The gene therapy is an AAV8 vector-based gene therapy...

The gene therapy is an AAV8 vector-based gene therapy administered by subretinal injection. It delivers a full-length functioning retinitis pigmentosa GTPase regulator (RPGR) protein in XLRP patients whose disease is caused by mutations in the RPGR gene. The theory is that this will lead to increased levels of the RPGR protein, which might slow, stop or prevent additional degradation of photoreceptors in these patients.

The study used a single subretinal injection of the therapy in males with genetically confirmed XLRP. Part I of the study ran for 24 months and was a dose-escalation trial. Part II was a 12-month dose-expansion study using a high and low dose chosen from Part I and a third untreated group as a control cohort.

Treated patients in Parts I and II were invited to take part in a separate long-term follow-up trial that will track efficacy and safety data up to five years after treatment.

I wasn't aware anyone else was working on RP. Not sure if this is good or bad news.


https://www.biospace.com/article/biogen-s-gene-therapy-for-eye-disease-flunks-phase-ii-iii-trial/?utm_campaign=GenePool&utm_medium=email&_hsmi=127207788&_hsenc=p2ANqtz-8QkyjAdHXewfzKojK0NiaUEm7uBWGy1o1Ln6SYi_9n0kBi5cfIpjSLInbB2TArqgYRnskIg26hs_UkSd6wAOjBYQJrqQ&utm_content=127207788&utm_source=hs_email