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Orphan Drugs: FDA Designation For NightstaRx’s Gene Therapy For Choroideremia

November 14, 2014

NightstaRx Ltd (“Nightstar”) is created in January 2014, as a spin-out from the University of Oxford (UK) and its research commercialization company, Isis Innovation. NightstaRx is focusing on developing and commercializing treatments for retinal dystrophies (degenerative conditions affecting vision).

NightstaRx’s first program is for a gene therapy for Choroideremia, an inherited form of progressive blindness. The gene therapy uses a modified virus, AAV.REP1, for delivering the correct version of the Choroideremia (CHM) gene to cells in the eye’s retina. AAV.REP1 is developed by a team of researchers led by Professor Robert MacLaren at the University of Oxford. In January 2014, a successful Phase I/II clinical trial is published in The Lancet journal.

Choroideremia is a rare genetic disorder of sight that includes progressive loss of peripheral field of vision and night blindness. It usually affects males and female carriers may have mild symptoms without loss of vision.

NightstaRx receives on November 5th, FDA Orphan Drug Designation (ODD) for its gene therapy for the rare eye disease, Choroideremia.

FDA ODD Database Record For NightstaRx’s Gene Therapy

Generic Name: Adeno-associated viral vector serotype 2 containing the human Rab escort protein 1 gene
Trade Name: n/a
Date Designated: 11-05-2014
Orphan Designation: Choroideremia
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: NightstaRx Ltd. 215 Euston Road NW1 2BE London, UK


Please Note: “Eye Iris XX” by che (“Petr Novák, Wikipedia”) [CC-BY-SA-3.0] | Wikimedia Commons.

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