Huntington's disease (ReN005)

Due to the relatively small patient population, we intend to target our cell therapy treatment for Huntington's disease, ReN005, exclusively as an Orphan Drug status treatment.

Market opportunity

Huntington's disease is an uncommon, inherited, progressive and fatal neurodegenerative disorder. In the US, approximately 35,000 patients show overt signs of the disease, with a further 75,000 carrying the abnormal gene. There are no existing treatments for the disease.

Progress

We have generated positive pre-clinical efficacy data in a Huntington's disease model with a cell line derived from the striatum region of the brain. This cell line is in pre-clinical development and the programme is being pursued in collaboration with academic and commercial partners under a UK DTI Technology Programme grant.

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