Recently, NightstaRx Limited, an eye disease treatment company based in London, UK, announced the completion of a $45 million Series C round of financing. Participants in this round of financing include the new investment institutions Wellington Management Company, Redmile Group and the company's existing investors Syncona and New Enterprise Associates.
Founded in 2013, NightstaRx Limited ("Nightstar") is a post-biopharmaceutical development company focused on the development of gene therapies for the treatment of hereditary retinal diseases. Nightstar currently has four research drugs, mainly for choroideremia, X-linked retinitis pigmentosa, and macular Degeneration.
(Nightstar product pipeline, image source: company official website)
The company's primary drug candidate, NSR AAV-REP1, is primarily used for advanced and critical clinical examination of choroidal disorders. No choroidal disease, also known as total choroidal vascular atrophy, is characterized by progressive onset of both eyes, from childhood blindness, diffuse full-thickness choroidal capillaries and RPE atrophy, and finally the patient's choroid disappears completely and leads to blindness. Currently, there is no effective way to treat this disease.
Nightstar's NSR AAV-REP1 therapy is expected to provide a one-time treatment for choroid-free disease by delivering a wild-type copy of the REP-1 gene (AAV-REP1) to the patient's retinal cells with a viral vector of adeno-associated virus (AAV). . The procedure is performed under local anesthesia, involving standard ophthalmic surgery to separate the patient's retina, and then injecting approximately one-tenth of a milliliter of fluid through a very narrow needle, all of which are performed by experienced retinal surgeons. The patient's risk is relatively small.
(Source: company's official website)
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