UK stem cell specialist ReNeuron has filed for approval to start clinical trials of a treatment for the genetic eye disease retinitis pigmentosa (RP) in the US.
The company is asking the FDA for approval to carry out a phase I/II trial of its human retinal progenitor cell (hRPC) line at Massachusetts Eye and Ear clinic in Boston, one of the leading centres for the study of retinal diseases.
RP is a group of hereditary diseases of the eye that lead to progressive loss of sight due to cells in the retina becoming damaged and eventually dying, according to the company.
Preclinical studies have shown that when implanted into the retina, hRPC cells – also known as ReN003 – can not only prevent further degeneration but also colonise the retina and form new light receptor cells, which potentially could lead to restoration of vision.
ReNeuron is already conducting trials of a neural stem cell product called CTX in stroke (phase II) and critical limb ischaemia (phase I). Taking a second stem cell type into the clinic – in what will be its first trial in the US – is a major milestone in what is shaping up to be an eventful 2015 for the company.
Preliminary results from the PISCES trial of CTX – which is trying to show a recovery in motor skills in stroke patients with stable upper limb disability – are due later this year and the trial could complete early in 2016.
Thanks to legislation passed in 2013 in Japan to allow conditional approval of stem cell therapies, ReNeuron could potentially press ahead with a regulatory filing on the strength of phase II data, according to analysts at Edison.
Meanwhile, ReNeuron will shortly move into a 28,000 sq. ft. purpose-built cell manufacturing and research facility in Wales, which will support its clinical programmes and also allow it to make commercial quantities of its stem cells in-house.
The Guildford-based company is hoping to get approval to start the open-label, dose-escalation trial of hRPC later this year, with an enrolment target of 15 RP patients.
The stem cells will be delivered via a single sub-retinal injection with the patients followed for 12 months to assess the treatment’s safety and provide initial read-outs of its impact on visual acuity.