Ann: Presentation of Clinical Proof of Concept in Lead Program, page-32

I am not qualified to fully understand the difference between RP11 and XLRP but thought it worth posting this as it very much relates to what we are trying to achieve, and demonstrates that large Pharma do have a history in this area.

Johnson & Johnson bought the full rights to a rare disease gene therapy a few years ago, but now the Big Pharma may be experiencing buyer’s remorse. The treatment failed to improve the vision-guided mobility of patients with the eye disease X-linked retinitis pigmentosa (XLRP) in a phase 3 trial, according to resultsshared May 2.

TheLUMEOS trialenrolled 95 patients, 58 of whom received either a low or high single dose of botaretigene sparoparvovec (bota-vec), an investigational gene therapy that uses an adeno-associated virus to shuttle a functional copy of the retinitis pigmentosa GTPase regulator (RPGR) gene to the retina.

XLRP is a severe and rare form of retinitis pigmentosa, a progressive eye disease that causes photoreceptors to break down over time, eventually leading to blindness. The disease often manifests in childhood and mainly affects boys, with the LUMEOS trial only enrolling a handful of women with the disease.

In a pooled analysis of all who received bota-vec, the trial’s primary endpoint of improvement in the ability of patients to visually navigate through a virtual maze was not met, though J&J said the result was “directionally supportive."

This below is from my small amount of research, assisted by Dr Google obviously