindness and loss of peripheral vision.

The size of these pipelines ranges from six products in Usher syndrome to 54 in retinitis pigmentosa. Gene therapies represent the most common type of therapy among these diseases, followed by small molecules. This reflects the therapeutic aim of repairing the defective gene in order to correct the patient’s phenotype. Likewise, the molecular targets which are acted on are typically clustered around the causative gene within each disease, although there are exceptions. Within retinitis pigmentosa in particular, there is a diversity of molecular targets.

Scope

  • Which companies are the most active within the pipeline for genetic ophthalmological disorder therapeutics?
  • Which pharmaceutical approaches are the most prominent at each stage of the pipeline and within each indication?
  • To what extent do universities and institutions play a role within this pipeline, compared to pharmaceutical companies?
  • What are the most important R&D milestones and data publications to have happened in the field of genetic ophthalmological disorders?

Reasons To Buy

  • Understand the overall pipeline, with an at-a-glance overview of all products in therapeutic development for each indication
  • Assess the products in development in granular detail, with an up-to-date overview of each individual pipeline program in each indication, and a comprehensive picture of recent updates and milestones for each
  • Analyze the companies, institutions and universities currently operating in the pipeline, and the products being fielded by each of these
  • Understand the composition of the pipeline in terms of molecule type, molecular target, mechanism of action and route of administration

Executive Summary
Table of Contents
List of Tables
List of Figures
Introduction
Genetic Ophthalmology Disorders Report Coverage
Juvenile Macular Degeneration (Stargardt Disease) – Overview
Leber Congenital Amaurosis (LCA) – Overview
Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) – Overview
Usher Syndrome – Overview
Retinitis Pigmentosa (Retinitis) – Overview
Therapeutics Development
Juvenile Macular Degeneration (Stargardt Disease)
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Leber Congenital Amaurosis (LCA)
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Usher Syndrome
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Retinitis Pigmentosa (Retinitis)
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Therapeutics Assessment
Juvenile Macular Degeneration (Stargardt Disease)
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Leber Congenital Amaurosis (LCA)
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Usher Syndrome
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Retinitis Pigmentosa (Retinitis)
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Companies Involved in Therapeutics Development
Juvenile Macular Degeneration (Stargardt Disease)
Acucela Inc
Alkeus Pharmaceuticals Inc
Astellas Pharma Inc
Copernicus Therapeutics Inc
Grupo Ferrer Internacional SA
Iris Pharma
Sanofi
Leber Congenital Amaurosis (LCA)
AmpliPhi Biosciences Corp
Editas Medicine Inc
Novelion Therapeutics Inc
ProQR Therapeutics NV
Spark Therapeutics Inc
Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
Alkeus Pharmaceuticals Inc
Biovista Inc
GenSight Biologics SA
Ixchel Pharma LLC
Khondrion BV
Mitotech SA
Spark Therapeutics Inc
Stealth BioTherapeutics Inc
Usher Syndrome
Amgen Inc
Editas Medicine Inc
ProQR Therapeutics NV
Sanofi
Retinitis Pigmentosa (Retinitis)
Acucela Inc
Allergan Plc
Amarantus Bioscience Holdings Inc
Amgen Inc
Applied Genetic Technologies Corp
Asklepios BioPharmaceutical Inc
Astellas Pharma Inc
Caladrius Biosciences Inc
Dompe Farmaceutici SpA
GenSight Biologics SA
Grupo Ferrer Internacional SA
ID Pharma Co Ltd
InFlectis BioScience
Ionis Pharmaceuticals Inc
M's Science Corp
Mimetogen Pharmaceuticals Inc
Nanovector srl
Novartis AG
Novelion Therapeutics Inc
ProQR Therapeutics NV
ReNeuron Group Plc
SanBio Inc
Sanofi
Shire Plc
Spark Therapeutics Inc
Sun Pharma Advanced Research Company Ltd
Dormant Projects
Juvenile Macular Degeneration (Stargardt Disease)
Leber Congenital Amaurosis (LCA)
Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
Usher Syndrome
Retinitis Pigmentosa (Retinitis)
Discontinued Products
Retinitis Pigmentosa (Retinitis)
Product Development Milestones
Juvenile Macular Degeneration (Stargardt Disease)
Featured News & Press Releases
Leber Congenital Amaurosis (LCA)
Featured News & Press Releases
Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
Featured News & Press Releases
Usher Syndrome
Featured News & Press Releases
Retinitis Pigmentosa (Retinits)
Featured News & Press Releases
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

Choose License Type
 
Contact Information
24/7 Research Support
Phone: +1-855-455-8662
sales@acutemarketreports.com
Get in Touch with us
join us on Facebook
Follow us on Twitter
Follow us on LinkedIn
Add us on Google +