Most incurable forms of blindness are due to genetic diseases caused by faulty genes in the cells in the light-sensitive region of the eye, known as the retina. These defective genes eventually lead to the dysfunction and death of the affected cells. The progressive death of these cells causes a gradual degeneration of the retina, resulting in sight loss and ultimately blindness.
The promising results from their initial research enabled the team to commence a Phase 1/2 clinical trial in 2011 to test their AAV gene therapy for choroideremia. Following the positive outcomes observed in many of the choroideremia patients following treatment with the gene therapy, a gene therapy company (Nightstar) was spun out of their research programme in 2014 to expedite clinical evaluation of the choroideremia gene therapy and advance multiple gene therapies for inherited retinal disorders (all developed in the MacLaren laboratory) into human clinical trials. This enabled the team to commence a Phase 1/2 clinical trial in 2017 to test their AAV gene therapy for X-linked RP, followed by an international Phase 3 clinical trial of their choroideremia gene therapy in 2018.
On the back of this progress, Nightstar was acquired by Biogen for $877 million in early 2019 – just five years after its founding. The deal ranks as the third most valuable British biotech exit in the last two decades.
Funded by: National Institute for Health Research, Wellcome Trust, Medical Research Council and Nightstar Therapeutics