First in-human XLRP clinical trial

The results of the first human trial investigating a gene therapy to treat X-linked Retinitis Pigmentosa have been published in Nature Medicine. X-linked Retinitis Pigmentosa (XLRP) is the most common form of recessive RP, distinguished by the degeneration of rod and cone photoreceptors in early childhood, which results in severe vision loss. The RPGR gene which has the mutations causing XLRP is found on the X-chromosome and primarily affects men.

This paper describes the results from the first human RPGR-targeted gene therapy trials, whereby 18 patients were recruited and separated into six groups of 3 individuals. The adeno-associated viral vector targeting the RPGR gene was injected subretinally and visual function was monitored over a 6 month period. Administration of this gene therapy improved visual acuity which was maintained in a 6 month follow-up.

Although these are early stage results, the findings indicate that the AAV-RPGR gene therapy is capable of reversing vision loss and shows very promising signs for developing X-linked Retinitis Pigmentosa treatments in the future.

Read the full paper here:

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