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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