List of Tables
Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease) 12
Number of Products under Development by Companies, Juvenile Macular Degeneration (Stargardt Disease) 13
Number of Products under Development by Universities/Institutes, Juvenile Macular Degeneration (Stargardt Disease) 14
Products under Development by Companies, Juvenile Macular Degeneration (Stargardt Disease) 14
Products under Development by Universities/Institutes, Juvenile Macular Degeneration (Stargardt Disease) 15
Number of Products under Development for Leber Congenital Amaurosis (LCA) 16
Number of Products under Development by Companies, Leber Congenital Amaurosis (LCA) 17
Number of Products under Development by Universities/Institutes, Leber Congenital Amaurosis (LCA) 18
Products under Development by Companies, Leber Congenital Amaurosis (LCA) 19
Products under Development by Universities/Institutes, Leber Congenital Amaurosis (LCA) 19
Number of Products under Development for Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 20
Number of Products under Development by Companies, Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 21
Number of Products under Development by Universities/Institutes, Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 22
Products under Development by Companies, Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 22
Products under Development by Universities/Institutes, Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 23
Number of Products under Development for Usher Syndrome 23
Number of Products under Development by Companies, Usher Syndrome 24
Number of Products under Development by Universities/Institutes, Usher Syndrome 25
Products under Development by Companies, Usher Syndrome 25
Products under Development by Universities/Institutes, Usher Syndrome 25
Number of Products under Development for Retinitis Pigmentosa (Retinitis) 26
Number of Products under Development by Companies, Retinitis Pigmentosa (Retinitis) 28
Number of Products under Development by Universities/Institutes, Retinitis Pigmentosa (Retinitis) 29
Products under Development by Companies, Retinitis Pigmentosa (Retinitis) 30
Products under Development by Universities/Institutes, Retinitis Pigmentosa (Retinitis) 32
Number of Products by Stage and Target, Juvenile Macular Degeneration (Stargardt Disease) 34
Number of Products by Stage and Mechanism of Action, Juvenile Macular Degeneration (Stargardt Disease) 36
Number of Products by Stage and Route of Administration, Juvenile Macular Degeneration (Stargardt Disease) 38
Number of Products by Stage and Molecule Type, Juvenile Macular Degeneration (Stargardt Disease) 40
Number of Products by Stage and Target, Leber Congenital Amaurosis (LCA) 42
Number of Products by Stage and Mechanism of Action, Leber Congenital Amaurosis (LCA) 44
Number of Products by Stage and Route of Administration, Leber Congenital Amaurosis (LCA) 46
Number of Products by Stage and Molecule Type, Leber Congenital Amaurosis (LCA) 48
Number of Products by Stage and Target, Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 49
Number of Products by Stage and Mechanism of Action, Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 51
Number of Products by Stage and Route of Administration, Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 53
Number of Products by Stage and Molecule Type, Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 55
Number of Products by Stage and Target, Usher Syndrome 56
Number of Products by Stage and Mechanism of Action, Usher Syndrome 58
Number of Products by Stage and Route of Administration, Usher Syndrome 60
Number of Products by Stage and Molecule Type, Usher Syndrome 62
Number of Products by Stage and Target, Retinitis Pigmentosa (Retinitis) 64
Number of Products by Stage and Mechanism of Action, Retinitis Pigmentosa (Retinitis) 66
Number of Products by Stage and Route of Administration, Retinitis Pigmentosa (Retinitis) 68
Number of Products by Stage and Molecule Type, Retinitis Pigmentosa (Retinitis) 70
Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Acucela Inc 71
Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Alkeus Pharmaceuticals Inc 71
Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Astellas Pharma Inc 72
Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Copernicus Therapeutics Inc 72
Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Grupo Ferrer Internacional SA 73
Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Iris Pharma 73
Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Sanofi 74
Leber Congenital Amaurosis (LCA) – Pipeline by AmpliPhi Biosciences Corp 74
Leber Congenital Amaurosis (LCA) – Pipeline by Editas Medicine Inc 75
Leber Congenital Amaurosis (LCA) – Pipeline by Novelion Therapeutics Inc 75
Leber Congenital Amaurosis (LCA) – Pipeline by ProQR Therapeutics NV 76
Leber Congenital Amaurosis (LCA) – Pipeline by Spark Therapeutics Inc 76
Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) – Pipeline by Alkeus Pharmaceuticals Inc 77
Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) – Pipeline by Biovista Inc 77
Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) – Pipeline by GenSight Biologics SA 78
Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) – Pipeline by Ixchel Pharma LLC 78
Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) – Pipeline by Khondrion BV 79
Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) – Pipeline by Mitotech SA 79
Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) – Pipeline by Spark Therapeutics Inc 80
Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) – Pipeline by Stealth BioTherapeutics Inc 80
Usher Syndrome – Pipeline by Amgen Inc 81
Usher Syndrome – Pipeline by Editas Medicine Inc 81
Usher Syndrome – Pipeline by ProQR Therapeutics NV 82
Usher Syndrome – Pipeline by Sanofi 82
Retinitis Pigmentosa (Retinitis) – Pipeline by Acucela Inc 83
Retinitis Pigmentosa (Retinitis) – Pipeline by Allergan Plc 83
Retinitis Pigmentosa (Retinitis) – Pipeline by Amarantus Bioscience Holdings Inc 84
Retinitis Pigmentosa (Retinitis) – Pipeline by Amgen Inc 84
Retinitis Pigmentosa (Retinitis) – Pipeline by Applied Genetic Technologies Corp 85
Retinitis Pigmentosa (Retinitis) – Pipeline by Asklepios BioPharmaceutical Inc 85
Retinitis Pigmentosa (Retinitis) – Pipeline by Astellas Pharma Inc 86
Retinitis Pigmentosa (Retinitis) – Pipeline by Caladrius Biosciences Inc 86
Retinitis Pigmentosa (Retinitis) – Pipeline by Dompe Farmaceutici SpA 87
Retinitis Pigmentosa (Retinitis) – Pipeline by GenSight Biologics SA 87
Retinitis Pigmentosa (Retinitis) – Pipeline by Grupo Ferrer Internacional SA 88
Retinitis Pigmentosa (Retinitis) – Pipeline by ID Pharma Co Ltd 88
Retinitis Pigmentosa (Retinitis) – Pipeline by InFlectis BioScience 89
Retinitis Pigmentosa (Retinitis) – Pipeline by Ionis Pharmaceuticals Inc 89
Retinitis Pigmentosa (Retinitis) – Pipeline by M’s Science Corp 90
Retinitis Pigmentosa (Retinitis) – Pipeline by Mimetogen Pharmaceuticals Inc 90
Retinitis Pigmentosa (Retinitis) – Pipeline by Nanovector srl 91
Retinitis Pigmentosa (Retinitis) – Pipeline by Novartis AG 91
Retinitis Pigmentosa (Retinitis) – Pipeline by Novelion Therapeutics Inc 92
Retinitis Pigmentosa (Retinitis) – Pipeline by ProQR Therapeutics NV 92
Retinitis Pigmentosa (Retinitis) – Pipeline by ReNeuron Group Plc 93
Retinitis Pigmentosa (Retinitis) – Pipeline by SanBio Inc 93
Retinitis Pigmentosa (Retinitis) – Pipeline by Sanofi 94
Retinitis Pigmentosa (Retinitis) – Pipeline by Shire Plc 94
Retinitis Pigmentosa (Retinitis) – Pipeline by Spark Therapeutics Inc 95
Retinitis Pigmentosa (Retinitis) – Pipeline by Sun Pharma Advanced Research Company Ltd 95
Juvenile Macular Degeneration (Stargardt Disease) – Dormant Projects 96
Leber Congenital Amaurosis (LCA) – Dormant Projects 96
Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) – Dormant Projects 96
Usher Syndrome – Dormant Projects 97
Retinitis Pigmentosa (Retinitis) – Dormant Projects 97
Retinitis Pigmentosa (Retinitis) – Discontinued Products 99