Boost for RP stem cell research

31 October 2013

Stem cell research into retinitis pigmentosa (RP) was given a boost last month (Sept 2013) when the FDA in the US and the EMA granted ReNeuron, a UK company, orphan drug designation for its stem cell therapy, ReN003, for RP.

Drugs and treatments for rare diseases have a small and limited market so encouragement is needed for companies to focus on them by awarding orphan designation which gives benefits, such as reduced taxes and market protection support. An orphan designation is given to potential treatments for rare conditions that are life-threatening or chronically debilitating.

The designation given to ReNeuron will support their plan to launch a Phase I/II clinical trials for ReN003 in mid-2014 in the UK and the US. The company is partnering with the Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, to develop the treatment.

Dr Dolores Conroy, Director of Research at Fight for Sight said; “So many eye diseases and conditions can be defined as rare and qualify for orphan status. It is important that the FDA and EMA continue to recognise developments in treatments for rare eye diseases. This emerging treatment, ReN003, involves the transplantation of retinal progenitor cells, which are more mature than embryonic stem cells, but haven’t completely developed into photoreceptors, the cells in the retina that make vision possible. We look forward to the results of the Phase I/II trial.”

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