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Genetic Ophthalmology Disorders Drug Development Pipeline Review, 2017

Genetic Ophthalmology Disorders Drug Development Pipeline Review, 2017

Summary


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.There are a total of 14 products in development for this indication, by 11 companies and one academic institution. Key companies operating in this pipeline space include Astellas, and the biomedical charity Fondazione Telethon.

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. There are a total of nine products in development for this indication, by six companies and two academic institutions. Spark therapeutics, who are fielding two products in this indication, are the most active pipeline developer for Leber congenital amaurosis.

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. Males are affected much more often than females.There are a total of 12 products in development for this indication, by eight companies and one academic institution. Key companies operating in this pipeline space include Biovista, GenSight Biologics and Ixchel Pharma.

Usher syndrome is characterized by hearing impairment and progressive vision loss. The major symptoms of Usher syndrome are hearing loss and secondary retinitis pigmentosa.There are a total of six products in development for this indication, by four companies and one academic institution. ProQR Therapeutics are fielding two products in this indication, while all other players are fielding one each.

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. This has the most active pipeline, with a total of 54 products in development, by 36 companies and 11 academic institutions. Key companies operating in this pipeline space include Spark Therapeutics, Astellas, Horama, ID Pharma, Columbia University Medical Center and John Hopkins University.

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.

The report Genetic Ophthalmology Disorders Drug Development Pipeline Review, 2017, 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 Stargardtdisease, Lebercongenital amaurosis, Leber’s hereditary optic neuropathy, Usher Syndrome and Retinitis pigmentosa, and features dormant and discontinued projects.

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


  • GBI Research Report Guidance
  • Executive Summary
  • 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)
      • Table Figure 1: Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease)
      • Table Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease)
      • Table Figure 2: Number of Products under Development by Companies, Juvenile Macular Degeneration (Stargardt Disease)
      • Table Number of Products under Development by Companies, Juvenile Macular Degeneration (Stargardt Disease)
      • Table Number of Products under Development by Universities/Institutes, Juvenile Macular Degeneration (Stargardt Disease)
      • Table Products under Development by Companies, Juvenile Macular Degeneration (Stargardt Disease)
      • Table Products under Development by Universities/Institutes, Juvenile Macular Degeneration (Stargardt Disease)
    • Leber Congenital Amaurosis (LCA)
      • Table Figure 3: Number of Products under Development for Leber Congenital Amaurosis (LCA)
      • Table Number of Products under Development for Leber Congenital Amaurosis (LCA)
      • Table Figure 4: Number of Products under Development by Companies, Leber Congenital Amaurosis (LCA)
      • Table Number of Products under Development by Companies, Leber Congenital Amaurosis (LCA)
      • Table Figure 5: Number of Products under Development by Universities/Institutes, Leber Congenital Amaurosis (LCA)
      • Table Number of Products under Development by Universities/Institutes, Leber Congenital Amaurosis (LCA)
      • Table Products under Development by Companies, Leber Congenital Amaurosis (LCA)
      • Table Products under Development by Universities/Institutes, Leber Congenital Amaurosis (LCA)
    • Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Figure 6: Number of Products under Development for Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Number of Products under Development for Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Figure 7: Number of Products under Development by Companies, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Number of Products under Development by Companies, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Number of Products under Development by Universities/Institutes, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Products under Development by Companies, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Products under Development by Universities/Institutes, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
    • Usher Syndrome
      • Table Figure 8: Number of Products under Development for Usher Syndrome
      • Table Number of Products under Development for Usher Syndrome
      • Table Figure 9: Number of Products under Development by Companies, Usher Syndrome
      • Table Number of Products under Development by Companies, Usher Syndrome
      • Table Number of Products under Development by Universities/Institutes, Usher Syndrome
      • Table Products under Development by Companies, Usher Syndrome
      • Table Products under Development by Universities/Institutes, Usher Syndrome
    • Retinitis Pigmentosa (Retinitis)
      • Table Figure 10: Number of Products under Development for Retinitis Pigmentosa (Retinitis)
      • Table Number of Products under Development for Retinitis Pigmentosa (Retinitis)
      • Table Figure 11: Number of Products under Development by Companies, Retinitis Pigmentosa (Retinitis)
      • Table Number of Products under Development by Companies, Retinitis Pigmentosa (Retinitis)
      • Table Figure 12: Number of Products under Development by Universities/Institutes, Retinitis Pigmentosa (Retinitis)
      • Table Number of Products under Development by Universities/Institutes, Retinitis Pigmentosa (Retinitis)
      • Table Products under Development by Companies, Retinitis Pigmentosa (Retinitis)
      • Table Products under Development by Universities/Institutes, Retinitis Pigmentosa (Retinitis)
  • Therapeutics Assessment
    • Juvenile Macular Degeneration (Stargardt Disease)
      • Table Figure 13: Number of Products by Targets, Juvenile Macular Degeneration (Stargardt Disease)
      • Table Figure 14: Number of Products by Stage and Targets, Juvenile Macular Degeneration (Stargardt Disease)
      • Table Number of Products by Stage and Target, Juvenile Macular Degeneration (Stargardt Disease)
      • Table Figure 15: Number of Products by Mechanism of Actions, Juvenile Macular Degeneration (Stargardt Disease)
      • Table Figure 16: Number of Products by Stage and Mechanism of Actions, Juvenile Macular Degeneration (Stargardt Disease)
      • Table Number of Products by Stage and Mechanism of Action, Juvenile Macular Degeneration (Stargardt Disease)
      • Table Figure 17: Number of Products by Routes of Administration, Juvenile Macular Degeneration (Stargardt Disease)
      • Table Figure 18: Number of Products by Stage and Routes of Administration, Juvenile Macular Degeneration (Stargardt Disease)
      • Table Number of Products by Stage and Route of Administration, Juvenile Macular Degeneration (Stargardt Disease)
      • Table Figure 19: Number of Products by Molecule Types, Juvenile Macular Degeneration (Stargardt Disease)
      • Table Figure 20: Number of Products by Stage and Molecule Types, Juvenile Macular Degeneration (Stargardt Disease)
      • Table Number of Products by Stage and Molecule Type, Juvenile Macular Degeneration (Stargardt Disease)
    • Leber Congenital Amaurosis (LCA)
      • Table Figure 21: Number of Products by Targets, Leber Congenital Amaurosis (LCA)
      • Table Figure 22: Number of Products by Stage and Targets, Leber Congenital Amaurosis (LCA)
      • Table Number of Products by Stage and Target, Leber Congenital Amaurosis (LCA)
      • Table Figure 23: Number of Products by Mechanism of Actions, Leber Congenital Amaurosis (LCA)
      • Table Figure 24: Number of Products by Stage and Mechanism of Actions, Leber Congenital Amaurosis (LCA)
      • Table Number of Products by Stage and Mechanism of Action, Leber Congenital Amaurosis (LCA)
      • Table Figure 25: Number of Products by Routes of Administration, Leber Congenital Amaurosis (LCA)
      • Table Figure 26: Number of Products by Stage and Routes of Administration, Leber Congenital Amaurosis (LCA)
      • Table Number of Products by Stage and Route of Administration, Leber Congenital Amaurosis (LCA)
      • Table Figure 27: Number of Products by Molecule Types, Leber Congenital Amaurosis (LCA)
      • Table Figure 28: Number of Products by Stage and Molecule Types, Leber Congenital Amaurosis (LCA)
      • Table Number of Products by Stage and Molecule Type, Leber Congenital Amaurosis (LCA)
    • Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Figure 29: Number of Products by Targets, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Figure 30: Number of Products by Stage and Targets, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Number of Products by Stage and Target, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Figure 31: Number of Products by Mechanism of Actions, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Figure 32: Number of Products by Stage and Mechanism of Actions, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Number of Products by Stage and Mechanism of Action, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Figure 33: Number of Products by Routes of Administration, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Figure 34: Number of Products by Stage and Routes of Administration, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Number of Products by Stage and Route of Administration, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Figure 35: Number of Products by Molecule Types, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Figure 36: Number of Products by Stage and Molecule Types, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Number of Products by Stage and Molecule Type, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
    • Usher Syndrome
      • Table Figure 37: Number of Products by Targets, Usher Syndrome
      • Table Figure 38: Number of Products by Stage and Targets, Usher Syndrome
      • Table Number of Products by Stage and Target, Usher Syndrome
      • Table Figure 39: Number of Products by Mechanism of Actions, Usher Syndrome
      • Table Figure 40: Number of Products by Stage and Mechanism of Actions, Usher Syndrome
      • Table Number of Products by Stage and Mechanism of Action, Usher Syndrome
      • Table Figure 41: Number of Products by Routes of Administration, Usher Syndrome
      • Table Figure 42: Number of Products by Stage and Routes of Administration, Usher Syndrome
      • Table Number of Products by Stage and Route of Administration, Usher Syndrome
      • Table Figure 43: Number of Products by Molecule Types, Usher Syndrome
      • Table Figure 44: Number of Products by Stage and Molecule Types, Usher Syndrome
      • Table Number of Products by Stage and Molecule Type, Usher Syndrome
    • Retinitis Pigmentosa (Retinitis)
      • Table Figure 45: Number of Products by Top 10 Targets, Retinitis Pigmentosa (Retinitis)
      • Table Figure 46: Number of Products by Stage and Top 10 Targets, Retinitis Pigmentosa (Retinitis)
      • Table Number of Products by Stage and Target, Retinitis Pigmentosa (Retinitis)
      • Table Figure 47: Number of Products by Top 10 Mechanism of Actions, Retinitis Pigmentosa (Retinitis)
      • Table Figure 48: Number of Products by Stage and Top 10 Mechanism of Actions, Retinitis Pigmentosa (Retinitis)
      • Table Number of Products by Stage and Mechanism of Action, Retinitis Pigmentosa (Retinitis)
      • Table Figure 49: Number of Products by Routes of Administration, Retinitis Pigmentosa (Retinitis)
      • Table Figure 50: Number of Products by Stage and Routes of Administration, Retinitis Pigmentosa (Retinitis)
      • Table Number of Products by Stage and Route of Administration, Retinitis Pigmentosa (Retinitis)
      • Table Figure 51: Number of Products by Molecule Types, Retinitis Pigmentosa (Retinitis)
      • Table Figure 52: Number of Products by Stage and Molecule Types, Retinitis Pigmentosa (Retinitis)
      • Table Number of Products by Stage and Molecule Type, Retinitis Pigmentosa (Retinitis)
  • Companies Involved in Therapeutics Development
    • Juvenile Macular Degeneration (Stargardt Disease)
      • Table Juvenile Macular Degeneration (Stargardt Disease) –Pipeline by Acucela Inc
      • Table Juvenile Macular Degeneration (Stargardt Disease) –Pipeline by Alkeus Pharmaceuticals Inc
      • Table Juvenile Macular Degeneration (Stargardt Disease) –Pipeline by Astellas Pharma Inc
      • Table Juvenile Macular Degeneration (Stargardt Disease) –Pipeline by Copernicus Therapeutics Inc
      • Table Juvenile Macular Degeneration (Stargardt Disease) –Pipeline by Grupo Ferrer Internacional SA
      • Table Juvenile Macular Degeneration (Stargardt Disease) –Pipeline by Iris Pharma
      • Table Juvenile Macular Degeneration (Stargardt Disease) –Pipeline by Sanofi
    • Leber Congenital Amaurosis (LCA)
      • Table Leber Congenital Amaurosis (LCA) –Pipeline by AmpliPhi Biosciences Corp
      • Table Leber Congenital Amaurosis (LCA) –Pipeline by Editas Medicine Inc
      • Table Leber Congenital Amaurosis (LCA) –Pipeline by Novelion Therapeutics Inc
      • Table Leber Congenital Amaurosis (LCA) –Pipeline by ProQR Therapeutics NV
      • Table Leber Congenital Amaurosis (LCA) –Pipeline by Spark Therapeutics Inc
    • Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) –Pipeline by Alkeus Pharmaceuticals Inc
      • Table Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) –Pipeline by Biovista Inc
      • Table Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) –Pipeline by GenSight Biologics SA
      • Table Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) –Pipeline by Ixchel Pharma LLC
      • Table Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) –Pipeline by Khondrion BV
      • Table Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) –Pipeline by Mitotech SA
      • Table Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) –Pipeline by Spark Therapeutics Inc
      • Table Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) –Pipeline by Stealth BioTherapeutics Inc
    • Usher Syndrome
      • Table Usher Syndrome – Pipeline by Amgen Inc
      • Table Usher Syndrome – Pipeline by Editas Medicine Inc
      • Table Usher Syndrome – Pipeline by ProQR Therapeutics NV
      • Table Usher Syndrome – Pipeline by Sanofi
    • Retinitis Pigmentosa (Retinitis)
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Acucela Inc
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Allergan Plc
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Amarantus Bioscience Holdings Inc
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Amgen Inc
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Applied Genetic Technologies Corp
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Asklepios BioPharmaceutical Inc
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Astellas Pharma Inc
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Caladrius Biosciences Inc
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Dompe Farmaceutici SpA
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by GenSight Biologics SA
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Grupo Ferrer Internacional SA
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by ID Pharma Co Ltd
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by InFlectis BioScience
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Ionis Pharmaceuticals Inc
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by M's Science Corp
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Mimetogen Pharmaceuticals Inc
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Nanovector srl
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Novartis AG
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Novelion Therapeutics Inc
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by ProQR Therapeutics NV
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by ReNeuron Group Plc
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by SanBio Inc
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Sanofi
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Shire Plc
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Spark Therapeutics Inc
      • Table Retinitis Pigmentosa (Retinitis) –Pipeline by Sun Pharma Advanced Research Company Ltd
  • Dormant Projects
    • Juvenile Macular Degeneration (Stargardt Disease)
      • Table Juvenile Macular Degeneration (Stargardt Disease) –Dormant Projects
    • Leber Congenital Amaurosis (LCA)
      • Table Leber Congenital Amaurosis (LCA) –Dormant Projects
    • Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
      • Table Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) –Dormant Projects
    • Usher Syndrome
      • Table Usher Syndrome – Dormant Projects
    • Retinitis Pigmentosa (Retinitis)
      • Table Retinitis Pigmentosa (Retinitis) –Dormant Projects
  • Discontinued Products
    • Retinitis Pigmentosa (Retinitis)
      • Table Retinitis Pigmentosa (Retinitis) –Discontinued Products
  • Product Development Milestones
    • Juvenile Macular Degeneration (Stargardt Disease)
    • Leber Congenital Amaurosis (LCA)
    • Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
    • Usher Syndrome
    • Retinitis Pigmentosa (Retinitis)
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

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