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    Gene therapy trial launched for X-linked retinitis pigmentosa


    Nightstar is a biopharmaceutical spinout company of Oxford developing gene therapies for multiple inherited retinal diseases.

    It has already reported promising preliminary results on a retinal gene therapy for choroideremia, an X-linked genetic disease that causes blindness in about 1 in 50,000 people.

    Choroidermia results from mutations in the CHM gene. In the results reported in 2014 in the Lancet, researchers administered 0.6–1.0×1010 genome particles in 1 eye each of 6 male patients aged 35-63 years.

    Despite undergoing retinal detachment, 2 patients with advanced choroideremia and low baseline best corrected visual acuity (BCVA) experienced significant improvement over 6 months. One gained 21 letters and the other gained 11 letters. The other 4 patients with near normal BCVA at baseline recovered to within 1 to 3 letters.

    The mean gain in visual acuity was 3.8 letters. Maximal light sensitivity measured with dark-adapted microperimetry increased from 23.0 dB at baseline to 25.3 dB in the treated eye.

    The mean increase in retinal sensitivity in the treated eyes correlated with the vector dose administered per square millimetre of surviving retina.

    Meanwhile, small non-significant reductions were noted in the control eyes in both maximal and mean sensitivity.

    More than 40 patients with choroideremia have received doses of AAV to date, according to the Nightstar website. Researchers have now followed the 6 patients for followed for 5 years, and they will have 2 years of data on another cohort this year.

    The company “expected to enter phase III pivotal testing, pending final discussions with the United States Food and Drug Administration [FDA],” according to the company’s website.

    Similar clinical trials of AAV as a treatment for choroideremia are underway at the University of Miami, Miami and the University of Pennsylvania, Philadelphia, both in the United States; and at the University of Alberta, Edmonton, Canada.

    Nightstar anticipates beginning a phase I/II clinical trial of gene therapy for macular dystrophy in late 2018.

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