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Trustees of Columbia University in the city of New York — Rare Disease Pipeline | Kisho | Kisho

Trustees of Columbia University in the city of New York

Other

2

Orphan Designations

0

FDA Approvals

8

Rare Diseases

0

News Articles

Disease	Drug(s)	Designation	Approved
PDE6A-related retinopathy	Recombinant adeno associated virus vector targeting human PHD 2 protein	Orphan Designation	-
PDE6C-related retinopathy	Recombinant adeno associated virus vector targeting human PHD 2 protein	Orphan Designation	-
PDE6G-related retinopathy	Recombinant adeno associated virus vector targeting human PHD 2 protein	Orphan Designation	-
isolated microphthalmia 5	Adeno-Associated Virus Serotype 8 carrying the Membrane Frizzled Related Protein (MFRP) transgene	Orphan Designation	-
nanophthalmia	Adeno-Associated Virus Serotype 8 carrying the Membrane Frizzled Related Protein (MFRP) transgene	Orphan Designation	-
nanophthalmos 2	Adeno-Associated Virus Serotype 8 carrying the Membrane Frizzled Related Protein (MFRP) transgene	Orphan Designation	-
retinal cone dystrophy 3A	Recombinant adeno associated virus vector targeting human PHD 2 protein	Orphan Designation	-
retinitis pigmentosa 4	Recombinant adeno associated virus vector targeting human PHD 2 protein	Orphan Designation	-

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