Nightstar goes public to finance Phase 3 retinal gene therapy trials

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London based Oxford University spinout Nightstar Therapeutics has filed an Initial Public Offering (IPO) with Nasdaq which aims to raise up to $86 million to fund Phase 3 trialing of the company’s gene therapy, NSR-REP1, for the hereditary, degenerative retinal disease, choroideremia.

Earlier trials of NSR-REP1 found 90% of the 32 subject cohort maintained or improved vision acuity, maintained a year after the initial treatment. The lead product candidate comprises of an adeno associated viral (AAV) vector carrying the Rab escort protein 1 gene, encoding of which is mutated in people with choroideremia. The positive trial results to date have paved the way for Nightstar to expand their investigations into Phase 3, with 140 patients. This would set the company ahead of rival Spark Therapeutics who are still undergoing Phase 1/2 trials.

Nightstar has attracted significant investments to date, including $12 million from Syncona, and most recently $45 million in a Series C financing round. The IPO is new ground for Nightstar whose funding thus far has come from private rounds. The company will hope that public investors are receptive to their offering, in a similar vein to the successes of blulebird bio and Spark on the public market.

Source: Nightstar files for $86M IPO to fund gene therapy trials Press Release