--Must See--

Bioinformatics Summer Internship 2024 With Hands-On-Training + Project / Dissertation - 30 Days, 3 Months & 6 Months Duration

Oxford Spinout, Nightstar Therapeutics Debuts On Nasdaq With $75M IPO

Since the mutation causing Choroideremia is sex-linked, sufferers are almost always males, with the onset of symptoms in childhood often starting with night-blindness. The CHM gene is responsible for encoding the Rab-escort protein 1 in retinal cells, a mutant version of which is responsible for vision deterioration. You can read more about this rare disease through the Choroideremia Research Foundation.

Developed at Oxford’s Nuffield Lab of Ophthalmology in the UK, Nightstar has a gene therapy which uses an adeno-associated retroviral vector (AAV). This vector delivers a wild-type of the defective gene (CHM) into the retinal cells of the patient, to produce functional REP proteins and partially restore vision.

The spin out has now gone public on the American market with the aim of raising $75M for its gene-based retinal disease cures.

Oxford Spinout, Nightstar Therapeutics Debuts On Nasdaq With $75M IPO

Nightstar’s candidate for choroideremia demonstrated its efficacy in improving the vision of 6 patients in a Phase I/II trial three and a half years after treatment just last year, and the company has been raising a lot of funds to push it into a pivotal study.

The spinout, which was initially acknowledged as Nightstarx but rebranded in advance of the IPO, is developing treatment options for scarce inherited retinal

circumstances. Its guide prospect, NSR-REP1, targets choroideremia, a retinal dystrophy that gradually impairs vision up to blindness, and for which there are no effective treatment options now on the market.

The treatment takes advantage of a modified virus – AAV.REP1 – to accurate genetic info in cells. It is injected into the retina below area anaesthesia. A phase 1 and 2 scientific demo carried out by University of Oxford has now verified the long-time period rewards of NSR-REP1, like enhancement in vision.

The IPO proceeds will go to the completion of a phase 3 demo of the remedy for choroideremia. The funds will also make it possible for Nightstar to advance additional drug candidates by means of early-stage scientific trials.

In search of the perfect burger. Serial eater. In her spare time, practises her "Vader Voice". Passionate about dance. Real Weird.