The Pharmaletter

One To Watch

Nightstar Therapeutics

Nightstar is a clinical-stage company focused on developing and commercializing a pipeline of novel and potentially curative, one-time retinal gene therapies for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness, and, for which, there are no currently approved treatments.

The UK company's lead product, NSR-REP1, is being developed for the treatment of choroideremia, a rare, degenerative, X-linked genetic retinal disorder primarily affecting males that is caused by a mutation in the CHM gene.

In June 2018, NSR-REP1 received a Regenerative Medicine Advanced Therapy designation from the Food and Drug Administration.

Nightstar is one of the portfolio companies of life sciences investment trust, Syncona.

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