Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, whether he has undertaken any research on the potential effectiveness of gene therapy in treating pigmentosa.

The National Institute for Health Research (NIHR) contributes to retinitis pigmentosa research, including potential gene therapies. In the past five years, there have been five projects related to the condition directly funded through NIHR programmes and 22 studies supported by the NIHR’s infrastructure.

In October 2019, the National Institute for Health and Care Excellence (NICE) recommended voretigene neparvovec, an adeno-associated virus vector-based gene therapy, for treating inherited retinal dystrophies caused by RPE65 gene mutations. NICE has two additional pieces of guidance on potential gene therapies for retinal conditions currently in the development and proposal stages.